We are disappointed to learn of the news regarding Sarepta’s SRP-5051 MOMENTUM study. Sarepta has decided to stop developing SRP-5051, a potential treatment for Duchenne muscular dystrophy (DMD). This means the MOMENTUM study, which was testing SRP-5051, an exon-51 skipping treatment, has ended.
The goal with SRP-5051 was to provide a safe and effective treatment option for people with DMD. While early results showed promise in increasing dystrophin levels, the research team encountered side effects, such as hypomagnesemia (low blood magnesium levels) in some participants, an issue considered monitorable and manageable. However, some trial participants have reported hypomagnesemia after treatment discontinuation. Additionally, some patients experienced a decline in kidney function (measured via eGRF tests).
Drug development is a complex process. Sarepta will continue to review the data and will be sharing further updates. If you have been involved in the MOMENTUM, please contact your consultants for further information.
Next steps:
- The discontinuation of the MOMENTUM study does not have an impact on other clinical study programs or approved therapies (Sarepta’s gene therapy, or PMO exon skipping therapy). Concerns are linked to the specific cell penetrating peptide used in SRP-5051.
- Clinical trial participants will be scheduling their final visits, as the MOMENTUM study begins to wind down.
- All MOMENTUM clinical study sites have been informed of this decision, and the study teams are in the process of contacting participants and families.
Action Duchenne remains steadfast in supporting families and all members of the community. Please reach out to our team at info@actionduchenne.org / +44 (0)7535 498 506 if you have any further questions.
Our team has produced a series of bite-sized science videos to help you digest the latest scientific advancements with ease. Click the links below to find our resources:
Section 1 – Facts about Duchenne muscular dystrophy
Section 2 – Signs and Symptoms of Duchenne muscular dystrophy
Section 3 – Diagnosis of Duchenne muscular dystrophy
Section 4 – Crucial Genetic Terminology
Section 5 – Genetics – Blueprint of Duchenne muscular dystrophy
