WELLESLEY, Mass. — At an event with journalists Tuesday outside of Boston, U.S. Food and Drug Administration commissioner Robert Califf dismissed criticism that the agency is lowering standards for new drug approvals, even when clinical trials fail.
This summer, the FDA expanded Sarepta Therapeutics’ accelerated approval for the Duchenne muscular dystrophy gene therapy Elevidys, despite the drug failing its Phase 3 trial. Some decried the decision, including former FDA chief scientist Luciana Borio. She said that Center for Biologics Evaluation and Research director Peter Marks’ decision to overrule his staff and give the drug full approval was a “mockery of scientific reasoning and approval standards.”
The agency also recently converted another accelerated approval — a decision based on biomarker outcomes — to a full approval, even though the therapy did not meet its final endpoints. In August, Travere Therapeutics’ drug Filspari for IgAN, an autoimmune disease that affects the kidneys, was granted a full approval despite failing its Phase 3 confirmatory trial.
