Elevidys, the first gene therapy for pediatric patients aged 4 to 5 years with Duchenne muscular dystrophy (DMD) and a confirmed mutation in the DMD gene, has been approved by the U.S. Food and Drug Administration (FDA).
According to a press release by the FDA, Elevidys was approved through the department's Accelerated Approval pathway, which fast tracks FDA approval for drugs shown to be effective at treating serious diseases where there is an "an unmet medical need." Through this pathway, patients can have earlier access to new drugs while companies continue to conduct clinical trials.
"Today's approval addresses an urgent unmet medical need and is an important advancement in the treatment of Duchenne muscular dystrophy, a devastating condition with limited treatment options, that leads to a progressive deterioration of an individual's health over time," said Peter Marks, MD, PhD, director of the FDA's Center for Biologics Evaluation and Research. "The FDA remains committed to facilitating the development of innovative new therapies to reduce the impact of debilitating diseases and to improve outcomes and quality of life for those affected."
DMD is a rare genetic condition that leads to progressive muscle weakness and deterioration over time, according to the FDA. It primarily affects boys, with symptoms typically appearing between the ages of 3 and 6. DMD is caused by a defective gene that results in the absence of dystrophin, a vital protein for muscle cell integrity. Common symptoms include difficulty walking, frequent falls, fatigue, learning difficulties, heart problems and respiratory issues. The disease can be threatening, with most individuals succumbing to heart or respiratory failure in their 20s or 30s. Current treatments focus on managing symptoms, such as corticosteroid medications, exercise programs and assistive devices. While some genetic mutations can be targeted with antisense oligonucleotides (ASOs), they are limited in scope and require repeated administration.
Elevidys is a recombinant gene therapy that introduces a gene into the body, leading to the production of a shortened protein called Elevidys micro-dystrophin. This protein contains selected domains from the dystrophin protein found in normal muscle cells. The therapy is administered through a single intravenous dose. FDA approval was based on data submitted by the sponsor, including a study conducted in two parts. The study involved individuals who were treated with Elevidys or a placebo and followed for 48 weeks in part 1. In part 2, those who received placebo initially were switched to Elevidys, and vice versa. Additional follow-up was conducted for another 48 weeks.
It's important to note that the clinical benefit of Elevidys, such as improved motor function, has not been established. As a condition of approval, the FDA has required the company to conduct a clinical study to confirm the drug's effectiveness in improving physical function and mobility in ambulatory DMD patients with a confirmed mutation in the DMD gene. This study is ongoing and fully enrolled. The FDA will review the trial data as quickly as possible to determine if any further action, such as revising the indication or withdrawing Elevidys, is necessary.
