FDA Approves Muscular Dystrophy Gene Therapy Despite Failed Trials
WASHINGTON, D.C. —The FDA’s ill-advised approval of an expensive Duchenne Muscular Dystrophy (DMD) gene therapy that failed clinical trials highlights leadership failures at the drug safety agency, according to a new report from Public Citizen’s Health Research Group. The report focuses on the actions of an FDA senior director who twice overruled the agency’s scientific staff to approve the treatment.
DMD is a rare, progressive and deadly disease with limited treatment options that strikes in early childhood and mostly affects boys. The FDA granted the gene replacement therapy, delandistrogene moxeparvovec-rokl (Elevidys), accelerated approval in 2023 and full approval in June 2024. The therapy presently costs payers – patients, taxpayers and insurers – more than $3 million per treatment. Two randomized clinical trials of delandistrogene moxeparvovec-rokl failed to meet their primary endpoints for clinical benefit.
“The role of a single FDA leader in overruling the agency’s scientific staff who were assigned to evaluate this potential gene therapy is deeply troubling,” said Dr. Michael Abrams, senior health researcher at Public Citizen and lead author of the report. “The report offers a case study of a regulatory decision that sets a dangerous precedent for approval of biologic therapies for rare diseases. As the FDA reviews more gene therapies, the agency needs better decision-making processes that include measures to limit unwise actions by individual officials.”
The other authors of the report are Dr. Reshma Ramachandran of the Yale School of Medicine, and Dr. Robert Steinbrook, the director of Public Citizen’s Health Research Group.
“It is especially challenging to find better treatments for Duchenne muscular dystrophy and other rare but devastating conditions,” said Steinbrook. “Unfortunately, there is no convincing evidence that this very expensive gene therapy will help patients regain muscle function. The FDA’s approval of delandistrogene moxeparvovec-rokl underscores the need for both stronger approval standards and prompt reforms in the agency’s internal procedures.”