Infrequent
FDA Advisors Back Zevra’s Rare Disease Medication Based on Clinical Intuition
Zevra Therapeutics, FDA advisory committee, rare disease medication, clinical intuition, potential approval
Lexeo Therapeutics Reports Promising Early Data for Gene Therapy LX2006 in Friedreich Ataxia Cardiomyopathy
Lexeo Therapeutics, LX2006, Gene Therapy, Friedreich Ataxia, Cardiomyopathy, Clinical Trials, Rare Genetic Disorders
Beacon Therapeutics Raises $170 Million to Advance Ocular Gene Therapy
Beacon Therapeutics, ocular gene therapy, J&J, Series B financing, AGTC-501, rare ophthalmology
iOnctura Secures €80 Million in Series B Financing to Advance Rare Eye Cancer Treatment
iOnctura, Series B financing, rare eye cancer, uveal melanoma, roginolisib, cancer treatment
Amgen’s Uplizna Achieves Phase 3 Success in Treating Immunoglobulin G4-Related Disease, Paving Way for Label Expansion
Amgen, Uplizna, Phase 3, Immunoglobulin G4-Related Disease, IgG4-RD, Rare Autoimmune Disease, Label Expansion
FDA Approves Amgen’s Bkemv, First Interchangeable Biosimilar to Soliris for Rare Blood Disorders ###
Bkemv, Soliris, FDA approval, Interchangeable biosimilar, Rare blood disorders, Paroxysmal nocturnal hemoglobinuria (PNH), Atypical hemolytic uremic syndrome (aHUS), Amgen, AstraZeneca, Alexion Pharmaceuticals
Amgen Secures FDA Approval for Bkemv, the First Interchangeable Biosimilar to Soliris for Rare Blood Disorders
FDA approval, Amgen, Bkemv, Soliris, biosimilar, rare blood disorders, paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS)
Novartis Achieves Success in Two Phase III Trials for Rare Kidney Diseases, Eyes Regulatory Submissions
Novartis, Phase III trials, Rare kidney diseases, IgA nephropathy, C3 glomerulopathy, Regulatory submissions
Translarna by PTC Therapeutics: Awaiting Another EU Regulatory Review for Muscular Dystrophy Treatment
PTC Therapeutics, Translarna, muscular dystrophy, EU review, regulatory approval, Duchenne muscular dystrophy (DMD), European Medicines Agency (EMA), ataluren, rare disease treatment.
FDA Issues Complete Response Letter to Abeona’s Cell Therapy for Rare Skin Disease
FDA, Complete Response Letter (CRL), Abeona, cell therapy, rare skin disease, Epidermolysis Bullosa (EB)