The Biologics License Application (BLA) of the Duchenne muscular dystrophy (DMD) drug candidate SRP-9001 has been accepted for filing and has been granted priority review by the US Food and Drug Administration (FDA).

If approved, SRP-9001 would be the first gene therapy for DMD.

“Our BLA submission for an accelerated approval, along with the FDA’s acceptance of that BLA for filing and review, is a tremendously important milestone in our effort to bring a potentially life-changing gene therapy to Duchenne patients as rapidly as possible,” said Doug Ingram, president and chief executive officer of Sarepta Therapeutics, the developers of SRP-9001.


Continue Reading

SRP-9001 is an investigational gene therapy that aims to deliver a shorter but still functional version of the DMD gene, which is mutated in DMD, specifically to the muscles to ensure the targeted production of functional dystrophin protein. Dystrophin is essential for the health of muscle cells as it acts as a shock absorber during muscle contraction.

Read more about gene therapy and other experimental therapies for DMD

SRP-9001 has been investigated in many preclinical studies and tested in more than 80 patients with DMD in different clinical trials, showing positive results up to 4 years after treatment as well as a consistent safety profile.

DMD is caused by a mutation in the DMD gene, which results in no dystrophin protein being produced. This leads to progressive muscle weakness and wasting and results in symptoms such as difficulty walking, climbing stairs, or standing.

As the disease progresses, patients require a wheelchair for mobility and eventually lose the ability to breathe independently needing a ventilator. The disease, which is universally fatal, also affects the cardiac muscles and may cause heart failure.

Reference

Sarepta Therapeutics announces that U.S. FDA has accepted for filing and granted priority review for the biologics license application for SRP-9001, Sarepta’s gene therapy for the treatment of ambulant individuals with Duchenne muscular dystrophy. News release. Sarepta Therapeutics; November 28, 2022.