FDA expands approval of Elevidys to children aged 4 years and older with DMD gene mutation
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The FDA has announced approval of a label expansion for Elevidys to include patients with Duchenne muscular dystrophy aged 4 years and older with a confirmed mutation in the DMD gene.
According to a release from manufacturer Sarepta Theraputics, the expansion means Elevidys (delandistrogene moxeparvovec-rokl), a single-dose adeno-associated virus-based gene transfer therapy, received traditional approval in ambulatory patients aged 4 years and older, as well as accelerated approval in non-ambulatory patients.
The FDA had initially approved Elevidys for pediatric populations with Duchenne in June 2023.
“Representing many years of dedicated research, development, investment and creative energy, the 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,” Sarepta President and CEO Doug Ingram said in the release.
Continued approval for non-ambulatory patients with Duchenne may be contingent upon verification of clinical benefit in a confirmatory trial, Sarepta said in the release.
In a concurrent release, the FDA said accelerated approval was based on evidence that the mechanism of action of Elevidys was similar for both ambulatory and non-ambulatory individuals, with increased levels of micro-dystrophin likely to predict clinical benefit in non-ambulatory patients.
“Today’s approval broadens the spectrum of patients with Duchenne muscular dystrophy eligible for this therapy, helping to address the ongoing, urgent treatment need for patients with this devastating and life-threatening disease,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said in the release.
Reference:
FDA expands approval of gene therapy for patients with Duchenne muscular dystrophy. https://www.fda.gov/news-events/press-announcements/fda-expands-approval-gene-therapy-patients-duchenne-muscular-dystrophy. Published June 20, 2024. Accessed June 21, 2024.