DMD Gene Therapy Indication Expanded to Include Boys 4 and Older
The US Food and Drug Administration (FDA) recently approved an indication expansion for the gene therapy Elevidys (delandistrogene moxeparvovec-rokl) to include patients with Duchenne muscular dystrophy (DMD) who are 4 years and older and have a confirmed mutation in the DMD gene.
The gene therapy’s initial accelerated approval, which was granted in June 2023, restricted its use to ambulatory patients between ages 4 and 5, the website FirstWord Pharma reported. The article noted Elevidys’ expanded indication did not specify an upper-age limit for use despite clinical trial use in patients no older than 7.
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The expanded indication includes traditional FDA approval for patients who are ambulatory and accelerated approval for patients who are nonambulatory. Continued approval for nonambulatory patients may be contingent upon results from the ongoing phase 3 ENVISION study, a double-blind, randomized controlled trial that includes nonambulatory patients and older ambulatory patients with DMD, maker Sarepta Therapeutics stated.
“[T]he expansion of the Elevidys label to treat Duchenne patients aged 4 and above, regardless of ambulatory status, is a defining moment for the Duchenne community,” said Doug Ingram, president and chief executive officer, of Sarepta.
Elevidys is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene. The treatment is a single-dose, adeno-associated virus-based gene transfer therapy for intravenous infusion. It is designed to address mutations or changes in the DMD gene that result in the lack of dystrophin protein in patients with DMD.
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