FDA slaps clinical hold on Sarepta's DMD candidate SRP-5051 trial

Shares of Sarepta Therapeutics (Nasdaq: SRPT) dropped almost 9% to $65.39 in after-hours trading yesterday, after the company said the US Food and Drug Administration put a clinical hold on SRP-5051 (vesleteplirsen), a treatment for patients with Duchenne muscular dystrophy (DMD).

Vesleteplirsen is the US rare diseases specialist company’s next-generation peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) to treat patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping.

The hold in Part B of Study 5051-201, also known as MOMENTUM, follows a serious adverse event of hypomagnesemia. The FDA is requesting information on all cases of hypomagnesemia, including a small number of non-serious grade 2 cases, and to assess the adequacy of the risk mitigation and safety monitoring plan. In the next few days Sarepta says it will respond to the Agency with this information and proposed changes to the monitoring plan.