CBER Analysis: FDA Has Succeeded In Reducing Gene Therapy Holds

A new study from current and former FDA officials, including ousted gene therapy chief Nicole Verdun, says FDA succeeded in reducing clinical holds on gene therapy products through a 2023-2024 pilot program, coming as the agency and rare disease stakeholders continue to grapple with the fallout of holds on Sarepta Therapeutics’ muscular dystrophy treatments that created a major controversy for the Center for Biologics Evaluation and Research (CBER).