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CDE Includes Double-Crane’s Hydroxocobalamine and Sanofi Genzyme’s Fitusiran in Rare Disease Care Plan
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China’s Center for Drug Evaluation (CDE) has announced the inclusion of China Resources Double-Crane Pharmaceutical Co., Ltd’s (SHA: 600062) hydroxocobalamine and Sanofi (NASDAQ: SNY) Genzyme’s fitusiran in its patient-centered rare disease drug development pilot program, known as the Care Plan. Hydroxocobalamine for Metabolic Disorders in Children with MMAHydroxocobalamine, also referred…
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US FDA Grants ODD to Ractigen’s siRNA Therapy RAG-21 for ALS Treatment
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China-based small activating RNA (saRNA) drug developer, Ractigen Therapeutics, has announced that it has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for its drug candidate RAG-21. This FUS gene-targeted siRNA therapy is indicated for the treatment of amyotrophic lateral sclerosis (ALS), an incurable and…
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Exegenesis Bio’s Gene Therapy EXG110 Earns Orphan Drug Status for Fabry Disease
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Exegenesis Bio, a leading provider of epigenetics experimental and analytical services headquartered in Hangzhou, has announced that it has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for its proprietary gene therapy, EXG110, which is indicated for the treatment of Fabry disease. This designation highlights…
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ETERN Therapeutics’ ETS-006 Receives FDA Orphan Drug Designation for Pleural Mesothelioma
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ETERN Therapeutics, a Shanghai-based developer of small-molecule anti-tumor drugs, has announced that it has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for its compound ETS-006. The designation is for the treatment of pleural mesothelioma (PM), a rare and aggressive form of cancer. Development and…
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Ascentage Pharma’s Lisaftoclax and AceLink Therapeutics’ AL01211 Receive FDA Designations
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The Center for Drug Evaluation (CDE) website has indicated that China-based Ascentage Pharma’s (HKG: 6855) lisaftoclax (APG-2575) and AceLink Therapeutics(Suzhou)’s AL01211 are on track to receive priority review and breakthrough therapy designation (BTD) statuses respectively from the US Food and Drug Administration (FDA). Lisaftoclax, an oral BCL-2 inhibitor, is indicated…
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AskBio’s AB-1003 Receives Rare Pediatric Disease and Orphan Drug Designations from FDA
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Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned subsidiary of Bayer AG (ETR: BAYN), has announced that it has received both rare pediatric disease designation (RPDD) and orphan-drug designation (ODD) from the US Food and Drug Administration (FDA) for its investigational gene therapy, AB-1003 (LION-101). The therapy targets limb-girdle muscular dystrophy…
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Belief BioMed’s BBM-D101 Receives FDA Orphan Drug and Pediatric Rare Disease Designations for DMD
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Belief BioMed has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) and Pediatric Rare Disease Designation (RPDD) for its gene therapy drug, BBM-D101 injection, indicated for the treatment of Duchenne muscular dystrophy (DMD). BBM-D101: A Pioneering rAAV Gene Therapy for Duchenne Muscular DystrophyBBM-D101…
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Innorna Co., Ltd Receives FDA Rare Pediatric Disease Designation for mRNA Drug IN015
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Shenzhen-based Innorna Co., Ltd has announced that it has received Rare Pediatric Disease Designation (RPDD) from the US Food and Drug Administration (FDA) for its proprietary mRNA drug, IN015, developed for the treatment of progressive familial intrahepatic cholestasis (PFIC). Understanding PFIC and the Impact of IN015Progressive familial intrahepatic cholestasis (PFIC)…
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AstraZeneca’s Fasenra Approved by EU for Eosinophilic Granulomatosis Treatment
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UK pharmaceutical giant AstraZeneca Inc. (NASDAQ: AZN) has received marketing approval from the European Commission for its drug Fasenra (benralizumab) as an additional treatment for adult patients suffering from relapsing or refractory eosinophilic granulomatosis with polyangiitis (EGPA). The approval follows the successful outcomes of the MANDARA Phase III trial, marking…
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RemeGen’s Telitacicept Receives Priority Review Status from China’s CDE for gMG Treatment
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RemeGen (HKG: 9995), a China-based pharmaceutical company, has announced that the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) has accepted another market filing for its drug telitacicept with priority review status. The filing seeks approval for the treatment of generalized myasthenia gravis (gMG), a serious…
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Novo Nordisk’s Alhemo Receives CHMP Recommendation for Haemophilia Prophylaxis in Europe
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Novo Nordisk (NYSE: NVO), a Danish pharmaceutical company, has been notified that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the approval of Alhemo (concizumab). This would make Alhemo the first once-daily subcutaneous prophylactic treatment for individuals aged 12 years or…
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Jacobio Pharma’s G12C Inhibitor Gecirasib Earns Orphan Drug Designation in Europe for Pancreatic Cancer
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Jacobio Pharma (HKG: 1167), a China-based biopharmaceutical company, has announced that it has received Orphan Drug Designation (ODD) from the European Medicine Agency (EMA) for its KRAS G12C inhibitor, glecirasib, for the treatment of pancreatic cancer. This designation follows previous awards for glecirasib, including ODD in the US and Breakthrough…
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Johnson & Johnson’s Nipocalimab Shows Promise in Treating Adolescent Myasthenia Gravis in Phase II/III Trial
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Johnson & Johnson (J&J, NYSE: JNJ), a US pharmaceutical giant, has reported encouraging outcomes from its Phase II/III clinical trial, Vibrance-MG, which evaluated the efficacy of nipocalimab in treating adolescents with generalized myasthenia gravis (gMG) who tested positive for anti-AChR antibodies. The study successfully achieved its primary and secondary endpoints,…
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Pfizer’s Hympavzi Secures FDA Nod, Offering Novel Once-Weekly Treatment for Hemophilia B
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Pfizer Inc. (NYSE: PFE) has scored a significant win with the US Food and Drug Administration’s (FDA) approval of its drug Hympavzi (marstacimab-hncq) for routine prophylaxis in adults and pediatric patients aged 12 and older with hemophilia A, characterized by a congenital deficiency of factor VIII (FVIII), or hemophilia B,…
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Biogen’s China Unit Gets Conditional NMPA Nod for Qalsody (Tofersen) in ALS Treatment
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Biogen (NASDAQ: BIIB)’s Chinese subsidiary has announced that it has received conditional approval from the National Medical Products Administration (NMPA) for its drug Qalsody (tofersen) for the treatment of adult patients with amyotrophic lateral sclerosis (ALS) who have a superoxide dismutase 1 (SOD1) gene mutation. ALS is a rare disease,…
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China’s CDE Announces “Care Plan” to Include Pfizer’s Marstaximab for Rare Disease Drug Development
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The National Health Commission (NHC) of China, through its Center for Drug Evaluation (CDE), has announced that Pfizer (NSE: PFIZER, NYSE: PFE, ETR: PFE)’s Marstaximab (PF-06741086) has been included in the “Care Plan” for patient-centered rare disease drug development. The publicity period for this decision ends on October 12, 2024.…
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Beijing Aims to Establish Rare Disease Drug Security Pilot Zone with ‘White List’ System
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Beijing is set to follow in the footsteps of Boao Lecheng with the release of the “Implementation Plan for Promoting the Construction of Rare Disease Drug Security Pilot Zone in Beijing (Trial) (draft proposal),” which concluded its public feedback period on August 13. The two-year policy initiative aims to establish…
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AstraZeneca’s Fasenra Secures FDA Nod for EGPA Treatment, Strengthening Rare Disease Portfolio
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UK pharmaceutical giant AstraZeneca (NASDAQ: AZN, LON: AZN) has announced that the US Food and Drug Administration (FDA) has approved a new indication for its interleukin-5 (IL-5)-targeted antibody, Fasenra (benralizumab), for the treatment of eosinophilic granulomatosis with polyangiitis (EGPA), a rare and potentially fatal disease. The approval comes on the…
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CDE Launches CARE Plan to Streamline Rare Disease Drug Development
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The Center for Drug Evaluation has unveiled the ‘Pilot Work Plan for Patient-Centered Action for Rare Diseases Encouragement (CARE),’ following a public consultation period in May 2024. The final document retains the initial proposal’s structure, segmenting the drug R&D lifecycle into stages A through E, with the anticipation of including…
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Suzhou GenAssist Launches First-of-Its-Kind Base Editing Drug Trial for Duchenne Muscular Dystrophy
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Suzhou GenAssist Therapeutic Co., Ltd, a biopharmaceutical company based in China, has announced the commencement of an investigator-initiated-trial (ITT) for its base editing drug candidate, GEN6050X, in patients afflicted with Duchenne muscular dystrophy (DMD). DMD is a rare genetic disorder linked to the X-chromosome, typically caused by mutations in the…
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Jiangsu Hengrui’s ANGPTL3 Inhibitor SHR-1918 Earns Breakthrough Designation for HoFH Treatment
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Jiangsu Hengrui Medicine Co., Ltd (SHA: 600276), a leading pharmaceutical company in China, has announced that it has received Breakthrough Therapy Designation (BTD) from China’s Center for Drug Evaluation (CDE) for its drug candidate SHR-1918. This in-house developed monoclonal antibody (mAb) targeting angiopoietin-like protein 3 (ANGPTL3) is intended for the…
