Expanded indication sees DMD treatment approved for patients 4 years and up

Expanded indication sees DMD treatment approved for patients 4 years and up | Image Credit: © Calin - © Calin - stock.adobe.com.

The FDA has approved an expanded label for delandistrogene moxeparvovec-rokl (ELEVIDYS; Sarepta Therapeutics) to include Duchenne muscular dystrophy (DMD) patients aged 4 years and older with a confirmed mutation in the DMD gene, according to a press release from Sarepta.¹

For ambulatory Duchenne patients, delandistrogene moxeparvovec-rokl was granted traditional approval, while accelerated approval was granted for non-ambulatory patients.¹

"The initial approval of ELEVIDYS was a significant milestone," said Jerry Mendell, MD, co-inventor of ELEVIDYS, senior advisor for Medical Affairs at Sarepta. "The expanded indication means clinicians now have a treatment option for the great majority of boys and young men living with Duchenne. This expansion speaks to the success of the science, the evidence and the improvements in the trajectory of the disease we have seen to date across studies.”¹

Delandistrogene moxeparvovec-rokl was approved as the first gene therapy to treat pediatric patients aged 4 to 5 years with DMD on June 22, 2023. The original approval for the recombinant gene therapy that is administered as a single intravenous dose was supported by a randomized clinical trial. The trial established that delandistrogene moxeparvovec-rokl increased the expression of the Elevidys micro-dystrophin protein observed in delandistrogene moxeparvovec-rokl-treated patients aged 4 to 5 years with DMD.²

“Duchenne is a relentlessly progressive, degenerative disease, robbing children of muscle function,” said Jerry Mendell, MD, pediatric neurologist and principal investigator, Center for Gene Therapy at Nationwide Children’s Hospital at the time of original approval. “The increases in ELEVIDYS dystrophin expression and the functional results that we see can make a difference in the lives of our patients.”²

The disease occurs because of a defective gene resulting in the absence of dystrophin, a protein that helps keep the body’s muscle cells intact. Those with DMD can have symptoms like trouble with walking and running, frequent falls, learning disabilities or difficulties, fatigue, heart issues (due to impact on heart muscle functioning), and breathing problems (because of weakening respiratory muscles that involve lung function.)²

According to Sarepta, the company will conduct and submit results of a randomized controlled trial to verify and confirm clinical benefits of delandistrogene moxeparvovec-rokl in non-ambulatory DMD patients. The ENVISION study is a global, randomized, double-blind, and placebo-controlled phase 3 trial of delandistrogene moxeparvovec-rokl in non-ambulatory and older ambulatory patients. The study is underway, according to Sarepta, and is "intended to serve as this postmarketing requirement."¹

"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,” said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research in a statement from the FDA. “We remain steadfast in our commitment to help advance safe and effective treatments for patients who desperately need them.”³

References:

1. Sarepta Therapeutics Announces Expanded US FDA Approval of ELEVIDYS to Duchenne Muscular Dystrophy Patients Ages 4 and Above. Sarepta therapeutics. June 20, 2024. Accessed June 21, 2024. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-expanded-us-fda-approval-elevidys
2. Fitch, J. FDA approves first gene therapy to treat pediatric patients with Duchenne muscular dystrophy. Contemporary Pediatrics. June 22, 2023. Accessed June 21, 2024. https://www.contemporarypediatrics.com/view/fda-approves-first-gene-therapy-to-treat-pediatric-patients-with-duchenne-muscular-dystrophy
3. FDA expands approval of gene therapy for patients with Duchenne muscular dystrophy. FDA. Press release. June 20,2024. Accessed June 21, 2024. https://content.govdelivery.com/accounts/USFDA/bulletins/3a3fabc