Sarepta has been hit with another patent infringement lawsuit, this time from Sanofi and its subsidiary Genzyme alleging that the biotech used protected technology related to AAV vectors.
Tag Archive for: Sarepta Therapeutics
Sarepta Therapeutics’ gene therapy Elevidys will be available to Duchenne muscular dystrophy (DMD) patients who are at least 4 years old, regardless of whether they can walk, the FDA said Thursday.
While Elevidys was previously approved under accelerated approval for ambulatory individuals 4 through 5 years with a confirmed mutation in the DMD gene, Thursday’s expanded approval includes ambulatory and non-ambulatory patients 4 years of age and older with a confirmed mutation in the DMD gene.
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.
Sarepta’s Elevidys, approved Thursday as the first gene therapy for Duchenne muscular dystrophy, has been priced at $3.2 million per patient, making it one of the world’s most expensive medicines.
Shares of Sarepta Therapeutics soared 29% premarket on Monday as a backing by the U.S. health regulator’s advisers increased the certainty of an accelerated approval for the company’s gene therapy for a muscle-wasting genetic disorder.
Sarepta Therapeutics Inc.’s shares fell more than 6% on Thursday as Stat News reported that the U.S. health regulator’s staff were inclined to reject the company’s gene therapy which is currently under review.
Shares of Reata Pharmaceuticals Inc. soared 175% to hit a one-year high in early trading on Wednesday, after the U.S. Food and Drug Administration (FDA) approved its rare disease drug and ended years of uncertainty over its future.
The FDA has accepted Roche and Sarepta’s Biologic License Application for the accelerated approval of SRP-9001 (delandistrogene moxeparvovec), an investigational gene therapy for Duchenne muscular dystrophy (DMD).