FDA Relents, Allows Gene Therapy Limited Use

FDA is backpedaling its enforcement stance against Sarepta Therapeutics and Duchenne muscular dystrophy (DMD)gene therapy Elevidys after determining the latest death (see story) reported after a patient was administered the product was unrelated. “The U.S. Food and Drug Administration is recommending the removal of the voluntary hold for ambulatory patients who may now receive Elevidys …,” the agency said in a statement. “The FDA’s investigation has concluded that the death of the 8-year-old boy is unrelated to the gene therapy product itself.”

FDA says it will continue to work with the sponsor regarding non-ambulatory patients, which remains subject to a voluntary hold, following two deaths. The agency hinted in its statement that the DMD patient community played an important role in allowing limited use of the product while the investigation continues.