The U.S. drug regulator on Thursday granted accelerated approval to Sarepta Therapeutics’ first-of-its-kind gene therapy for Duchenne muscular dystrophy (DMD), an inherited progressive muscle wasting disorder that almost always affects young boys.
The U.S. Food and Drug Administration’s approval, for children aged between 4 and 5 years who can walk, comes after several delays as well as questions over the therapy’s effectiveness.
The one-time gene replacement therapy, to be sold under brand name Elevidys, could change the way DMD patients are treated. Currently approved steroids and some therapies for specific genetic mutations only control symptoms and slow disease progression.
Sarepta was initially seeking approval for all DMD patients who can walk, not just 4- and 5-year-olds, but the company said in May the FDA was likely to clear the drug initially only for that population.
In a mid-stage trial, Sarepta’s gene therapy was able to produce a mini version of the dystrophin protein needed to help keep muscles intact, but did not improve patient clinical outcomes like their ability to walk and stand.
A late-stage study to confirm the gene therapy’s effectiveness is underway and initial data from it is expected by December.
Current options for DMD patients, who rarely survive beyond their thirties, are limited, with treatment consisting mostly of cortocosteroids and four new “exon-skipping” therapies for a smaller group of patients with specific genetic mutations.