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Vcanbio’s Stem Cell Therapy VUM02 Gets NMPA Approval for Systemic Sclerosis Clinical Trial
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Vcanbio Cell & Gene Engineering Corp., Ltd (SHA: 600645), a biotechnology company based in China, has received approval from the National Medical Products Administration (NMPA) to initiate a clinical study for its Category 1 therapeutic biologic product, VUM02. This product, derived from human umbilical cord mesenchymal stem cells, is designed…
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AccurEdit Therapeutics’ Gene-Editing Drug ART001 Clears FDA Hurdle for US Clinical Trials
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SUZHOU—AccurEdit Therapeutics (Suzhou) Co., Ltd, a leading Chinese specialist in gene editing therapies, has announced that it has received approval from the US Food and Drug Administration (FDA) for its investigational drug ART001, which targets transthyretin amyloidosis. This milestone marks ART001 as the first in vivo gene-edited drug based on…
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Tonghua Golden-Horse, UCB Seek China Approvals for Alzheimer’s, Myasthenia Gravis Drugs
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China’s Tonghua Golden-Horse Pharmaceutical Industry Co. Ltd. and Belgium-based UCB (FRA: UNC) are seeking marketing approvals in China for two advanced treatments targeting neurodegenerative and autoimmune conditions, according to the Center for Drug Evaluation (CDE) website. Tonghua Golden-Horse’s octohydroaminoacridine succinate, designed to treat mild to moderate Alzheimer’s disease (AD), and…
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China’s Supreme Court Deals Blow to Sanofi’s Aubagio, Confirms Patent Cancellation
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BEIJING—In a recent development for the pharmaceutical industry, Sanofi’s multiple sclerosis therapy Aubagio (teriflunomide) has suffered a significant setback in China. According to the latest information, three decisions from China’s Supreme Court in May 2024 have upheld the cancellation of Sanofi’s formulation patents for Aubagio tablets, as requested by an…
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Gene Cradle Initiates First Regulatory Gene Therapy Study in China for Late-Onset Pompe Disease
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Gene Cradle, a Beijing-based gene therapy specialist, has achieved a milestone by administering its GC301 to the first patient with late-onset Pompe disease (LOPD) in a regulatory study in China. This marks the nation’s first patient dosing of an adeno-associated virus (AAV) gene therapy for LOPD. GC301 is an innovative…
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AstraZeneca’s Strensiq Approved for Rare Disease Therapy under Greater Bay Area’s Drug Access Policy
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AstraZeneca (NASDAQ: AZN, LON: AZN) , a leading UK pharmaceutical company, has received approval for its rare disease therapy Strensiq (asfotase alfa) from the Guangdong Medical Products Administration (MPA) bureau. This clearance allows Strensiq to be introduced to medical institutions under the “Hong Kong-Macau Drug and Device Access” policy, a…
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AstraZeneca’s Strensiq Gains Approval for Rare Disease Therapy in Guangdong Under “Hong Kong-Macau Drug and Device Access”
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LONDON—AstraZeneca (NASDAQ: AZN, LON: AZN) , a leading UK-based pharmaceutical company, has announced that its rare disease therapy Strensiq (asfotase alfa) has been granted market access by the Guangdong Medical Products Administration (MPA) bureau. This approval is a significant step under the “Hong Kong-Macau Drug and Device Access” policy, which…
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Ascendis Pharma’s Hypoparathyroidism Therapy Yorvipath Wins FDA Nod, Fills Market Gap
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This week, the U.S. Food and Drug Administration (FDA) granted market approval to Ascendis Pharma A/S (NASDAQ: ASND), a Denmark-based pharmaceutical company specializing in rare diseases, for its hypoparathyroidism treatment, palopegteriparatide. The therapy, to be marketed as Yorvipath, is a once-daily prodrug of parathyroid hormone (PTH[1-34]) designed to address the…
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Zelgen Biopharmaceuticals’ ZG006 Earns Orphan Drug Designation from FDA for Small-Cell Lung Cancer
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Suzhou Zelgen Biopharmaceuticals Co., Ltd (SHA: 688266), a biopharmaceutical company based in China, has announced that it has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for its pipeline candidate, ZG006, which is under development for the treatment of small-cell lung cancer (SCLC). ZG006 is…
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Insilico Medicine’s ISM6331 Earns FDA Orphan Drug Designation for Mesothelioma Treatment
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Insilico Medicine, a China-based biotechnology company at the forefront of generative artificial intelligence (AI), has announced that it has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for its pipeline candidate ISM6331. This potential best-in-class candidate drug is designed to treat mesothelioma, a rare and…
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BioNTech’s mRNA Cancer Immunotherapy BNT111 Achieves Primary Endpoint in Phase II Trial
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Germany-based BioNTech SE (NASDAQ: BNTX) has announced that its mRNA cancer immunotherapy BNT111, in combination with the programmed death-1 (PD-1) inhibitor Libtayo (cemiplimab), has achieved the primary endpoint in a Phase II clinical trial. The study, part of a collaboration with Regeneron, is assessing the combination and monotherapies in treating…
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Organon’s Liptruzet Launches in China for Hypercholesterolemia Treatment
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US pharmaceutical company Organon (NYSE: OGN) has announced the official market launch of its single-tablet compound preparation Liptruzet (ezetimibe, torvastatin) in China for the treatment of hypercholesterolemia and homozygous familial hypercholesterolemia (HoFH). The launch marks a significant step in addressing the lipid management needs of Chinese patients. Liptruzet combines the…
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Alphamab’s KN057 Earns FDA Orphan Drug Designation for Hemophilia B Treatment
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Alphamab Oncology (HKG: 9966), a biopharmaceutical company based in China, has received another orphan drug designation (ODD) from the US FDA for its monoclonal antibody (mAb) KN057, which targets the tissue factor pathway inhibitor (TFPI). This new ODD is for the potential treatment of hemophilia B, following the initial ODD…
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Belief BioMed’s Hemophilia B Gene Therapy BBM-H901 Slated for Priority Review in China
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The Center for Drug Evaluation (CDE) has indicated on its website that the market application for BBM-H901, a gene therapy treatment for hemophilia B developed by Belief BioMed Group (BBM), a Shanghai-based specialist in gene therapy, is on track for priority review. BBM-H901, co-developed with Japan’s Takeda, is an adeno-associated…
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Sinqi Pharmaceutical Gets NMPA Green Light for SQ-22031 Eye Drops to Treat Dry Eye Syndrome and Neurotrophic Keratitis
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Shenyang Sinqi Pharmaceutical Co., Ltd (SHE: 300573), a pharmaceutical company based in China, has received clinical trial approval from the National Medical Products Administration (NMPA) for its therapeutic biologic, SQ-22031 eye drops. This Category 1 therapeutic biologic is intended for the treatment of dry eye syndrome and neurotrophic keratitis, with…
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Belief BioMed and Takeda’s Hemophilia B Gene Therapy BBM-H901 Accepted for NMPA Review
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Belief BioMed Group (BBM), a Shanghai-based gene therapy specialist, and Takeda have announced that the National Medical Products Administration (NMPA) has accepted for review the New Drug Application (NDA) for BBM-H901, a gene therapy for hemophilia B. This development marks a significant milestone for the treatment, which is being designed…
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IASO Biotechnology’s CAR-T Therapy Fucaso Receives FDA IND Approval for Multiple Sclerosis
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China-based IASO Biotechnology has announced that it has received Investigational New Drug (IND) approval from the US Food and Drug Administration (FDA) to explore the potential of its BCMA-targeted chimeric antigen receptor (CAR) T cell therapy, Fucaso (equecabtagene autoleucel), in treating multiple sclerosis (MS). This marks the second IND nod…
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China’s NMPA Considers Breakthrough Designations for Xiangxue and Innostellar’s Therapies
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Xiangxue Pharmaceutical Co., Ltd (SHE: 300147) and Innostellar Biotherapeutics Co., Ltd have both been noted by the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) as potential candidates for breakthrough therapy designations (BTDs). Xiangxue’s TAEST16001, a T-cell receptor TCR affinity-enhanced specific T-cell (TAEST) therapy, and Innostellar’s…
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AccurEdit Therapeutics Receives CDE Approval for Gene Editing Drug ART001 Targeting Amyloidosis
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AccurEdit Therapeutics (Suzhou) Co., Ltd, a leading specialist in gene editing therapies based in China, has received approval from the Center for Drug Evaluation (CDE) for its investigational drug ART001. This drug is designed to target transthyroxine protein amyloidosis, a severe genetic disorder. ART001 marks a significant advancement in China’s…
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Bain Capital-Backed Tenacia Wins NMPA Approval for Ganaxolone Oral Suspension for CDKL5 Deficiency
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Tenacia, a central nervous system (CNS) specialist company incubated by Bain Capital, has announced that it has received marketing approval from the National Medical Products Administration (NMPA) for its in-licensed drug, ganaxolone oral suspension. This medication is indicated for the treatment of epileptic seizures in patients aged 2 years and…
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Mabwell Bioscience Lands Orphan Drug Status for B7-H3 Targeting ADC in Small Cell Lung Cancer
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Mabwell (Shanghai) Bioscience Co., Ltd (SHA: 688062) has been granted Orphan Drug Designation (ODD) by the National Medical Products Administration (NMPA) for its antibody drug conjugate (ADC), 7MW3711. The drug targets B7-H3, a receptor overexpressed in various cancers, and is specifically indicated for the treatment of small cell lung cancer…
