MARYLAND (Reuters) – The U.S. health regulator has accelerated approval of Sarepta Therapeutics’ first-of-its-kind gene therapy for Duchenne muscular dystrophy (DMD). This inherited progressive muscle-wasting disorder almost always affects young boys.
Sarepta said that the Food and Drug Administration had approved the treatment for children aged between 4 and 5 years who can walk. It was initially seeking approval for all DMD patients who can walk.
The agency’s decision comes after several delays and questions over the effectiveness of the therapy, which will be sold under the brand name Elevidys.
Elevidys could change how DMD patients, who rarely survive beyond their thirties, are treated. Currently, approved steroids and some therapies for specific genetic mutations only control symptoms and slow disease progression.
In a mid-stage trial, Sarepta’s gene therapy produced a mini version of the dystrophin protein needed to help keep muscles intact. Still, it did not improve patient clinical outcomes like their ability to walk and stand.
A late-stage study is underway to confirm the treatment’s effectiveness, and initial data from it is expected by December.
Current options for DMD patients are limited. Three of four new “exon-skipping” therapies – intended for a smaller group of patients with specific genetic mutations – are from Sarepta.
They require weekly infusions and work by skipping specific parts of genes, called exons, to allow the body to form shorter forms of the dystrophin protein.
The company said Elevidys should not be used in patients with any deletion in the DMD gene or in exon eight and exon 9.
Experts and analysts have said that a green light for Sarepta’s treatment could open the doors for more effective gene therapies. Pfizer is also developing a gene therapy for DMD.
A late-stage trial of 126 patients is ongoing at Sarepta and will be pivotal in expanding the patient population and gaining formal approval.