The FDA’s authorization of therapies that fail their efficacy endpoints casts doubt on the regulator’s accelerated approval pathway, according to an article from David Rind, chief medical officer of the drug pricing non-profit Institute for Clinical and Economic Review, published in the Journal of the American Medical Association. In his viewpoint piece, Rind specifically called out Sarepta Therapeutics’ gene therapy Elevidys, which won the FDA’s accelerated approval in June 2023—despite falling short of its primary efficacy endpoint in two studies.
Tag Archive for: Duchenne muscular dystrophy
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.
PTC Therapeutics said on Thursday the European health regulator’s advisory panel reaffirmed a negative opinion for its drug used to treat an inherited progressive muscle-wasting disorder, sending its shares down over 11% in extended trading.
Shares of drug developer Dyne Therapeutics doubled in premarket trading on Wednesday after its experimental therapy for a muscle-wasting disorder showed promise in a small, early-stage 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.
Santhera Pharmaceuticals and partner Catalyst Pharmaceuticals got the FDA’s greenlight for their Duchenne muscular dystrophy drug Agamree, which is expected to launch in the first quarter of 2024.
The New England Journal of Medicine study suggests that the 2022 death of a Duchenne muscular dystrophy patient was due to an adverse effect of recombinant AAV used to deliver the gene therapy.
Following a handful of clinical failures and changes to its leadership, FibroGen is trying to chart a more positive path forward by focusing on four strategic areas.
The company was evaluating the efficacy and safety of pamrevlumab in a late-stage trial in patients with non-ambulatory Duchenne muscular dystrophy (DMD), where upper limb function of the patients are affected, versus a placebo.