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Sanofi’s Qfitlia Approved by FDA for Hemophilia A and B Prophylaxis
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France-based Sanofi (EPA: SAN, NASDAQ: SNY) has announced receiving marketing approval from the US Food and Drug Administration (FDA) for Qfitlia (fitusiran). This antithrombin-lowering (AT) therapy is now approved for routine prophylaxis to prevent or reduce bleeding episodes in adult and pediatric patients (aged 12 or older) with hemophilia A…
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Luye Pharma’s BA1302 Receives FDA Orphan Drug Designation for Lung and Pancreatic Cancer
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China-based Luye Pharma Group’s (HKG: 2186) controlling subsidiary Shandong Boan Biotechnology Co., Ltd (HKG: 6955) has announced receiving orphan drug designation (ODD) from the US FDA for its novel antibody drug conjugate (ADC) BA1302. The designation covers two indications: squamous non-small cell lung cancer and pancreatic cancer. Innovative ADC DevelopmentBA1302,…
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Sanofi’s Tolebrutinib Accepted for FDA Review in Non-Relapsing Secondary Progressive Multiple Sclerosis
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Sanofi (EPA: SAN, NASDAQ: SNY) announced that a market filing for its tolebrutinib as a treatment for non-relapsing secondary progressive multiple sclerosis (nrSPMS) has been accepted for review by the US FDA. The agency is expected to make its decision by September 28, 2025. A regulatory submission is also under…
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SineuGene Therapeutics’ SNUG01 Receives FDA Clearance for ALS Clinical Study
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Beijing-based SineuGene Therapeutics, a developer of gene therapies for brain disorders, announced last week that it has received clearance from the US Food and Drug Administration (FDA) to conduct a clinical study for its lead candidate SNUG01. This best-in-class (BIC) TRIM72-targeted gene therapy will be evaluated for the treatment of…
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Suzhou Xueji Biotech’s XJ-MK-002 Gets FDA Orphan Drug Designation for CAMT
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China-based cell therapy developer Suzhou Xueji Biotechnology Co., Ltd’s (HemaCell) XJ-MK-002, described as the world’s first platelet-related cell therapy, received orphan drug designation (ODD) from the US Food and Drug Administration (FDA) last week. The designation is for the treatment of congenital amegakaryocytic thrombocytopenia (CAMT), a rare hereditary bone marrow…
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Novartis’ Fabhalta Approved by FDA for C3 Glomerulopathy Treatment
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Swiss pharmaceutical giant Novartis’ (NYSE: NVS) iptacopan, marketed as Fabhalta, has received approval from the US Food and Drug Administration (FDA) for the treatment of adults with C3 glomerulopathy (C3G). This marks the first and only approved therapy specifically targeting this rare kidney disease, which is characterized by a poor…
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AccurEdit Therapeutics Receives FDA Orphan Drug Designation for ATTR Treatment
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China-based AccurEdit Therapeutics (Suzhou) Co., Ltd announced receiving Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for its investigational drug ART001, targeting transthyretin amyloidosis (ATTR). This marks a significant milestone in the development of this innovative gene-editing therapy. Drug Profile and MechanismART001 is the first non-viral…
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Johnson & Johnson’s Nipocalimab Receives FDA Fast Track Designation for Sjögren’s Disease
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US giant Johnson & Johnson (J&J; NYSE: JNJ) announced receiving Fast Track designation (FTD) from the US FDA for its nipocalimab as a treatment for adult patients with moderate-to-severe Sjögren’s disease (SjD). This designation follows the monoclonal antibody’s Breakthrough Therapy Designation (BTD) received from the agency late last year. Mechanism…
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Merck & Co.’s Winrevair Approved for Pulmonary Arterial Hypertension in Hainan
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US-based giant Merck & Co., Inc. (NYSE: MRK) announced receiving approval from the Hainan Medical Products Administration for its Winrevair (sotatercept-csrk) to be imported by Shanghai Ruijin Hospital’s Hainan subsidiary. This approval is significant as it provides a new treatment option for patients with pulmonary arterial hypertension (PAH) in China.…
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Visirna Therapeutics Announces Positive Phase III Results for Plozasiran in FCS Treatment
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Shanghai-based Visirna Therapeutics announced positive topline results from the Phase III study of plozasiran in Chinese patients with familial chylomicronemia syndrome (FCS). The trial successfully met its primary efficacy endpoint and all key secondary endpoints, marking a significant advancement in the treatment of this rare lipid disorder. Study Details and…
