The FDA indicated it could potentially grant an accelerated approval for the therapy, called SRP-9001, initially for use in Duchenne patients aged 4 and 5.
Tag Archive for: Duchenne muscular dystrophy
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.
The potential one-time treatment is the first gene therapy for the rare muscle-wasting disorder to reach the FDA’s doorstep, and could change the way patients with the disease are treated if approved.
The FDA is kicking off May with three target action dates and three advisory committee meetings, including one for a high-profile investigational gene therapy.
Shares of Sarepta Therapeutics slumped as much as 21% on Friday over uncertainty on approval of its gene therapy for Duchenne muscular dystrophy after the U.S. health regulator reversed its decision on a panel meeting.
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).
Two struggling Boston-based gene therapy companies – Solid Biosciences and AavantiBio – announced Friday they are merging to develop a portfolio of neuromuscular and cardiac programs, initially focusing on a lead candidate to treat Duchenne muscular dystrophy.
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.
The company’s original plan was to not apply to regulators until 2023, but now, it plans to submit the drug to the FDA in the next few months, with plans to launch the drug in mid-2023.
For years, PTC Therapeutics has attempted to win regulatory approval for its Duchenne Muscular Dystrophy drug, Translarna (ataluren), but time and time again, the U.S. Food and Drug Administration has rejected the attempts. Now, the company believes it has data that will support another filing for possible approval.