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Johnson & Johnson’s Opsumit Faces Generic Competition with Qilu Pharmaceutical’s Entry
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Johnson & Johnson (J&J, NYSE: JNJ)’s pulmonary hypertension treatment, Opsumit (macitentan), is set to encounter intensified competition in China following the approval of a generic version by local pharmaceutical company Qilu Pharmaceutical last month. Macitentan, classified as an endothelin receptor antagonist (ERA), initially received marketing approval in the US in…
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Staidson Pharmaceutical Gets NMPA Green Light for Neurotrophic Keratitis Eye Drop Clinical Trial
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Staidson (Beijing) Pharmaceutical Co., Ltd (SHE: 300204), a biopharmaceutical company based in China, has announced that it has received clinical clearance from the National Medical Products Administration (NMPA) for its investigational STSP-0902 eye drops, which are intended for the treatment of neurotrophic keratitis (NK). Neurotrophic keratitis, also referred to as…
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Novo Nordisk’s Esperoct Receives NMPA Approval, First Long-Acting Factor VIII for Hemophilia A in China
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The National Medical Products Administration (NMPA) has granted approval to Novo Nordisk (NYSE: NVO), a Denmark-based pharmaceutical giant, for its drug Esperoct (turoctocog alfa pegol). This marks Esperoct as the first and only long-acting recombinant coagulation factor VIII approved in China for on-demand treatment and control of bleeding events, as…
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CStone Pharmaceuticals Partners with Hengrui for Promotion of Ayvakit in China
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CStone Pharmaceuticals (HKG: 2616), a biopharmaceutical company based in China, has announced a licensing agreement with Jiangsu Hengrui Pharmaceuticals Co., Ltd. (SHA: 600276), another Chinese pharmaceutical firm. The agreement grants Hengrui exclusive promotion rights to Ayvakit (avapritinib), CStone’s precision therapy, in mainland China. CStone maintains all rights to Ayvakit in…
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Staidson Pharmaceutical’s Bemiltenase Alfa Earns Orphan Drug Designation from US FDA for Hemophilia Treatment
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Staidson (Beijing) Pharmaceutical Co., Ltd (SHE: 300204), a biopharmaceutical company based in China, has announced that it has received orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) for its Category 1 biologic bemiltenase alfa (STSP-0601). This designation is for the treatment of hemophilia A or B.…
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Johnson & Johnson Achieves Positive Phase III Results for Nipocalimab in Generalized Myasthenia Gravis
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Johnson & Johnson (J&J, NYSE: JNJ), a leading US-based healthcare company, has announced positive results from the Phase III trial for nipocalimab, a potential first-in-class anti-FcRn (neonatal Fc receptor) antibody, for the treatment of generalized myasthenia gravis (gMG). The company has achieved its primary endpoint and is preparing for regulatory…
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Harbour BioMed Resubmits Biological License Application for Batoclimab in China
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Harbour BioMed (HKG: 2142), a biopharmaceutical company with operations in the United States, the Netherlands, and Suzhou, China, has announced the resubmission of a biological license application (BLA) to China’s National Medical Products Administration (NMPA) for its drug candidate batoclimab (HBM9161) for the treatment of generalized myasthenia gravis (gMG). Batoclimab…
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Qilu Pharmaceutical’s Generic Version of Vyndaqel Receives NMPA Marketing Approval
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The National Medical Products Administration (NMPA) has granted marketing approval to Qilu Pharmaceutical’s generic version of Pfizer’s Vyndaqel (tafamidis meglumine), marking it as the first generic to pass the generic quality consistency evaluation (GQCE) in China. This development is highlighted on the NMPA’s official website. Tafamidis meglumine is indicated for…
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Takeda’s Soticlestat Misses Primary Endpoints in Phase III Epilepsy Trials
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Takeda Pharmaceutical Co., Ltd (TYO: 4502; NYSE: TAK), a leading Japanese pharmaceutical company, has announced that its drug candidate soticlestat did not meet the primary endpoints in two Phase III trials. The drug is currently under development for the treatment of two rare forms of epilepsy—Dravet syndrome and Lennox-Gastaut syndrome—and…
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AbbVie-Calico Partnership Advances with FDA Approval for Fosigotifator Trial in Rare Brain Disorder
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The US Food and Drug Administration (FDA) has welcomed an experimental therapy, fosigotifator, developed by AbbVie Inc. (NYSE: ABBV) and Calico Life Sciences, into its Rare Disease Therapy Pilot Program. The investigational eIF2B activator is currently undergoing a Phase Ib/II clinical trial for the treatment of vanishing white matter (VWM)…
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Wanbangde’s Huperzine A Earns Orphan Drug Status from FDA for Treating Neonatal Brain Injury
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Wanbangde New Building Materials Co., Ltd (SHE: 002082), a company operating in China, has announced that its drug candidate, huperzine A, has been granted an orphan drug designation (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of neonatal hypoxic-ischemic encephalopathy (HIE). This designation is a significant…
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Amgen’s Bkemv Becomes First Biosimilar to Challenge AstraZeneca’s Soliris with FDA Approval
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Amgen Inc. (NASDAQ: AMGN), a leading biopharmaceutical company, has been granted regulatory approval by the US Food and Drug Administration (FDA) for Bkemv, marking a significant milestone as the first biosimilar in the US to compete with AstraZeneca’s (AZ; NASDAQ: AZN) Soliris (eculizumab). Both the originator drug from AstraZeneca and…
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RareStone Group’s Pitolisant Secures NMPA Approval to Treat Narcolepsy in Children and Adolescents
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RareStone Group, a China-based company specializing in rare diseases (formerly known as Citrine Medicine), has secured marketing approval from the National Medical Products Administration (NMPA) for pitolisant, its in-licensed drug candidate. The histamine 3 (H3) receptor antagonist/inverse agonist is now approved for treating excessive daytime sleepiness (EDS) or cataplexy in…
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Novartis’ Fabhalta Shows Promise in Late-Stage C3 Glomerulopathy Trial
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Novartis AG (NYSE: NVS), a leading Swiss pharmaceutical company, last week announced positive preliminary data from a late-stage study for its complement inhibitor Fabhalta (iptacopan) in patients with the ultra-rare kidney disease C3 glomerulopathy (C3G). The study showed that compared to placebo, iptacopan achieved a 35.1% reduction in proteinuria and…
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Novartis’ Atrasentan Shows Significant Reduction in Proteinuria in IgA Nephropathy Trial
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Novartis AG (NYSE: NVS), a Swiss pharmaceutical company, last week presented interim results for the primary endpoint of a Phase III trial for atrasentan in patients with IgA nephropathy (IgAN). The trial indicated that the endothelin A (ETA) receptor antagonist demonstrated a 36.1% reduction in proteinuria at 36 weeks compared…
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Gilead’s Seladelpar Shows Interim Benefits in PBC-Related Itch Clinical Trial
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Gilead Sciences (NASDAQ: GILD) has announced interim results from a long-term study assessing the safety and efficacy of seladelpar, a PPAR-delta agonist, in treating pruritus associated with primary biliary cholangitis (PBC). The trial involved patients who had an inadequate response or were intolerant to ursodeoxycholic acid (UDCA), with 70% of…
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Novo Nordisk’s Mim8 for Haemophilia A Exceeds Endpoints in Late-Stage Trial
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Denmark-based healthcare company Novo Nordisk (CPH: NOVO-B) has announced positive results from a late-stage trial of its bispecific antibody (BsAb) Mim8, used for the treatment of haemophilia A. The trial successfully met its primary endpoints, which focused on the reduction of treated bleeding episodes. The study demonstrated that once-weekly and…
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Wanbangde Pharmaceutical’s Huperzine A Earns FDA Rare Pediatric Disease Designation for HIE Treatment
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China’s Wanbangde New Building Materials Co., Ltd (SHE: 002082) has announced that its subsidiary, Wanbangde Pharmaceutical Group, has received a rare pediatric disease (RPD) designation from the US Food and Drug Administration (FDA) for its drug huperzine A. This designation is for the treatment of neonatal hypoxic-ischemic encephalopathy (HIE), a…
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Mabwell Bioscience’s 9MW2821 Earns Orphan Drug Status from FDA for Esophageal Cancer
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Mabwell (Shanghai) Bioscience Co., Ltd (SHA: 688062), a biopharmaceutical company based in China, has announced that it has received orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for its pipeline candidate, the antibody-drug conjugate (ADC) 9MW2821. This designation is for the drug’s potential in treating esophageal…
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HuidaGene’s MECP2 Duplication Syndrome Therapy Earns Positive EMA Opinion
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HuidaGene (Shanghai) Biotechnology Co., Ltd, a biotech company based in China, has announced that it has received positive opinions from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) regarding its orphan drug filing for HG204. HG204 is an RNA editing therapy developed independently by HuidaGene,…
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Pfizer’s Gene Therapy Beqvez Wins FDA Nod for Moderate-to-Severe Hemophilia B
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The US Food and Drug Administration (FDA) has granted marketing authorization to Pfizer (NYSE: PFE) for its gene therapy Beqvez (fidanacogene elaparvovec) for the treatment of adults with moderate-to-severe hemophilia B. The one-time treatment is indicated for patients using factor IX prophylaxis therapy, those experiencing life-threatening hemorrhages, or those with…
