Sarepta Therapeutics, Inc.
NEWS
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.
Gene therapy company Sarepta Therapeutics provided additional data for SRP-5051 in treating Duchenne muscular dystrophy patients who are amenable to exon 51 skipping.
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.
Recent FDA approvals of novel drugs based on less-than-stellar clinical evidence point to a trend toward regulatory flexibility—particularly in indications with very high unmet need.
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