Sarepta makes Duchenne pipeline cuts as sales of Elevidys rise

With the launch of gene therapy Elevidys proceeding to plan, Sarepta Therapeutics is prioritizing its portfolio, including halting development of its next-gen exon skipping treatment for Duchenne.

According to Sarepta's third quarter financial results, Elevidys' net product revenue for the quarter totaled $181 million, plus another $9.5 million in royalty revenue from ex-U.S. sales, handled by Roche. 

The FDA granted accelerated approval for Elevidys, a one-time AAV gene therapy, in June 2023, for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy who have a confirmed mutation in the DMD gene. 

A year later, the treatment won a label expansion to include all Duchenne patients who are at least 4 years of age, regardless of ambulatory status, with a confirmed mutation in the DMD gene. The accelerated approval for ambulatory patients was converted to traditional approval.

Now, given "the evolving therapeutic landscape for Duchenne" as well as a risk-benefit analysis and FDA feedback, Sarepta says it will discontinue the development of SRP-5051 (vesleteplirsen).

SRP-5051 is a peptide phosphorodiamidate morpholino oligomer (PPMO) treatment for patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping. The drug was designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. The phase 2 drug was being positioned as a follow-on drug to Sarepta's  approved therapy, Exondys 51.