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Novartis’s Iptacopan (Fabhalta) Receives Marketing Approval in China for PNH Treatment
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Swiss pharmaceutical giant Novartis (NYSE: NVS) has received marketing approval from China for its iptacopan (trade name: Fabhalta) for the treatment of adult paroxysmal nocturnal hemoglobinuria (PNH) in patients previously untreated with complement inhibitor. PNH is a rare and life-threatening blood disorder characterized by anemia, hemoglobinuria, bone marrow failure, and…
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Novartis Receives Positive Interim Results for Antimalarial Coartem in Infants
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Swiss pharmaceutical company Novartis (NYSE: NVS) has announced interim results from a Phase II/III clinical trial for its antimalarial drug Coartem (artemether + lumefantrine) in infants with uncomplicated Plasmodium falciparum malaria. The trial data indicates that the tablet formulation meets pharmacokinetic (PK), efficacy, and safety requirements, achieving the primary PK…
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Abbisko Therapeutics’ Irpagratinib Earns Orphan Drug Designation from US FDA for Hepatocellular Carcinoma
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Shanghai-based biotechnology company Abbisko Therapeutics Co., Ltd (HKG: 2256) has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for its novel small-molecule FGFR4 inhibitor, irpagratinib. The designation is in recognition of the drug’s potential in treating hepatocellular carcinoma (HCC). Irpagratinib, an FGFR4 inhibitor originally discovered…
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Sanofi’s Rilzabrutinib Meets Primary Endpoint in Phase III ITP Trial
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French pharmaceutical company Sanofi (NASDAQ: SNY) has announced that its Phase III trial for rilzabrutinib, a potential best-in-class BTK inhibitor, has successfully met its primary endpoint of durable platelet response in patients with immune thrombocytopenia (ITP). The trial also produced positive results in key secondary endpoints and did not raise…
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Ipsen’s Somatuline Receives First Prescription in China for GEP-NETs Treatment
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French biopharmaceutical company Ipsen (EPA: IPN; OTCMKTS: IPSEY) has announced that the first prescription of its Somatuline (lanreotide) sustained-release injection has been issued in China for the treatment of gastrointestinal pancreatic neuroendocrine tumors (GEP-NETs). Somatuline, the only somatostatin analogue (SSA) for deep subcutaneous self-injection, received approval in China in December…
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KPC Pharmaceuticals Gets NMPA Green Light for Clinical Trial on Familial Mediterranean Fever Treatment
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Yunnan-based KPC Pharmaceuticals Inc., (SHA: 600422) has announced that it has received clinical trial approval from the National Medical Products Administration (NMPA) for its Category 2.2 product KPC-149, which is intended for the treatment of familial Mediterranean fever (FMF). FMF is an autosomal recessive autoimmune disease associated with mutations in…
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Shanghai’s 9th People’s Hospital Launches Groundbreaking Gene Therapy Trial for Congenital Deafness
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In a medical first, Shanghai’s 9th People’s Hospital has initiated the world’s inaugural clinical trial for a gene-editing therapy targeting congenital deafness attributed to otoferlin (OTOF) gene mutations. The therapy, developed by HuidaGene Therapeutics Co., Ltd, represents a significant advance in the treatment of auditory neuropathy, a leading cause of…
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Ipsen Secures NMPA Approval for Somatuline in Advanced Neuroendocrine Tumors
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Ipsen (EPA: IPN; OTCMKTS: IPSEY), a France-based biopharmaceutical company, has received new indication approval from the National Medical Products Administration (NMPA) for its Somatuline (lanreotide) sustained-release injection. This approval allows for the treatment of unresectable, highly differentiated or moderately differentiated, locally advanced, or metastatic gastrointestinal pancreatic neuroendocrine tumors (GEP-NETs), aimed…
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Alexion’s Voydeya Earns FDA Nod as Add-on Therapy for PNH-Associated Extravascular Hemolysis
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Alexion, the rare disease unit of AstraZeneca (AZ; NASDAQ: AZN) based in the UK, has secured regulatory approval from the US Food and Drug Administration (FDA) for its Factor D inhibitor Voydeya (danicopan). The drug is approved as an add-on therapy to ravulizumab or eculizumab, which are marketed as Ultomiris…
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J&J’s Nipocalimab Fast-Tracked by FDA for Reducing FNAIT Risk in Pregnant Adults
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The US Food and Drug Administration (FDA) has accepted Johnson & Johnson’s (J&J; NYSE: JNJ) FcRn blocker nipocalimab into its fast-track program for development as a therapy to reduce the risk of fetal neonatal alloimmune thrombocytopenia (FNAIT) in alloimmunized pregnant adults. This move comes after the drug was classified as…
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Takeda Gains MHLW Approval for Adzynma, Targeting Rare Blood Disorder cTTP
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Takeda Pharmaceutical Company (TYO: 4502) has secured regulatory approval from Japan’s Ministry of Health, Labour and Welfare (MHLW) for its enzyme replacement therapy, Adzynma (apadamtase alfa), to treat congenital thrombotic thrombocytopenic purpura (cTTP) in patients aged 12 and older. cTTP is a rare and potentially life-threatening blood clotting disorder resulting…
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Alexion’s Ultomiris Secures FDA Approval for Anti-AQP4 Positive NMOSD Treatment
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AstraZeneca’s (AZ; NASDAQ: AZN) rare disease unit, Alexion, has registered its biologic Ultomiris (ravulizumab) with the US Food and Drug Administration (FDA) for the treatment of anti-AQP4 antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD) in adults. This registration follows an initial rejection by the FDA last year and positions Ultomiris…
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Jiangsu Hengrui’s Generic Version of Tepadina Approved for Transplantation Conditioning in China
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Jiangsu Hengrui Medicine Co., Ltd (SHA: 600276), a leading pharmaceutical company based in China, has announced that it has received market approval from the National Medical Products Administration (NMPA) for its generic version of Tepadina (thiotepa), originally developed by Switzerland-based Adienne S.r.l. The infusion is indicated as a conditioning treatment…
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EMA Panel Backs Novartis’s Fabhalta for PNH Treatment, Awaits EC Decision
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The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the approval of Novartis’s (NYSE: NVS) Factor B inhibitor Fabhalta (iptacopan) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in adults with hemolytic anemia. The European Commission (EC) is anticipated to make a…
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iRegene’s NouvSight001 Gains Orphan Drug Designation for Retinitis Pigmentosa Treatment
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Wuhan-based cell therapy innovator iRegene has secured Orphan Drug Designation (ODD) from the U.S. FDA for its ophthalmic product NouvSight001, aimed at treating retinitis pigmentosa (RP). NouvSight001 is heralded as the world’s first universal ophthalmic cell therapy derived from induced pluripotent stem cells (iPSCs), developed through an innovative “AI+chemical induction”…
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Shanghai BDgene Partners with SPH RD to Advance Gene Therapy Development
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Shanghai BDgene Technology Co., Ltd., a China-based specialist in gene therapy, has entered into a strategic partnership with SPH RD, a wholly owned subsidiary of Shanghai Pharmaceuticals Holding Co., Ltd (SHA: 601607, HKG: 2607). This collaboration aims to leverage BDgene’s advanced carrier technology and gene therapy R&D achievements alongside SPH…
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Boehringer Ingelheim’s Spevigo Receives NMPA Approval for GPP Treatment in China
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German pharmaceutical giant Boehringer Ingelheim (BI) has announced that it has received marketing approval from China’s National Medical Products Administration (NMPA) for its biologic drug Spevigo (spesolimab) subcutaneous injection. The drug is indicated for reducing the incidence of generalized pustular psoriasis (GPP) in adolescents aged 12 and above weighing at…
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Novartis Gene Therapy Zolgensma Shows Continued Benefits in Broader SMA Patient Group
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Swiss pharmaceutical giant Novartis (NYSE: NVS) has presented Phase III data this week, demonstrating that the clinical benefits of its gene therapy Zolgensma (onasemnogene abeparvovec) for spinal muscular atrophy (SMA) extend beyond patients up to 2 years of age. The new study has shown similar benefits in older children, including…
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BMS Reports Positive Long-Term Data for Zeposia in Multiple Sclerosis Trials
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Bristol Myers Squibb (BMS; NYSE: BMY) has unveiled results from several Phase III trials for its S1P receptor modulator Zeposia (ozanimod) in the treatment of relapsing forms of multiple sclerosis (MS). In a long-term study, the oral medication demonstrated a low annualized relapse rate, with disability progression observed in only…
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Merck’s Mavenclad Demonstrates Lasting Efficacy in RMS Patients, New Data Show
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Merck KGaA (ETR: MRK), a German multinational pharmaceutical, chemical, and life sciences company, has this week unveiled new clinical data for its cytotoxic drug Mavenclad (cladribine) in the treatment of highly active relapsing multiple sclerosis (RMS). The results indicate that over 95% of participants noted clinically meaningful improvements in cognitive…
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Hainan Poly Pharm Secures Tentative FDA Approval for Generic Version of UCB’s Briviact
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Hainan Poly Pharm Co., Ltd (SHE: 300630), a Chinese pharmaceutical company, has announced that it has received tentative marketing approval from the US Food and Drug Administration (FDA) for its generic version of Belgium-based UCB’s Briviact (brivaracetam). Briviact is recognized as a selective high-affinity synaptic vesicle protein 2A (SV2A) ligand,…
