FDA Shockers, Breakthrough Trials & Outbreak Alerts – Biotech & Health News Roundup (Aug 24–25, 2025)
The FDA approved Ionis’ Dawnzera as the first RNA-targeted preventive treatment for hereditary angioedema, after trials showed an 81% drop in attack rates. Regulators rejected PTC Therapeutics’ vatiquinone for Friedreich’s ataxia, citing insufficient efficacy data and requesting another trial. Jazz Pharmaceuticals secured approval for dordaviprone to treat rare pediatric brain tumors.
