Sarepta Therapeutics has made significant strides in its clinical trials for limb-girdle muscular dystrophy (LGMD), particularly for types 2C/R5, 2D/R3, and 2E/R4.

The U.S. FDA has approved the start of screening and dosing for Study SRP-9005-101, a first-in-human trial aimed at treating LGMD type 2C/R5, also known as gamma-sarcoglycanopathy.

Following a pre-BLA meeting with the FDA that confirmed eligibility for accelerated approval, Sarepta plans to file a Biologics License Application for SRP-9003 in the latter half of 2025.

The phase 3 trial for SRP-9003, which targets LGMD type 2E/R4, has successfully completed enrollment and dosing, with data anticipated by mid-2025.

Enrollment and dosing for Study SRP-9004-102, a phase 1 trial focusing on SRP-9004 for LGMD type 2D/R3, have also been completed, emphasizing the safety and expression of the alpha-sarcoglycan protein.

Both investigational gene therapies, SRP-9003 and SRP-9004, utilize the AAVrh74 vector to effectively deliver treatment to skeletal and cardiac muscles, addressing critical symptoms of their respective LGMD subtypes.

Sarepta's pipeline includes gene therapies targeting over 70% of known LGMD cases, and the company is actively enrolling participants in a natural history study named JOURNEY.

LGMDs comprise a group of genetic diseases that lead to muscle weakness, with more than 30 subtypes and currently no approved disease-modifying treatments available.