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.
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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.
One of the tenets of “personalized medicine” is that medications will be tailored to individuals based on their personalized genetics. Last year, the U.S. Food and Drug Administration’s Center for Drug Evaluation and Research approved 50 drugs. A new study found that 33, or 66% of them, were supported with genomic data.
Sarepta Therapeutics announced Thursday that the U.S. Food and Drug Administration has put the Phase II trial of its Duchenne muscular dystrophy (DMD) candidate SRP-5051 (vesleteplirsen) on clinical hold following a serious safety signal.