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CANbridge Pharmaceuticals Partners with Peking Union Medical College to Advance Rare Disease Research
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CANbridge Pharmaceuticals Inc. (HKG: 1228), a specialist in rare diseases based in China, has announced a partnership with Peking Union Medical College Hospital to collaborate on an innovative study spanning drug discovery to clinical translation for rare diseases. The partnership aims to establish a rare disease innovation development platform focused…
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Jiangsu Nhwa Secures Exclusive Rights to Teva’s Austedo in China with $30 Million Deal
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Jiangsu Nhwa Pharmaceutical Co., Ltd. (SHE: 002262), based in China, has entered into a cooperation agreement with Teva Pharmaceutical Industries Ltd. (NASDAQ: TEVA), securing exclusive commercialization rights for Austedo (deutetrabenazine) in mainland China. Austedo, a vesicle monoamine transporter 2 (VMAT2) inhibitor, received its initial approval in the U.S. in 2017…
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Immune-Onc Therapeutics’ IO-202 Earns Orphan Drug Designation for CMML from US FDA
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Immune-Onc Therapeutics, the Hangzhou-based subsidiary of US company Immune-Onc Therapeutics Inc., has announced that it has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for its drug candidate IO-202, which is being developed to treat chronic myelomonocytic leukemia (CMML). This potential first-in-class LILRB4 antagonist was…
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MSD’s Belzutifan and Sperogenix’s Vamorolone Targeted for Priority Review by China’s CDE
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The Center for Drug Evaluation (CDE) website has indicated that Merck, Sharp & Dohme’s (MSD; NYSE: MRK) belzutifan and Sperogenix Therapeutics Ltd’s vamorolone are on track to receive priority review statuses in China. Belzutifan, which targets von Hippel Lindau (VHL) disease-associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastoma,…
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Biogen Appoints Ding Weibo as Head of Asia-Pacific Region, Succeeding Francis Wan
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Biogen (NASDAQ: BIIB) has announced the appointment of Ding Weibo as the new head of its Asia-Pacific region, reporting directly to Fraser Hall, the head of Biogen’s Intercontinental Market. Ding succeeds Francis Wan in this pivotal role. Ding Weibo brings a wealth of experience from his tenure at several multinational…
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Hainan Poly Pharm Wins FDA Nod for Generic Version of UCB’s Vimpat
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Hainan Poly Pharm Co., Ltd (SHE: 300630), a Chinese pharmaceutical company, has announced that it has received marketing approval from the US Food and Drug Administration for its generic version of UCB’s Vimpat (lacosamide), originally developed by the Belgium-headquartered UCB. The approved generic will be used for the treatment of…
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Roche’s Piasky (Crovalimab) Secures NMPA Approval for Untreated Paroxysmal Nocturnal Hemoglobinuria
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The National Medical Products Administration (NMPA) has granted marketing approval to Swiss pharmaceutical giant Roche (SWX: ROG) for Piasky (crovalimab) under priority review status. This approval is specifically for the treatment of adult and adolescent patients aged 12 and older with paroxysmal nocturnal hemoglobinuria who have not previously received treatment…
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AstraZeneca Reports 6% Revenue Growth in 2023, Driven by Oncology and Diabetes Drug Sales
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AstraZeneca (AZ; NASDAQ: AZN), the UK-based multinational, reported its financial results for 2023 this week, revealing a 6% year-on-year increase in annual global revenues, reaching USD 45.8 billion at constant exchange rates. Oncology revenue surged by 21%, while cardiovascular, renal, and metabolism (CVRM) sales rose 18%. Respiratory and immunology (R&I)…
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European Commission Approves Takeda’s Hyqvia for Chronic Inflammatory Demyelinating Polyneuropathy
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The European Commission (EC) has granted regulatory approval to Takeda Pharmaceutical Company Limited (TYO: 4502) for its plasma-derived therapy Hyqvia (human immune globulin infusion 10% with recombinant human hyaluronidase) as a maintenance treatment for chronic inflammatory demyelinating polyneuropathy (CIDP). This follows a similar indication extension for the subcutaneous drug in…
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Astellas Forges Partnership with Mass General Brigham to Boost Oncology and Rare Disease Research
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Astellas Pharma Inc. (TYO: 4503), a leading Japanese pharmaceutical company, has announced a strategic partnership with Mass General Brigham, a prominent academic institution in the United States. This collaboration aims to drive scientific and clinical advancements in critical areas, including oncology, rare diseases, and cell and gene therapy. Astellas will…
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Takeda’s Gammagard Liquid Approved by FDA for Chronic Inflammatory Demyelinating Polyneuropathy
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The US Food and Drug Administration (FDA) has granted regulatory approval for Takeda’s (TYO: 4502) plasma-derived therapy Gammagard Liquid, a human immune globulin, for use as induction therapy and treatment for chronic inflammatory demyelinating polyneuropathy (CIDP) in adults. This decision is supported by clinical data demonstrating that the intravenous immunoglobulin…
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Shanghai Fosun Pharmaceutical Receives NMPA Approval for Phase II Study of FCN-338 for Light Chain Amyloidosis
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Shanghai Fosun Pharmaceutical (Group) Co., Ltd (SHA: 600196, HKG: 2196), a leading pharmaceutical company based in China, has announced that it has received approval from the National Medical Products Administration (NMPA) to proceed with a Phase II clinical study for its pipeline drug candidate, FCN-338. This small-molecule Bcl-2 inhibitor will…
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IASO Biotechnology Secures NMPA Approval for Fucaso in Refractory Myasthenia Gravis
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China-based IASO Biotechnology has received clinical trial approval from the National Medical Products Administration (NMPA) for its BCMA-directed CAR T cell therapy, Fucaso (equecabtagene autoleucel), now indicated for the treatment of refractory systemic myasthenia gravis (MG). Originally approved for marketing in China in June 2022 for recurrent and refractory multiple…
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Boan Biotech Secures FDA Orphan Drug Designations for Two Claudin18.2 Targeting Candidates
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Boan Biotech (HKG: 6955), a Chinese biotechnology company, has announced that it has received orphan drug designation (ODD) awards from the US Food and Drug Administration (FDA) for two of its pipeline candidates. The ODDs were granted to the antibody BA1105 and the antibody-drug conjugate (ADC) BA1301, both of which…
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Lingyi Biotechnology’s Gene Therapy Candidate LY-M001 Gets FDA Green Light for Gaucher’s Disease
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Lingyi Biotechnology Co., Ltd, a China-based gene therapy developer, has secured clinical trial approval from the US Food and Drug Administration (FDA) for its proprietary AAV gene therapy candidate, LY-M001, which is being developed to treat type I or III Gaucher’s disease. LY-M001 utilizes a recombinant adeno-associated virus (rAAV) as…
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Sanofi Acquires Rare Disease Candidate INBRX-101 in $1.7 Billion Deal
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French pharmaceutical giant Sanofi (NASDAQ: SNY) has announced the acquisition of INBRX-101, a promising candidate for rare genetic diseases, from U.S. biotechnology firm Inhibrx (NASDAQ: INBX). INBRX-101, currently in Phase II trials for alpha-1 antitrypsin deficiency (AATD), is a recombinant human AAT-Fc fusion protein designed to restore serum AAT activity,…
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Visirna Therapeutics Completes Enrollment in Phase III Study for FCS Drug VSA001
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Visirna Therapeutics, a Shanghai-based biopharmaceutical company, has announced the completion of patient enrollment in a Phase III clinical study for its investigational drug VSA001, which is being studied for the treatment of familial chylomicronemia syndrome (FCS) in China. A total of 36 FCS patients have been enrolled and will be…
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HuidaGene Therapeutics’ CRISPR Therapy Earns Orphan Drug Status for Duchenne Muscular Dystrophy
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China-based biotechnology company HuidaGene Therapeutics has announced that it has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for its CRISPR-Cas12 DNA editing therapy, HG302, intended for the treatment of Duchenne muscular dystrophy (DMD), a severe and often fatal neuromuscular genetic disorder. This designation follows…
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Novartis Lutathera Combo Therapy Shows Significant Progress in GEP-NETs
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Swiss pharmaceutical giant Novartis (NYSE: NVS) has announced preliminary results from a Phase III study that evaluated the efficacy of radioligand therapy (RLT) Lutathera (lutetium (177Lu) oxodotreotide) in combination with the symptom-control drug octreotide as a first-line therapy for SSTR+ well-differentiated grade 2/3 advanced gastroenteropancreatic neuroendocrine tumors (GEP-NETs). The clinical…
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Yifan Pharmaceutical Wins Nod for Generic Diazoxide Oral Suspension in China
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Yifan Pharmaceutical Co., Ltd (SHE: 002019), a Chinese pharmaceutical company, has announced that it has received marketing approval from the National Medical Products for its generic version of German pharmaceutical giant Bayer’s diazoxide oral suspension. This signifies that the drug has successfully passed the generic quality consistency evaluation (GQCE) process…
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Drug Farm’s DF-003 Earns FDA Rare Pediatric Disease Designation for ROSAH Syndrome
