FDA Faces Scrutiny on Rare Disease and Duchenne Drug Reviews

Recent investigative reports highlight FDA Commissioner involvement in rejecting KalVista Pharmaceuticals' rare disease drug application and ongoing agency review of Sarepta Therapeutics' Duchenne muscular dystrophy therapy following patient deaths. These developments underscore intense regulatory scrutiny and challenges within rare disease drug approval pathways. Continued examination by the FDA reflects heightened diligence amid safety concerns and impacts industry perceptions of regulatory dynamics in orphan drug evaluations.