Sarepta Therapeutics gains expanded FDA approval for DMD treatment
Betsy Goodfellow | June 21, 2024 | News story | Medical Communications | DMD, Musculo-skeletal disorder, Sarepta Therapeutics
Sarepta Therapeutics has announced that the US Food and Drug Administration (FDA) has approved an expansion to the labelled indication for Elevidys (delandisttrogene moxaparvovec-rokl) to include individuals with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who are at least four years of age.
The FDA has approved the drug for ambulatory patients, as well as granted accelerated approval for non-ambulatory patients. The continued approval for non-ambulatory DMD patients may depend upon verification of the clinical benefit in a confirmatory trial.
Sarepta has confirmed that it has committed to conducting and sharing results from a randomised, controlled trial in order to verify and confirm the clinical benefit of Elevidys in patients with DMD who are non-ambulatory.
Doug Ingram, president and chief executive officer at Sarepta, commented: “Representing many years of dedicated research, development, investment and creative energy, the expansion of the Elevidys label to treat Duchenne patients aged four and above, regardless of ambulatory status, is a defining moment for the Duchenne community. Today also stands as a watershed occasion for the promise of gene therapy and a win for science. At this pivotal moment, I want to give warm thanks to Drs Jerry Mendell and Louise Rodino-Klapac for their dogged, 20-year pursuit of a gene therapy to treat this ruthless and life-robbing disease; to the FDA for following the scientific evidence to speed delivery of a therapy for a life-threatening rare disease to waiting patients; and to the many clinical investigators and courageous Duchenne families who have participated in the multiple studies that led to this important day.”
Betsy Goodfellow
Related Content
Pfizer shares update on DMD gene therapy clinical trial
Pfizer has announced an update from its phase 3 randomised, multicentre, double-blind, placebo-controlled CIFFREO trial, …
Santhera announces Early Access Program by partner Sperogenix for DMD treatment
Santhera Pharmaceuticals has announced that its partner Sperogenix Therapeutics has launched a paid-for Early Access …
FDA pre-meeting report raises concerns over Sarepta’s gene therapy for Duchenne muscular dystrophy
The US Food and Drug Administration (FDA) has released a report in preparation for its …
