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.
Tag Archive for: Sarepta Therapeutics
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).
Sarepta Therapeutics and Alnylam Pharmaceuticals have announced the pricing of $1 billion and $900 million in convertible senior notes respectively.
The FDA removed the clinical hold on Sarepta Therapeutics’ investigational Duchenne muscular dystrophy (DMD) therapy Tuesday after the company agreed to adjust its clinical trial protocols to include expanded monitoring of urine biomarkers.
The nonprofit biotech Cure Rare Disease (CRD) is headed to the clinic with a CRISPR therapeutic aimed at halting the progression of Duchenne muscular dystrophy in single-patient dosing.