Posted by Michael Wonder on 15 Jun 2023
FDA should reject accelerated approval of gene therapy SRP-9001
15 June 2023 - The US FDA should reject the accelerated approval of the gene therapy SRP-9001 (Sarepta Therapeutics) for the treatment of ambulatory Duchenne muscular dystrophy, Public Citizen’s Health Research Group stated in a letter today to the agency.
The treatment, intended for children aged 4-7 years with Duchenne muscular dystrophy failed to demonstrate significant muscle-function sparing in the only randomised 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.
