FDA Updates: Faster Adverse Event Reporting and Rare Disease Drug Moves

The FDA initiated daily publication of adverse event reports to enhance drug safety surveillance, a shift from the previous quarterly updates. Separately, Ionis Pharmaceuticals gained FDA approval for Dawnzera, treating hereditary angioedema, and Stealth BioTherapeutics’ elamipretide for Barth syndrome nears expedited review, marking advances in rare disease therapeutics.