The US Food and Drug Administration has expanded the permitted medical use of a gene therapy for Duchenne muscular dystrophy (DMD), making it available to patients who are both ambulatory and non-ambulatory. DMD is a genetic disorder characterised by a progressive deterioration of muscle due to abnormalities in, or the absence of, dystrophin protein which helps keep muscle cells intact. The product, Elevidys (delandistrogene moxeparvovec), was given an accelerated approval in June 2023 for individuals four to five years of age who had a confirmed DMD gene mutation and were still able to walk.
On 20 June, the FDA announced a full approval for this indication and lifted the age. It also issued a new accelerated approval for non-ambulatory individuals four years of age and above. A confirmatory study in the non-ambulatory population is underway.
Peter Marks, head of the FDA’s Center for Biologics Evaluation and Research, said that broadening the number of patients eligible for the treatment meets an ongoing, urgent medical need. Elevidys is a recombinant therapy delivered by an adeno-associated virus vector into the bloodstream and muscle cells prompting these cells to produce dystrophin protein. The protein produced by Elevidys is micro-dystrophin, a shortened version of the protein in normal muscle cells. Data supporting the new accelerated approval showed that an increased level of micro-dystrophin was reasonably likely to predict clinical benefit in the non-ambulatory population. Elevidys was developed by Sarepta Therapeutics Inc.
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