Sarepta Gene Therapy Gets Fast Track Approval

Sarepta Therapeutics has announced FDA accelerated approval of its Elevidys gene therapy for the treatment of children aged 4 through 5 years with Duchenne muscular dystrophy (DMD) and a confirmed mutation in the dystrophin gene. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).

Elevidys is intended to treat the underlying cause of Duchenne, which is characterized by mutations in the dystrophin gene that results in the lack of dystrophin protein. In the absence of dystrophin, which is required to strengthen and protect muscles, muscles become weakened and damaged. Elevidys is intended to deliver a gene that codes for a shortened, functional form of dystrophin to muscle cells.

Duchenne muscular dystrophy is a rare, fatal neuromuscular genetic disease that occurs in approximately one in every 3,500-5,000 newborn males worldwide. Symptoms of DMD usually appear in infants and toddlers and usually include developmental delays such as difficulty in walking, climbing stairs or standing from a sitting position. As the disease progresses, muscle weakness in the lower limbs spreads to the arms and other areas. The condition is universally fatal, and patients usually succumb to the disease in their twenties.

“Our confirmatory trial, should read out in the fourth quarter of this year. If [it] confirms the benefits seen in our prior trials, Sarepta will move rapidly to submit a BLA supplement to expand the approved label as broadly as good science permits.” said Sarepta President and CEO Doug Ingram.  (Source: Sarepta Therapeutics Website, 22 June, 2023)