The FDA has issued multiple notable regulatory decisions impacting the biotech sector. This includes lifting the clinical hold on Rocket Pharmaceuticals’ gene therapy trial for Danon disease with modifications to dosing and pre-treatment protocols. The agency also rejected PTC Therapeutics’ Friedreich’s ataxia drug vatiquinone, requesting new studies for demonstration of efficacy. Celldex Therapeutics discontinued its eosinophilic esophagitis antibody program following clinical data review. These developments underscore ongoing regulatory challenges and shifts for rare disease therapies and emerging biotech products.