Duchenne muscular dystrophy
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 three FDA approvals in the past 10 months, there is a lot of momentum in the Duchenne muscular dystrophy space. Here are five companies looking to keep it going.
Duvyzat, which will be sold in the U.S. by ITF Therapeutics, joins a growing market that includes recently approved gene therapy Elevidys and corticosteroid Agamree.
In advance of a March 21 PDUFA date, Italfarmaco on Tuesday reported that its drug candidate givinostat met the primary endpoint for treating Duchenne muscular dystrophy.
The FDA will close out a hectic month of March with a flurry of target action dates, including ones for lymphoma and CKD anemia treatments.
The recently launched company shared two posters at the Muscular Dystrophy Association’s annual conference this week ahead of a March 21 PDUFA date.
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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