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Sino-US biotech company Drug Farm has announced that it has received Rare Pediatric Disease Designation (RPDD) from the US Food and Drug Administration (FDA) for its first-in-class drug candidate DF-003, which is being investigated as a potential treatment for the ROSAH (retinal malnutrition, optic edema, splenomegaly, anhidrosis, and headache) syndrome.…
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Amgen Licenses XeriJect Technology from Xeris Biopharma for Subcutaneous Tepezza Development
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Amgen (NASDAQ: AMGN) has entered into a licensing agreement with Xeris Biopharma (NASDAQ: XERS) for the XeriJect drug delivery technology, aimed at developing, manufacturing, and commercializing a subcutaneous formulation of its orphan drug Tepezza (teprotumumab). The XeriJect suspensions enable concentrations exceeding 400 mg/ml, which helps to reduce injection volumes, lessen…
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Abbisko Therapeutics’ Pimicotinib Earns Orphan Drug Designation from EMA for TGCT Treatment
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Abbisko Therapeutics Co., Ltd (HKG: 2256), a Shanghai-based biotechnology company, has announced that it has received orphan drug designation (ODD) from the European Medicines Agency (EMA) for its proprietary colony stimulating factor-1 receptor (CSF-1R) inhibitor, pimicotinib (ABSK021). The drug is under development for the treatment of inoperable tenosynovial giant cell…
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Shanghai BDgene Technology’s Thalassemia Gene Therapy BD211 Clears NMPA for Clinical Trials
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Shanghai BDgene Technology Co., Ltd, a specialist in gene therapy, has received clinical trial approval from the National Medical Products Administration (NMPA) for its therapy BD211. This gene-modified autologous hematopoietic stem cell therapy is designed for the treatment of thalassemia. In a preliminary study, BD211 demonstrated safety and efficacy in…
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Belief BioMed Group Initiates Clinical Study for Hemophilia A Gene Therapy BBM-H803 in China
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Belief BioMed Group, a Shanghai-based gene therapy specialist, has announced the first subject dosing in a regulatory clinical study for its in-house developed adeno-associated virus (AAV) gene therapy, BBM-H803, for the treatment of hemophilia A in China. This marks a significant step forward in addressing the unmet medical need for…
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BMS’ Augtyro Receives EMA Review for Advanced Lung Cancer and Solid Tumors Treatments
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The European Medicines Agency (EMA) has accepted for review a filing submitted by Bristol Myers Squibb (BMS; NYSE: BMY) for its orphan drug, Augtyro (repotrectinib), which targets ROS1-positive locally advanced or metastatic non-small cell lung cancer (NSCLC) and NTRK-positive locally advanced or metastatic solid tumors. The next-generation tyrosine kinase inhibitor…
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Boan Biotech Secures FDA Orphan Drug Designations for Pancreatic Cancer Treatments
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China-based Boan Biotech (HKG: 6955) has announced that its novel antibody BA1105 and antibody-drug conjugate (ADC) BA1301 have received orphan drug designations (ODDs) from the U.S. Food and Drug Administration (FDA). Both therapies target Claudin18.2 (CLDN18.2) for the treatment of pancreatic cancer. BA1105 is being developed for Claudin18.2 positive advanced…
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Janssen Acquires Rights to Gene Therapy Botaretigene Sparoparvovec from MeiraGTx for XLRP Treatment
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Janssen Pharmaceuticals, a subsidiary of Johnson & Johnson (J&J, NYSE: JNJ), announced last week its acquisition of development, manufacturing, and commercialization rights for the gene therapy botaretigene sparoparvovec from MeiraGTx Holdings (NASDAQ: MGTX) for the treatment of X-linked retinitis pigmentosa (XLRP). Janssen will pay an upfront fee of USD 65…
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AstraZeneca Secures FDA Approval for Self-Administered hATTR-PN Therapy Wainua
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AstraZeneca (AZ, NASDAQ: AZN), headquartered in the UK, announced last week that the U.S. Food and Drug Administration (FDA) has granted regulatory approval for its ligand-conjugated antisense oligonucleotide (LICA) Wainua (eplontersen) as a treatment for hereditary transthyretin-mediated amyloidosis polyneuropathy (hATTR-PN/ATTRv-PN) in adults. Developed in collaboration with Ionis Pharmaceuticals (NASDAQ: IONS),…
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Simcere Affiliate Xianbo Biotech Gets China Nod for CAR-T Therapy SNC109
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Shanghai Xianbo Biotech Co., Ltd, known as Simnova outside of China and an affiliate of Simcere Investment Group, has received tacit clinical trial approval from Chinese authorities for its SNC109, an in-house developed treatment for recurrent glioblastoma. SNC109 is a dual chimeric antigen receptor (CAR)-T cell therapy designed on Xianbo…
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HuidaGene Secures Rare Pediatric Disease Designation for CRISPR Therapy HG302 Targeting DMD
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HuidaGene Therapeutics, based in China, has announced that it has received Rare Pediatric Disease Designation (RPDD) from the U.S. FDA for its CRISPR-Cas12 DNA editing therapy, HG302. This designation highlights the therapy’s potential to treat Duchenne muscular dystrophy (DMD), a severe neuromuscular genetic disorder. HG302 employs high fidelity Cas12 (hfCas12Max)…
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EMA’s CHMP Gives Green Light to Sanofi’s Fexinidazole for Acute Sleeping Sickness Treatment
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Last week, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a positive opinion on Sanofi’s (NASDAQ: SNY) Fexinidazole Winthrop (fexinidazole) as a 10-day treatment for acute sleeping sickness caused by Trypanosoma brucei (T.b.) rhodesiense in both adults and children. The EMA is…
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Sino Biopharmaceutical Submits Market Approval Application for Recombinant Coagulation Factor VIIa
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Sino Biopharmaceutical Ltd (HKG: 1177), a leading biopharmaceutical company based in China, has announced the submission of a market approval application for its in-house developed recombinant human coagulation factor VIIa. The application has been accepted for review by China’s National Medical Products Administration (NMPA). The drug candidate is being developed…
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Lepu Biotechnology’s Pancreatic Cancer Drug Earns Orphan Drug Status from FDA
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Lepu Biotechnology Co., Ltd (HKG: 2157), a Chinese pharmaceutical company, has announced that it has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for its proprietary antibody-drug conjugate (ADC), MRG004A, which is being developed to treat pancreatic cancer. MRG004A is an innovative ADC that targets…
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AstraZeneca Acquires Icosavax to Boost Infectious Disease Pipeline with VLP Vaccine Technology
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UK-based pharmaceutical giant AstraZeneca (AZ; NASDAQ: AZN) has announced the acquisition of US vaccines specialist Icosavax (NASDAQ: ICVX), including its protein virus-like particle (VLP) platform. AstraZeneca will pay approximately USD 800 million upfront for all outstanding shares, plus about USD 300 million in milestone payments. The acquisition is set to…
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Sanofi Abandons MZE001 Licensing Deal Following FTC Intervention Over Pompe Disease Monopoly Concerns
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Sanofi (NASDAQ: SNY) has withdrawn from an agreement announced earlier this year to in-license the glycogen synthase 1 (GYS1) inhibitor MZE001 from Maze Therapeutics, following an administrative complaint by the Federal Trade Commission (FTC), the US antitrust agency. The FTC sought a temporary restraining order and preliminary injunction in federal…
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Skyline Therapeutics’ Gene Therapy SKG0201 for SMA Gets Green Light from China’s NMPA
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Skyline Therapeutics has received clinical trial approval from China’s National Medical Products Administration (NMPA) for its proprietary AAV gene therapy, SKG0201 injection, which is being developed as a treatment for type I spinal muscular atrophy (SMA). SKG0201 is an SMN1 gene replacement therapy that is administered through a single intravenous…
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Novartis Reports Kisqali Success in Reducing Recurrence Risk in Early Breast Cancer
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Novartis (NYSE: NVS) has announced an update on the late-stage trial for its CDK4/6 inhibitor Kisqali (ribociclib) in early breast cancer. Data from 78.3% of patients who completed treatment showed a significant reduction in the risk of recurrence, with 30% and 24.5% decreases for stage II and stage III tumors,…
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Novartis’ Fabhalta Wins FDA Nod as First-in-Class Treatment for Paroxysmal Nocturnal Hemoglobinuria
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Swiss pharmaceutical giant Novartis (NYSE: NVS) has secured regulatory approval from the US Food and Drug Administration (FDA) for its first-in-class orphan drug Fabhalta (iptacopan) as an oral monotherapy for adults with paroxysmal nocturnal hemoglobinuria (PNH). The approval was granted based on Phase III clinical data that demonstrated a significant…
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Stem-Cell Startup Xellsmart Earns FDA Orphan Drug Designation for ALS Therapy
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Xellsmart, a stem-cell startup headquartered in Suzhou, has announced that it has received orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for its induced pluripotent stem cell (iPSC) derived subtype neural precursor cell therapy, targeting amyotrophic lateral sclerosis (ALS). ALS is a devastating motor neuron disease…
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China’s State Council Unveils Comprehensive Plan to Boost Beijing’s Healthcare Sector
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The State Council has unveiled a comprehensive “Work Plan” aimed at enhancing Beijing’s service industry capabilities, with a particular focus on healthcare and medical services. Titled “Work Plan Supporting the Construction of Comprehensive Demonstration Zones to Deepen National Service Industry Openness in Beijing,” the document outlines several key proposals that…
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BMS and Avidity Biosciences Join Forces on $2.2 Billion Cardiology Antibody Oligonucleotide Conjugate Deal
