AskBio’s AB-1003 Receives Rare Pediatric Disease and Orphan Drug Designations from FDA
Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned subsidiary of Bayer AG (ETR: BAYN), has announced...
Rare / orphan disease drugs
Belief BioMed’s BBM-D101 Receives FDA Orphan Drug and Pediatric Rare Disease Designations for DMD
Belief BioMed has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan...
Innorna Co., Ltd Receives FDA Rare Pediatric Disease Designation for mRNA Drug IN015
Shenzhen-based Innorna Co., Ltd has announced that it has received Rare Pediatric Disease Designation (RPDD)...
AstraZeneca’s Fasenra Approved by EU for Eosinophilic Granulomatosis Treatment
UK pharmaceutical giant AstraZeneca Inc. (NASDAQ: AZN) has received marketing approval from the European Commission...
RemeGen’s Telitacicept Receives Priority Review Status from China’s CDE for gMG Treatment
RemeGen (HKG: 9995), a China-based pharmaceutical company, has announced that the Center for Drug Evaluation...
Novo Nordisk’s Alhemo Receives CHMP Recommendation for Haemophilia Prophylaxis in Europe
Novo Nordisk (NYSE: NVO), a Danish pharmaceutical company, has been notified that the Committee for...
Jacobio Pharma’s G12C Inhibitor Gecirasib Earns Orphan Drug Designation in Europe for Pancreatic Cancer
Jacobio Pharma (HKG: 1167), a China-based biopharmaceutical company, has announced that it has received Orphan...
Johnson & Johnson’s Nipocalimab Shows Promise in Treating Adolescent Myasthenia Gravis in Phase II/III Trial
Johnson & Johnson (J&J, NYSE: JNJ), a US pharmaceutical giant, has reported encouraging outcomes from...
Pfizer’s Hympavzi Secures FDA Nod, Offering Novel Once-Weekly Treatment for Hemophilia B
Pfizer Inc. (NYSE: PFE) has scored a significant win with the US Food and Drug...
Biogen’s China Unit Gets Conditional NMPA Nod for Qalsody (Tofersen) in ALS Treatment
Biogen (NASDAQ: BIIB)’s Chinese subsidiary has announced that it has received conditional approval from the...
China’s CDE Announces “Care Plan” to Include Pfizer’s Marstaximab for Rare Disease Drug Development
The National Health Commission (NHC) of China, through its Center for Drug Evaluation (CDE), has...
Beijing Aims to Establish Rare Disease Drug Security Pilot Zone with ‘White List’ System
Beijing is set to follow in the footsteps of Boao Lecheng with the release of...
AstraZeneca’s Fasenra Secures FDA Nod for EGPA Treatment, Strengthening Rare Disease Portfolio
UK pharmaceutical giant AstraZeneca (NASDAQ: AZN, LON: AZN) has announced that the US Food and...
CDE Launches CARE Plan to Streamline Rare Disease Drug Development
The Center for Drug Evaluation has unveiled the ‘Pilot Work Plan for Patient-Centered Action for...
Suzhou GenAssist Launches First-of-Its-Kind Base Editing Drug Trial for Duchenne Muscular Dystrophy
Suzhou GenAssist Therapeutic Co., Ltd, a biopharmaceutical company based in China, has announced the commencement...
Jiangsu Hengrui’s ANGPTL3 Inhibitor SHR-1918 Earns Breakthrough Designation for HoFH Treatment
Jiangsu Hengrui Pharmaceuticals Co., Ltd (SHA: 600276), a leading pharmaceutical company in China, has announced...
Vcanbio’s Stem Cell Therapy VUM02 Gets NMPA Approval for Systemic Sclerosis Clinical Trial
Vcanbio Cell & Gene Engineering Corp., Ltd (SHA: 600645), a biotechnology company based in China,...
AccurEdit Therapeutics’ Gene-Editing Drug ART001 Clears FDA Hurdle for US Clinical Trials
SUZHOU—AccurEdit Therapeutics (Suzhou) Co., Ltd, a leading Chinese specialist in gene editing therapies, has announced...
Tonghua Golden-Horse, UCB Seek China Approvals for Alzheimer’s, Myasthenia Gravis Drugs
China’s Tonghua Golden-Horse Pharmaceutical Industry Co. Ltd. and Belgium-based UCB (FRA: UNC) are seeking marketing...
China’s Supreme Court Deals Blow to Sanofi’s Aubagio, Confirms Patent Cancellation
BEIJING—In a recent development for the pharmaceutical industry, Sanofi’s multiple sclerosis therapy Aubagio (teriflunomide) has...