FDA expands approval of gene therapy for patients with Duchenne Muscular Dystrophy

The U.S. Food and Drug Administration recently  expanded the approval of Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for the treatment of Duchenne muscular dystrophy (DMD) for ambulatory and non-ambulatory individuals four  years of age and older with DMD with a confirmed mutation in the DMD gene. 

Elevidys was previously approved under accelerated approval for ambulatory individuals four through five years of age with DMD with a confirmed mutation in the DMD gene. With the recent action taken, Elevidys received traditional approval in ambulatory individuals four years of age and older with DMD with a confirmed mutation in the DMD gene, and accelerated approval in non-ambulatory individuals four years of age and older with DMD with a confirmed mutation in the DMD gene. 

In making this decision, the FDA considered the totality of the evidence, including the potential risks associated with the product, the life-threatening and debilitating nature of the disease and the urgent unmet medical need.

“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, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. “We remain steadfast in our commitment to help advance safe and effective treatments for patients who desperately need them.” 

Duchenne muscular dystrophy is a rare and serious genetic condition which worsens over time, leading to weakness and wasting away of the body’s muscles. The disease occurs due to a defective gene that results in abnormalities in, or absence of, dystrophin, a protein that helps keep the body’s muscle cells intact. 

Elevidys is a recombinant gene therapy designed to deliver into the body a gene that leads to production of Elevidys micro-dystrophin, a shortened protein (138 kDa, compared to the 427 kDa dystrophin protein of normal muscle cells) that contains selected domains of the dystrophin protein present in normal muscle cells. The product is administered as a single intravenous dose. 

Elevidys was initially approved in June 2023 through the Accelerated Approval pathway, under which the FDA may approve drugs for serious or life-threatening diseases where there is an unmet medical need and the drug is shown to have an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit to patients (improving how patients feel or function, or whether they survive longer), or an effect on a clinical endpoint that can be measured earlier than irreversible morbidity or mortality that is reasonably likely to predict an effect on irreversible morbidity or mortality or other clinical benefit. This pathway can allow earlier approval, while the company conducts clinical trials to verify the predicted clinical benefit.