FDA Broadens Approval for Duchenne Muscular Dystrophy Gene Therapy

On MONDAY, June 24, 2024, the U.S. Food and Drug Administration (FDA) announced the expanded approval of Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy specifically designed for individuals aged 4 and older with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene. This therapy involves a single-dose, intravenous administration that aims to produce Elevidys micro-dystrophin, a shortened version of the dystrophin protein, to help mitigate the effects of DMD.

Duchenne muscular dystrophy is a severe genetic disorder characterized by progressive muscle degeneration and weakness. It primarily affects boys and results from mutations in the DMD gene, which encodes the dystrophin protein critical for muscle cell function. The standard dystrophin protein is much larger (427 kDa) compared to the Elevidys micro-dystrophin (138 kDa), but Elevidys includes selected domains of this essential protein to provide therapeutic benefits.

The FDA's decision to grant traditional approval for Elevidys in ambulatory individuals aged 4 and older with a confirmed mutation in the DMD gene follows extensive clinical trials. These included two double-blind, placebo-controlled studies and two open-label studies, involving a total of 218 male participants. Although Elevidys did not achieve its primary endpoint of improvement in the North Star Ambulatory Assessment compared to a placebo, it successfully met several key secondary outcome measures. These included time to rise from the floor, the 10-meter walk/run test, time to ascend four steps, and levels of creatine kinase, an enzyme indicative of muscle damage.

Additionally, the FDA granted accelerated approval for Elevidys in nonambulatory individuals aged 4 and older based on these trials. The agency concluded that increased levels of micro-dystrophin are likely to predict clinical benefits for the nonambulatory population, justifying the accelerated approval for these patients. This expanded approval underscores the FDA's commitment to addressing the critical and urgent treatment needs posed by DMD.

The most common side effects reported during the trials for Elevidys included nausea, vomiting, acute liver injury, fever, and thrombocytopenia (a condition characterized by low platelet counts). Despite these potential side effects, the expanded approval of Elevidys offers new hope for a broader range of patients dealing with this debilitating condition.

Dr. Peter Marks, M.D., Ph.D., from the FDA Center for Biologics Evaluation and Research, highlighted the significance of this approval, stating, "The 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."

The expanded approval for Elevidys was granted to Sarepta Therapeutics, the company responsible for developing this innovative gene therapy. This marks a significant milestone in the treatment of Duchenne muscular dystrophy, offering a new therapeutic option for those affected by this genetic disorder.

How to obtain the latest research advancements in the field of biopharmaceuticals?

In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!