Sarepta Therapeutics announced Thursday afternoon that the Food and Drug Administration approved Elevidys, its gene therapy to treat Duchenne muscular dystrophy (DMD).
The FDA greenlit the investigational drug under its accelerated approval pathway, clearing the gene therapy to be administered to children between the ages of 4 and 5.
DMD is a rare, degenerative disease that affects fewer than 50,000 Americans but is notable for the progressive weakness of skeletal muscles that control movement. The disease affects 1 in 5,000 live male births annually and is typically inherited through the mother, according to the Muscular Dystrophy Association.
Currently, there is no cure for DMD but Sarepta’s treatment is viewed by many in the industry as a major step forward.
“Today’s approval addresses an urgent unmet medical need and is an important advancement in the treatment of Duchenne muscular dystrophy, a devastating condition with limited treatment options, that leads to a progressive deterioration of an individual’s health over time,” said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, in a statement.
The decision to approve the treatment came just over one month after the FDA’s outside experts voiced concerns about Elevidys in a high stakes, all-day deliberation. Ultimately, the committee gave Elevidys a positive vote but the FDA ssubsequently delayed its decision date by one month.
Thirty minutes prior to the FDA’s announcement, Sarepta’s stock was halted from trading, though it resumed trading at a slightly higher level after the news was released.
Sarepta CEO Doug Ingram said in a statement that the approval is a “watershed moment” for treating DMD and brings the company closer to significantly altering the trajectory of the disease.
“As we prepare to launch Elevidys, we should acknowledge and celebrate the decades of dedication and work from the patient community, families, clinicians, and our Sarepta colleagues that resulted in today’s approval,” he stated. “Our confirmatory trial, EMBARK, should read out in the fourth quarter of this year. If EMBARK 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.”
Looking forward, Sarepta is now shifting focus to prepare for the launch of Elevidys in the U.S. Sarepta also said it would host a conference call later Thursday afternoon to discuss the FDA approval.