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Bristol Myers Squibb (BMS, NYSE: BMY) has announced a strategic partnership with Avidity Biosciences (NASDAQ: RNA) focused on the discovery, development, and commercialization of cardiology-oriented antibody oligonucleotide conjugates (AOCs). This innovative class of RNA therapeutics merges the specificity of monoclonal antibodies with the precision of oligonucleotide therapies, building on insights…
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Sichuan Kelun Pharmaceutical’s RET Inhibitor A400 Earns FDA Orphan Drug Designation
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Sichuan Kelun Pharmaceutical Co., Ltd (SHE: 002422), a Chinese pharmaceutical company, through its innovative drug development subsidiary Sichuan Kelun-Biotech Biopharmaceutical Co., Ltd (HKG: 6990), has announced that it has received orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for its RET inhibitor A400/EP0031 in the treatment…
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China’s CDE Releases Draft Guidelines for Decentralized Trials in Rare Disease Drug Development
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China’s Center for Drug Evaluation (CDE) has released draft “Technical Guidelines for the Application of Decentralized Clinical Trials in Rare Disease Drug Clinical Research and Development,” open for public feedback for one month. The guidelines have been formulated by a research team that extensively reviewed the guidance and suggestions from…
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MSD to Acquire Caraway Therapeutics for USD 610 Million to Boost Neurodegenerative Disease Portfolio
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Merck, Sharp & Dohme (MSD; NYSE: MRK) has announced an agreement to purchase Caraway Therapeutics, a US-based pre-clinical company, for a potential total of USD 610 million, which includes upfront and milestone payments. This acquisition will enhance MSD’s portfolio with a range of small-molecule candidates targeting neurodegenerative and rare diseases.…
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Medilink Therapeutics’ YL201 Earns Orphan Drug Designation for Esophageal Cancer
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Suzhou-based Medilink Therapeutics has announced that it has obtained orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for its antibody-drug conjugate (ADC) YL201, which is being developed to treat esophageal cancer (EC). YL201, a B7H3-targeted ADC product, is based on Medilink’s Tumor Microenviroment Activable LINker (TMALIN)…
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BMS’s Augtyro Receives FDA Approval for ROS1-Positive NSCLC
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The US Food and Drug Administration (FDA) has granted Bristol Myers Squibb (BMS, NYSE: BMY) registration approval for its next-generation tyrosine kinase inhibitor (TKI) Augtyro (repotrectinib) for the treatment of locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC) in adults. This decision follows a priority review of Phase…
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Vertex Pharmaceuticals and CRISPR Therapeutics Receive Conditional Approval for CRISPR Gene-Editing Therapy
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Vertex Pharmaceuticals (NASDAQ: VRTX) and CRISPR Therapeutics (NASDAQ: CRSP) have received conditional approval from the UK Medicines and Healthcare products Regulatory Agency (MHRA) for their jointly developed therapy, Casgevy (exagamglogene autotemcel), a treatment for sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) and for transfusion-dependent beta thalassemia (TDT) in…
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Novartis Acquires HDAC6 Inhibitor CKD-510 from CKD Pharmaceutical in a $1.31 Billion Deal
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Swiss pharmaceutical major Novartis (NYSE: NVS) announced last week that it has struck a deal to acquire the HDAC6 small-molecule inhibitor (SMI) CKD-510 from South Korea’s Chong Kun Dang (CKD) Pharmaceutical. The transaction includes an upfront payment of USD 80 million, with the potential for up to USD 1.23 billion…
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Huadong Medicine’s Arcalyst (Rilonacept) Filing for CAPSs Accepted by China’s NMPA
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Huadong Medicine Co., Ltd (SHE: 000963), a China-based pharmaceutical company, has announced that the National Medical Products Administration (NMPA) in China has accepted its market filing for Arcalyst (rilonacept) for the treatment of cryo-pyrin-associated periodic syndromes (CAPSs). The drug is specifically filed for approval in treating familial cold autoimmune syndrome…
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Clinical Research on rAAV Gene Drug ZS805 for Fabry Disease Launched by Zhishan Weixin
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Zhishan Weixin Biotechnology Co., Ltd, a China-based biotech company, has announced the launch of the first clinical research project in China focused on a recombinant adeno-associated virus (rAAV) genetic therapy for the treatment of Fabry disease. The study will evaluate the adeno-associated virus vector ZS805 as a potential treatment across…
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ImmuneOnco Biopharmaceuticals’ Timdarpacept (IMM01) Earns Orphan Drug Designation for CMML
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ImmuneOnco Biopharmaceuticals (Shanghai) Co., Ltd (HKG: 1541) has announced that it has received orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for its investigational drug timdarpacept (IMM01). This marks a significant milestone for the company’s first SIRPαFc fusion protein targeting CD47, which was designed in China.…
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Takeda Partners with New Horizon Health Technology to Manage Helicobacter pylori in China
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At the 6th China International Import Expo (CIIE) in Shanghai, Japan-based Takeda has inked a partnership with China-based New Horizon Health Technology Co., Ltd (HKG: 6606), a leading cancer diagnostics developer. The collaboration aims to develop a comprehensive management solution for Helicobacter pylori patients in China, underscoring Takeda’s (TYO: 4502)…
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Novo Nordisk Reports 33% YOY Growth in Q3 2023 with Diabetes and Obesity Care Driving Sales
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Denmark-based pharmaceutical giant Novo Nordisk (NYSE: NVO) has released its Q3 2023 earnings report, showcasing a 33% year-on-year (YOY) growth in constant exchange rates (CER) to DKK 166.4 billion (USD 23.7 billion) over the first nine months. The company identified diabetes care and obesity care as the key growth drivers,…
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Novartis’ Cosentyx Gains FDA Approval for Hidradenitis Suppurativa Treatment
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The US Food and Drug Administration (FDA) has issued regulatory approval for Novartis’ (NYSE: NVS) IL-17A-targeting drug Cosentyx (secukinumab) for the treatment of adult patients with moderate-to-severe hidradenitis suppurativa (HS). This approval positions Cosentyx as a new entrant in the HS biologic therapy market, potentially challenging AbbVie’s (NYSE: ABBV) Humira…
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Roche’s Gene Therapy Elevidys Misses Primary Endpoint in DMD Trial
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Switzerland-headquartered pharmaceutical giant Roche (SWX: ROG) has announced that a Phase III trial for its gene transfer therapy Elevidys (delandistrogene moxeparvovec) did not meet its primary endpoint in Duchenne muscular dystrophy (DMD) ambulatory patients aged 4 to 7 years. The trial aimed to measure the change from baseline in motor…
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Amgen Reports Q3 2023 Financials: 5% Growth Driven by Volume Expansion
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Amgen (NASDAQ: AMGN) has released its Q3 2023 financial results, showcasing a 5% year-on-year (YOY) increase in global sales, reaching USD 6.5 billion. This growth comes despite a 3% decrease in net selling price, primarily fueled by an impressive 11% increase in volume. Notably, this marks the company’s fourth consecutive…
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Novartis Announces Positive Interim Results for Atrasentan in IgA Nephropathy Phase III Study
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Novartis (NYSE: NVS), a global healthcare company, has released interim results from an ongoing Phase III study for its endothelin A receptor antagonist (ERA) atrasentan, indicating a significant milestone in the treatment of IgA nephropathy (IgAN), a rare kidney disease. The trial successfully met its primary endpoint, demonstrating a clinically…
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Novartis Q3 Earnings Reflect Strong Growth Post-Sandoz Spinoff
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Novartis (NYSE: NVS) has released its Q3 2023 financial results, marking the first report since the company sharpened its focus on innovative medicines following the spinoff of Sandoz (SWX: SDZ) earlier this month. The financials reveal a robust performance with net sales reaching USD 34.0 billion for the first nine…
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Accuredit Therapeutics Initiates First Dosing of Gene Therapy ART001 for ATTR
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China-based gene therapy start-up Accuredit Therapeutics (Suzhou) Co., Ltd has announced the first dosing of its investigational drug ART001 in seven subjects with transthyretin amyloidosis (ATTR), as part of an investigator-initiated trial (ITT). The drug candidate has demonstrated good safety and achieved a clinically significant decrease in transthyroxine (TTR) protein…
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HuidaGene Receives FDA Rare Pediatric Disease Designation for MECP2 Syndrome Therapy
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China-based HuidaGene (Shanghai) Biotechnology Co., Ltd has announced that it has received Rare Pediatric Disease Designation (RPDD) from the US FDA for its RNA editing therapy, HG204, targeting MECP2 duplication syndrome (MDS). This designation is a significant step forward in the development of novel treatments for this rare pediatric neurodevelopmental…
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AstraZeneca’s Soliris Gains Approval in China for NMOSD Treatment
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UK-based pharmaceutical giant AstraZeneca Plc (AZ, NASDAQ: AZN) has announced that its rare disease drug Soliris (eculizumab) has received marketing approval in China for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in AQP4 antibody positive (Ab+) adults. This approval marks a significant expansion of Soliris’s application in the Chinese…
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Belief BioMed Partners with Takeda for BBM-H901 Commercialization in Asia
