Sarepta Therapeutics is anticipating full FDA approval and a label expansion for its gene therapy, Elevidys, with the company indicating on its Q1 earnings call last week that a draft label is imminent. But the therapy has come under the microscope regarding its high cost and clinical effectiveness.
Tag Archive for: Elevidys
Sarepta Therapeutics called the launch of the gene therapy for Duchenne muscular dystrophy “exceptional” but the company does not expect to see significant growth in the first half of 2024 due to its currently limited patient pool.
Sarepta Therapeutics said on Friday that the U.S. Food and Drug Administration would review an application seeking traditional approval for its gene therapy to treat a muscle-wasting disorder by June 21, months after it failed the main goal of a confirmatory trial.
Despite failing to hit the primary endpoint in the Phase III EMBARK study, the company plans to file for a label expansion for its Duchenne muscular dystrophy gene therapy Elevidys.
