disease drug – Talk Bio

BridgeBio Shifts Filing Plans After Phase 3 Success in Rare Disease Drug BBP-418

1 month ago talkbio0Tagged Acoramidis, Approved, BBP-418, Biological Markers, BridgeBio, commercial, disease drug, File (record), Infrequent, Limb-girdle muscular dystrophy type 2A, NDA, New Drug Application, Phase 3 success, Ribitol, United States Food and Drug Administration

BridgeBio; BBP-418; Phase 3 success; limb-girdle muscular dystrophy type 2I/R9; FDA filing; rare disease drug; NDA (New Drug Application); ribitol; full approval; biomarker; acoramidis; commercial strategy

FDA Delays Decision on Biohaven’s Troriluzole for Rare Disease, Plans Advisory Committee Review

7 months ago talkbio0Tagged Advisory Committees, Ataxia, Spinocerebellar, Biohaven, Deferred, disease drug, Extension, Infrequent, Neurodegenerative Disorders, PDUFA, Troriluzole, United States Food and Drug Administration

Biohaven; FDA delay; troriluzole; spinocerebellar ataxia (SCA); rare disease drug; PDUFA extension; advisory committee; neurodegenerative disease

Applied Therapeutics Undergoes Leadership Changes Amid FDA Rejection and Regulatory Challenges

12 months ago talkbio0Tagged Changing, disease drug, Govorestat, Leadership, regulatory, Rejection, United States Food and Drug Administration

Applied Therapeutics, leadership changes, FDA rejection, rare disease drug, govorestat, regulatory challenges