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Shanghai-based gene therapy specialist Belief BioMed Group (BBM) has announced a strategic partnership with Japan’s Takeda Pharmaceutical Company (TYO: 4502), granting Takeda commercialization rights for its investigational product, BBM-H901. This agreement encompasses the territories of mainland China, Hong Kong, and Macau. Financial details of the partnership have not been disclosed.…
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Zhejiang Huahai Pharmaceutical Secures Orphan Drug Designation for HB0034
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China-based Zhejiang Huahai Pharmaceutical Co., Ltd (SHA: 600521) has announced that it has received orphan drug designation (ODD) from the US FDA for its interleukin-36 receptor monoclonal antibody (mAb) HB0034. This designation highlights the drug’s potential as a treatment for generalized pustular psoriasis (GPP). Mechanism of Action and Broader Implications…
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CANbridge’s Maralixibat Receives Priority Review for PFIC Treatment in China
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China-based rare disease specialist CANbridge Pharmaceuticals Inc. (HKG: 1228) has announced that a New Drug Application (NDA) for its maralixibat (CAN108) has been accepted for review by the National Medical Products Administration (NMPA) in China, with the added benefit of priority review status. This marks a significant step forward in…
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Roche’s Ocrevus Demonstrates Long-Term Efficacy in Multiple Sclerosis Treatment
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Swiss pharmaceutical giant Roche (SWX: ROG) has presented compelling long-term efficacy data for its selective immunosuppressant Ocrevus (ocrelizumab) in the treatment of multiple sclerosis (MS). The 10-year results from a Phase III extension study provide significant insights into the drug’s sustained impact on patients with relapsing and primary progressive forms…
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Kintor Pharmaceutical Secures NMPA Approval for Phase II Study of GT1708F in Idiopathic Pulmonary Fibrosis
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China-based Kintor Pharmaceutical Ltd (HKG: 9939) has announced that it has received approval from the National Medical Products Administration (NMPA) to initiate a Phase II clinical study for its Hedgehog/SMO inhibitor, GT1708F, targeting idiopathic pulmonary fibrosis (IPF). Understanding the Role of Hedgehog Signaling in IPF Recent research has highlighted the…
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Gritgen Therapeutics Commences Operations of Commercial GMP Facilities in Suzhou
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Suzhou-based gene therapy specialist, Gritgen Therapeutics Co., Ltd, has announced the validation completion and initiation of operations at its commercial Good Manufacturing Practice (GMP) facilities located in the Suzhou Industrial Park. This development marks a significant step forward for the company in advancing its capabilities in gene therapy production. Facility…
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GenFleet Therapeutics’ CDK9 Inhibitor SLS009 Earns Orphan Drug Status for AML
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Shanghai-based biotechnology company GenFleet Therapeutics has announced that its CDK9 inhibitor, SLS009 (GFH009), has received orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for the treatment of acute myeloid leukemia (AML). This designation is a significant milestone, recognizing the drug’s potential to address a rare and…
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Shanghai BDgene’s BD112 Earns Orphan Drug Designation from US FDA for Huntington’s Disease
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Shanghai BDgene Technology Co., Ltd, a specialist in gene therapy, has announced that its drug candidate BD112, which utilizes virus-like particle (VLP) delivery technology for in vivo gene editing, has received orphan drug designation (ODD) from the US Food and Drug Administration (FDA). This follows a previous ODD granted by…
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Alphamab Oncology Enters Licensing Agreement with Grand Life Sciences for KN057 in Greater China
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China-based Alphamab Oncology (HKG: 9966) has announced a licensing agreement with fellow Chinese firm Grand Life Sciences for its monoclonal antibody, KN057. This innovative therapy targets the tissue factor pathway inhibitor (TFPO) and is aimed at treating hemophilia A and hemophilia B in the Greater China region. Agreement Details and…
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Sanofi’s Nexviazyme Approved in China as Enzyme Replacement Therapy for Pompe Disease
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Sanofi’s (NASDAQ: SNY) next-generation enzyme replacement therapy (ERT), Nexviazyme (avalglucosidase alfa), has received marketing approval from the National Medical Products Administration (NMPA) as a long-term treatment for Pompe disease. This marks Sanofi’s second drug approved for Pompe disease in China, following the approval of Myozyme (alglucosidase alfa) in October 2015.…
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Amicus Therapeutics Secures FDA Approval for Pombiliti and Opfolda Combination Therapy
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Amicus Therapeutics (NASDAQ: FOLD), headquartered in the United States, has announced that it has received marketing approval from the U.S. Food and Drug Administration (FDA) for its combination therapy of Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) 65 mg capsules. This innovative two-component therapy is specifically indicated for adults diagnosed with…
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Novo Nordisk and Evotec Launch LAB eN² to Accelerate Drug Discovery for Rare Diseases
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Global healthcare company Novo Nordisk (NYSE: NVO) and drug discovery alliance Evotec (NASDAQ: EVO) have jointly launched LAB eN², a drug discovery accelerator dedicated to translating academic research into innovative therapeutics for cardiometabolic conditions, rare blood diseases, and rare endocrine disorders. This initiative marks a significant step in bridging the…
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Sanofi’s Altuviiio Receives Marketing Approval from Japan’s MHLW for Hemophilia A Treatment
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French pharmaceutical giant Sanofi (NASDAQ: SNY) has announced that it has received marketing approval from Japan’s Ministry of Health, Labour and Welfare (MHLW) for its first-in-class drug Altuviiio (Fc-VWF-XTEN fusion protein). This new treatment is indicated for controlling the bleeding tendency in patients with hemophilia A, a genetic disorder that…
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Novartis’ Lutathera Meets Primary Endpoint in Phase III Trial for GEP-NETs
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Swiss pharmaceutical giant Novartis (NYSE: NVS) has announced that a Phase III trial for its radioligand therapy (RLT) Lutathera (lutetium (177Lu) oxodotreotide) combined with the symptom-control drug octreotide has successfully met its primary endpoint of progression-free survival (PFS) in patients with SSTR-positive, aggressive, advanced gastroenteropancreatic neuroendocrine tumors (GEP-NETs). This achievement…
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EU Chamber of Commerce in China Releases Position Paper, Addresses Pharma and Healthcare
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The EU Chamber of Commerce in China has released its bi-annual “European Business in China Position Paper 2023/2024,” which compiles recommendations from over 1,700 member companies. The document aims to foster constructive dialogue between Europe and China, enhancing engagement between European firms and Chinese stakeholders for mutual benefit. The paper…
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Organon’s Liptruzet Approved in China for Hypercholesterolemia Treatment
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US-based Organon (NYSE: OGN) has announced that it has received market approval from the National Medical Products Administration (NMPA) for its drug Liptruzet (ezetimibe, atorvastatin), which is indicated for the treatment of hypercholesterolemia and homozygous familial hypercholesterolemia (HoFH) in China. DYSIS China Study and Liptruzet’s ImpactThe DYSIS China study reveals…
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China’s NHC Expands Rare Disease List, Includes 21 Cancer Types
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The National Health Commission (NHC) of China has released an updated national list of rare diseases, doubling the number of conditions to 86, including 21 cancer types. This marks the first update to China’s orphan disease schedule since the initial list of 121 conditions was introduced in 2018. Market Access…
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Caliway Biopharmaceuticals’ CBL-514 Achieves Milestones in Dercum’s Disease Trial
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Taiwan-based Caliway Biopharmaceuticals, a specialist in medical aesthetics and inflammatory medicine, has announced that its trial assessing the efficacy and safety of CBL-514 in subjects with Dercum’s Disease lipomas has met all primary and secondary efficacy endpoints. CBL-514 is a first-in-class small-molecule injection lipolysis drug designed to induce adipocyte apoptosis…
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AstraZeneca’s Fasenra Shows Non-Inferior Efficacy to Nucala in EGPA: Phase III Results
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UK-based pharmaceutical giant AstraZeneca (AZ; NASDAQ: AZN) has unveiled the results of a Phase III study, demonstrating the non-inferiority of its IL-5 antagonist Fasenra (benralizumab) compared to GlaxoSmithKline’s (GSK; NYSE: GSK) Nucala (mepolizumab) in the treatment of relapsing or refractory eosinophilic granulomatosis with polyangiitis (EGPA). The study highlights that Fasenra…
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NMPA Pilots Priority Review for Rare Disease Formula Foods, Aims to Reduce Approval Time
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The National Medical Products Administration (NMPA) has initiated a pilot program to implement “Priority review and approval procedures applied to formula foods for rare diseases and special medical purposes that are urgently needed in clinical practice.” According to the People’s Daily, these procedures are expected to be incorporated into the…
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Grand Pharmaceutical Receives NMPA Approval for Generic Carbaglu for Hyperammonemia Treatment
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China-based Grand Pharmaceutical Group Limited (HKG: 0512) has announced that it has received marketing approval from the National Medical Products Administration (NMPA) for its generic version of Italy’s Recordati’s Carbaglu (carglumic acid). This marks the first approval of such a generic drug in China, offering a treatment for hyperammoniaemia caused…
