Sarepta Therapeutics, Inc.
NEWS
Since its inception in 1992, the FDA’s accelerated approval pathway has helped shepherd nearly 300 new drugs to the market. However, recent years have seen a number of high-profile market withdrawals and failed confirmatory trials.
The recent invalidation of an AAV gene therapy patent overlooks the complexity of innovation in biotechnology and could put a broad swath of intellectual property at risk.
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.
As Sarepta Therapeutics moves closer to full approval and an expanded label for its gene therapy, some experts push back on clinical efficacy and cost while others note the hope it provides patients with Duchenne muscular dystrophy.
Ahead of the FDA’s June target action date for Sarepta’s Duchenne muscular dystrophy gene therapy, ICER Chief Medical Officer David Rind blasted the regulator’s accelerated pathway in a JAMA viewpoint article.
With several recent approvals in the space and more on the horizon, BioSpace looks at some of the key decisions and their larger significance both for patients and science.
The annual observance highlights how far the industry has come and serves as a wake-up call for how far it must still go to meet the needs of patients with rare diseases.
Sarepta Therapeutics on Wednesday 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.
The regulator has granted a priority review of the efficacy supplement for Sarepta Therapeutics’ gene therapy Elevidys with a target decision date of June 21, 2024.
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