Sarepta says its experimental Duchenne drug more effective than older medicine, ET HealthWorld


New Delhi: Sarepta Therapeutics’ experimental drug produced higher levels of a specific protein deficient in some patients with Duchenne muscular dystrophy (DMD) than its older drug Exondys 51 when tested in a mid-stage trial, the company said on Monday.

DMD is a genetic muscle wasting disorder that affects an estimated one-in-3,500 male births worldwide, and the majority of the patients lack the protein dystrophin which keeps muscles intact.

The drug SRP-5051 and Exondys 51 both belong to a class of “exon-skipping” therapies, which work by skipping specific parts of genes, called exons, to allow the body to make shorter forms of the dystrophin protein. Both drugs are given to specific patients who can be treated by skipping exon 51.

Sarepta said the new drug, when given every four weeks, showed an over 12-fold increase in dystrophin expression and a nearly 25-fold increase in exon skipping after 28 weeks compared to weekly dosing of Exondys 51.

Exondys 51, the company’s first DMD treatment that treats the disorder by skipping exon 51, was approved in 2016 and generated revenue of $409.6 million in the first nine months of 2023 and was Sarepta’s biggest product.

The company’s portfolio also consist of two more drugs for DMD, Vyondys 53 and Amondys 45, which are used to treat patients by skipping exon 53 and 45, respectively.

The company has also secured the U.S. Food and Drug Administration‘s accelerated approval in June last year for a gene-therapy to treat four to five-year olds with DMD. It is currently awaiting full approval for the therapy.

  • Published On Jan 30, 2024 at 02:54 PM IST

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