SRP-4053 (VYONDYS 53) is the second antisense oligonucleotide to be authorised in the US by the FDA for DMD

On 12 December 2019, Sarepta Therapeutics announced, in a press release, that it had obtained conditional marketing authorisation from the FDA for the antisense oligonucleotide, SRP-4053, now referred to as VYONDYS 53. SRP-4053 targets DMD gene exon 53 skipping, an abnormality that affects approximately
 8% of boys with Duchenne muscular dystrophy (DMD). This is the second antisense oligonucleotide developed by Sarepta Therapeutics to be authorised on the American market in the treatment of Duchenne muscular dystrophy, after EXONDYS 51. It should be noted that EXONDYS 51 has still not obtained European marketing authorisation.

An increase in muscle dystrophin, but a clinical benefit that has yet to be demonstrated
This decision is based on the interim results of two clinical trials for SRP-4053 conducted in the United States and Europe (including one investigating centre in France):

  • the phase I/II, double-blind versus placebo trial for SRP-4053, now completed,
  • and the ESSENCE trial, currently ongoing.

These results show an increase in the production of dystrophin in the muscles of the participants being treated with SRP-4053, which, according to Sarepta Therapeutics, could lead to a functional benefit for patients.
To confirm this marketing authorisation in the long-term, the FDA has asked for efficacy results, in particular via the currently ongoing ESSENCE trial.

 

Access Sarepta Therapeutics press release « Sarepta Therapeutics Announces FDA Approval of VYONDYS 53™ (golodirsen) Injection for the Treatment of Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 53 »