FDA Faces Rare Disease Drug Review Challenges

BioCentury reports the FDA is conducting landmark reviews of several rare disease drug approvals, testing its commitment to biomarker-based accelerated approval pathways for orphan drugs. Additional reports include the FDA investigating Sarepta Therapeutics’ Duchenne muscular dystrophy therapy amid patient deaths and internal tensions surrounding KalVista Pharmaceuticals’ rare disease drug submissions. These developments highlight regulatory uncertainty influencing rare disease therapy approval landscapes.