Sarepta tests FDA flexibility with bid to expand Duchenne gene therapy’s approval

 Sarepta Therapeutics has asked the Food and Drug Administration to expand approval of its gene therapy for Duchenne muscular dystrophy, banking on the regulator’s flexibility in reviewing conflicting clinical trial data.

The biotechnology company is requesting the FDA clear its treatment, called Elevidys, for people with Duchenne and a confirmed mutation in the relevant gene. Currently, Elevidys is approved only for patients aged 4 through 5 years old who can still walk. The neuromuscular disease, which primarily affects boys, progressively wastes away the muscles of affected individuals, often confining them to a wheelchair at an early age.

Elevidys is meant to compensate for the genetic mutations that cause Duchenne. Delivered via an engineered virus, it delivers instructions for a shortened form of a crucial-muscle protecting protein that treated patients lack.

Sarepta won a conditional clearance from the FDA in June on the basis of evidence showing Elevidys led to production of that protein, micro-dystrophin, which the agency judged was “reasonably likely” to translate to clinical benefits. The biotech had a late-stage study ongoing designed to prove whether it did.

However, in late October, Sarepta said that study actually missed its main goal. Treatment did not show a statistically significant difference from placebo on a functional scale known as the North Star Ambulatory Assessment. Trial participants treated with Elevidys improved by 2.6 points, compared to 1.9 points among those given the placebo.

Sarepta pointed to what it described as consistent treatment benefit on a number of other measures, such as how quickly participants could rise or walk 10 meters. While typically a negative primary result would necessitate further study, Sarepta said the disease’s severity and the collective picture painted by Elevidys’ data made it appropriate to expand the treatment’s approval.

Friday’s application puts the ball in the FDA’s court. The agency has pledged flexibility in considering drugs for disease like Duchenne, and already shown it in the course of reviewing Elevidys the first time. High-ranking FDA officials have also been closely involved in discussions, and the regulator faces pressure from patient groups to move quickly.

https://finance.yahoo.com/m/8840bc0d-7ee4-3d30-9c67-381fd1ac48ec/sarepta-tests-fda-flexibility.html