Tag: rare disease treatment
FDA Targets Changes to Rare Disease Drug Development, Calls for Public Feedback
The US Food and Drug Administration has launched the Rare Disease Framework Initiative, a project to rethink how medicines and biologics for rare conditions...
FDA Grants Historic First Approval for Gene Therapy Fayuvi to Treat Sanfilippo Syndrome Type A
The U.S. Food and Drug Administration has cleared Fayuvi(rebisufligene etisparvovec-hopf) as the first approved treatment for children with mucopolysaccharidosistype IIIA (MPS IIIA), commonly known as Sanfilipposyndrome type A...
Orsini Partners with Priovant to Support Access to New Dermatomyositis Therapy LISRAYA
Orsini, a specialty pharmacy known for its work in rare disease care, has been named a specialty pharmacy partner for LISRAYA (brepocitinib), a newly...
Amylyx Pharmaceuticals Announces Positive Phase 3 Trial Results for Avexitide, for Post-Bariatric Hypoglycemia
Amylyx Pharmaceuticals has announced positive top-line results from its pivotal Phase 3 LUCIDITY trial evaluating avexitide, a first-in-class glucagon-like peptide-1 (GLP-1) receptor antagonist being...
Zydus Collaborates with ICMR to Address Sickle Cell Disease as Desidustat Enters Phase III
Zydus Lifesciences Limited, in collaboration with the Indian Council of Medical Research (ICMR), has announced the successful completion of Phase II Proof-of-Concept trials and approval...