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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…
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Innorna’s IN013 Receives FDA Rare Pediatric Designation for Wilson’s Disease Treatment
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Shenzhen-based Innorna Co., Ltd, a platform company leveraging mRNA and lipid nanoparticle (LNP) delivery technologies, announced that its IN013 has received Rare Pediatric Disease Designation (RPDD) from the U.S. Food and Drug Administration (FDA). The mRNA drug is designed to treat hepatolenticular degeneration (HLD), also known as Wilson’s disease, a…
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Gene Cradle and Byongen Therapeutics Partner on Next-Generation DMD Gene Therapies
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Gene Cradle and Byongen Therapeutics, both Beijing-based gene therapy specialists, have formed a strategic alliance to develop next-generation gene therapies for Duchenne muscular dystrophy (DMD). Collaboration FocusGene Cradle, which initiated its DMD gene therapy project in 2021, has focused on optimizing Dystrophin proteins and regulatory elements. The company has designed…
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Biogen Licenses Stoke Therapeutics’ Zorevunersen for Dravet Syndrome Outside Americas
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US-based Biogen Inc. (NASDAQ: BIIB) has entered into a licensing agreement with Stoke Therapeutics, Inc. (NASDAQ: STOK), securing exclusive commercialization rights to zorevunersen, a drug for Dravet syndrome, in all territories outside the United States, Canada, and Mexico. Drug ProfileZorevunersen is an antisense oligonucleotide (ASO) targeting the SCN1A gene, the…
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Merck’s Welireg Wins Conditional EU Approval for VHL Disease and Advanced RCC
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US pharmaceutical giant Merck, Sharp & Dohme (MSD, NYSE: MRK) announced that it has received conditional approval from the European Commission (EC) for its oral drug Welireg (belzutifan) for the treatment of von Hippel-Lindau (VHL) disease and advanced clear cell renal cell carcinoma (RCC). Drug ProfileWelireg is a hypoxia-inducible factor-2…
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Wanbangde New Building Materials’ Mecobalamin Earns FDA Orphan Drug Designation for ALS
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China’s Wanbangde New Building Materials Co., Ltd (SHE: 002082) announced that its mecobalamin product has received an orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) for the treatment of amyotrophic lateral sclerosis (ALS). The designation highlights the drug’s potential to address unmet medical needs in ALS,…
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Bayer’s Acoramidis (Beyonttra) Gets EMA Marketing Approval for ATTR-CM Treatment in EU
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Germany-headquartered Bayer (ETR: BAYN) has received marketing approval from the European Medicines Agency (EMA) for its drug acoramidis, commercially known as Beyonttra. This near-complete (≥90%) transthyretin (TTR) stabilizer can now be used to treat wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM) in the European Union (EU).…
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Roche Transfers InterMune and Esbriet to Legacy Pharma in IP Deal
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Swiss pharma giant Roche (SWX: ROG) has transferred US-based InterMune, Inc., acquired for USD 8 billion in 2014, and the intellectual property (IP) rights to Esbriet (pirfenidone) in the US to Legacy Pharma Inc. SEZC. Financial terms were undisclosed. FinancialsEsbriet, an idiopathic pulmonary fibrosis (IPF) drug, saw peak sales of…
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Sinopep-Allsino Wins NMPA Approval for Generic Liptruzet in China
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Jiangsu-based CDMO Sinopep-Allsino Biopharmaceutical Co., Ltd (SHA: 688076) has received marketing approval from China’s National Medical Products Administration (NMPA) for its generic version of Organon’s (NYSE: OGN) Liptruzet (ezetimibe, atorvastatin), becoming the first such product approved in China. The drug, used to treat hypercholesterolemia and homozygous familial hypercholesterolemia (HoFH), was…
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Regend Therapeutics’ Pulmovinci Receives FDA Orphan Drug Designation for IPF
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Suzhou-based stem cell therapy specialist Regend Therapeutics announced that the US Food and Drug Administration (FDA) has granted its airway basal layer stem cell product, Pulmovinci, Orphan Drug Designation (ODD) for the treatment of idiopathic pulmonary fibrosis (IPF). This designation is a significant milestone in the development of Pulmovinci, highlighting…
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Novo Nordisk’s Mim8 Shows Promise in Hemophilia A Trial with 98% Caregiver Preference
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Denmark-based Novo Nordisk A/S (NYSE: NVO) released interim results from the Phase III FRONTIER3 study, demonstrating the benefit of Mim8 in 70 children (aged 1-11 years old) with hemophilia A, both with and without inhibitors. The trial initially assessed once-weekly prophylaxis treatment with the investigational drug, offering participants the option…