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Lundbeck’s Amlenetug Receives Orphan Drug Designation in Japan for Multiple System Atrophy
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Denmark-based pharmaceutical company Lundbeck A/S (OTCMKTS: HLUBF) announced receiving Orphan Drug Designation (ODD) from Japan’s Ministry of Health, Labor, and Welfare (MHLW) for its investigational drug amlenetug. This designation positions amlenetug as a promising new treatment option for patients with multiple system atrophy (MSA), a rare and progressive neurodegenerative disorder.…
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Suzhou GenAssist’s GEN6050X Receives FDA Clearance for Duchenne Muscular Dystrophy Treatment
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China-based Suzhou GenAssist Therapeutic Co., Ltd has announced receiving clearance from the US Food and Drug Administration (FDA) to initiate clinical trials for its base editing drug, GEN6050X, as a treatment for Duchenne muscular dystrophy (DMD). This groundbreaking therapy represents a significant advancement in gene editing technologies for rare diseases.…
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CDE Launches SPARK Project to Boost Pediatric Antitumor Drug R&D
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China’s Center for Drug Evaluation (CDE) has released a notification to solicit feedback on its Support Anti-tumor drugs R&D for Kids (SPARK) project within one month. This initiative marks a significant step forward in promoting the development of pediatric antitumor drugs. Project BackgroundThe SPARK project is a pilot effort in…
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Huadong Medicine’s ADC HDM2005 Granted FDA Orphan Drug Designation for MCL
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China-based Huadong Medicine Co., Ltd (SHE: 000963) announced that its antibody-drug conjugate (ADC) HDM2005 has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of mantle cell lymphoma (MCL). Drug Mechanism and Preclinical ResultsPreclinical studies have shown that after HDM2005 enters the…
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InnoCare Pharma’s Orelabrutinib Shows Efficacy in RRMS at ACTRIMS Forum
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Beijing-based InnoCare Pharma (HKG: 9969, SHA: 688428) announced the presentation of Phase II study results for its Bruton’s tyrosine kinase (BTK) inhibitor orelabrutinib in relapsing-remitting multiple sclerosis (RRMS) at the 10th annual Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) Forum. Study HighlightsThe data demonstrated significant efficacy of…
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YolTech Therapeutics’ YOLT-203 Shows Promise in Primary Hyperoxaluria Type 1 Trial
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YolTech Therapeutics, a Shanghai-based startup specializing in lipid nanoparticle delivery and gene editing, announced positive results from an investigator-initiated trial (ITT) of YOLT-203 in patients with primary hyperoxaluria type 1 (PH1). The therapy marks the first in vivo gene-editing treatment to demonstrate positive clinical data for PH1, showing excellent safety…
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CANbridge Pharmaceuticals Doses First Patient in CAN108 Biliary Atresia Study
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China-based rare disease specialist CANbridge Pharmaceuticals Inc. (HKG: 1228) announced that the first patient has been dosed in the Phase II EMBARK study of CAN108 (maralixibat) for biliary atresia (BA). The global, multi-center, randomized, controlled trial aims to enroll 72 patients, including 20 in China, to evaluate the drug’s efficacy…
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CDE Includes Chiral Tech’s Cerdelga Generic in Rare Disease Care Plan
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China’s Center for Drug Evaluation (CDE) announced the inclusion of Beijing Chiral Tech’s generic version of Sanofi Genzyme’s Cerdelga (eliglustat) in the patient-centered rare disease drug development pilot program, known as the Care Plan. The move aims to accelerate access to affordable treatments for rare diseases. Drug ProfileEliglustat, a glucosylceramide…
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Pfizer’s Hympavzi (Marstacimab) Launched in China’s Boao Lecheng Pilot Zone for Hemophilia
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Pfizer Inc. (NYSE: PFE) announced the implementation of its hemophilia therapy Hympavzi (marstacimab) in China’s Boao Lecheng Medical Tourism Pilot Zone. The drug, approved in the U.S. and European Union, is the first innovative therapy to be administered subcutaneously once weekly via a pre-filled injection pen for hemophilia A and…
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Takeda’s Takhzyro Receives EMA Approval for New Administration Form in HAE Patients
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Japan-headquartered Takeda (NYSE: TAK, TYO: 4502) announced that the European Medicines Agency (EMA) has approved a label extension for Takhzyro (lanadelumab) for subcutaneous administration in adolescents aged 12 and above and adult patients with Hereditary Angioedema (HAE) using a 2 mL pre-filled pen. Drug ProfileTakhzyro is now approved in the…
