Press release
Sarepta Therapeutics Provides Update on SRP-5051 for the Treatment of Duchenne Muscular Dystrophy
- Sarepta to host conference call at 4:15 p.m. Eastern time CAMBRIDGE, Mass., June 23, 2022 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the
About this update from Sarepta Therapeutics, Inc.
[{"type":"text","content":"- Sarepta to host conference call at 4:15 p.m. Eastern time CAMBRIDGE, Mass., June 23, 2022 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has placed a clinical hold on SRP-5051 (vesleteplirsen), the 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. 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 we will respond to the Agency with this information and proposed changes to the monitoring plan. “Patient safety is always our top priority. The hypomagnesemia was identified through lab tests conducted as part of the monitoring outlined in the protocol of the MOMENTUM study and is similar to previously observed cases of hypomagnesemia in clinical trials of SRP-5051. The hypomagnesemia was transient and patients’ magnesium levels returned to normal following additional supplementation,” said Louise Rodino-Klapac, Ph.D., executive vice president and chief scientific officer, Sarepta Therapeutics. “Globally, we have enrolled approximately half of the planned patients in Part B of MOMENTUM. The study is ongoing, and we remain on track to complete enrollment by the end of the year. We will work to share information with FDA with the goal of resuming screening and dosing in the U.S. as quickly as possible.” Sarepta will host an investor conference call on Thurs., June 23, 2022 at 4:15 pm Eastern time, to discuss this update. The presentation will be webcast live under the investor relations section of Sarepta's website at https://investorrelations.sarepta.com/events-presentations and archived there following the call for one year. Please connect to Sarepta's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary. The conference call may be accessed by dialing (844) 534-7313 for domestic callers and (574) 990-1451 for internat...