Sarepta Therapeutics gene therapy, Elevidys, has received traditional FDA approval after failing its confirmatory test, expanding use to a wider group of Duchenne muscular dystrophy patients. The therapy works by producing a smaller version of dystrophin, a protein key to muscle function. The therapy was initially approved based on Phase 2 data showing micro-dystrophin production, with a confirmatory study to show the therapy’s benefits.
While Elevidys did not meet its main goal in the Phase 3 test, it showed significant improvements in secondary endpoints such as walking speed and muscle function. FDA reviewers were unconvinced by the data, citing an inability to determine whether the benefits were from Elevidys or by chance. However, the FDA’s top biologics official, Peter Marks, found the observations meaningful for patients, acknowledging that a partial correction in muscle problems could be critical for functional abilities.
This approval comes after Pfizer’s gene therapy for Duchenne failed its Phase 3 trial and is still under review. Elevidys has a $3.2 million wholesale price for the one-time treatment, with projected sales of $3.2 billion in 2025. The approval is expected to have a lasting impact on the gene therapy sector, shaping FDA reviews for deadly diseases with limited treatment options.
Sarepta is conducting additional clinical tests to expand Elevidys treatment to younger patients and those who are no longer ambulatory. Roche holds the rights to market Elevidys outside the U.S. as part of a partnership. The approval of Elevidys represents a significant milestone in the treatment of Duchenne muscular dystrophy and is expected to have a positive impact on the gene therapy landscape.
June 21, 2024
Artificial Intelligence
FDAcop
