fb tracking

FDA Should Reject Accelerated Approval of Gene Therapy SRP-9001

Washington, D.C. — The U.S. Food and Drug Administration (FDA) should reject the accelerated approval of the gene therapy SRP-9001 (Sarepta Therapeutics) for the treatment of ambulatory Duchenne muscular dystrophy (DMD), Public Citizen’s Health Research Group stated in a letter today to the agency.

The treatment, intended for children aged 4-7 years with DMD, failed to demonstrate significant muscle-function sparing in the only randomized clinical trial so far. Moreover, the treatment has safety concerns — most notably that the viral vector needed to deliver this gene therapy cannot be used repeatedly, even for another treatment that might later prove to be safe and effective.

“If this potential treatment for DMD is fast-tracked with accelerated approval, other companies may be encouraged to rush unconfirmed gene therapies to the marketplace,” said Michael Abrams, M.P.H., Ph.D., senior health researcher at Public Citizen.

Public Citizen’s letter adds to oral testimony they delivered to the FDA’s external advisory committee about SRP-9001 last month. The new letter adds that those advisory committee proceedings were wrongly “steeped in anecdotes over evidence,” and FDA officials at the meeting did not fully caution the committee about the potential consequences of accelerated approval.

“Once a therapy is approved by the FDA, via accelerated approval or otherwise, it is very difficult to remove it from the market. FDA officials should have been more forthcoming with the advisory committee about this ‘genie out of the bottle’ effect,” Abrams said.

A final FDA decision on the application is expected by June 22, 2023.