The U.S. Food and Drug Administration has approved the expanded use of Sarepta Therapeutics' gene therapy, Elevidys, for patients with Duchenne muscular dystrophy aged four and older.
The U.S. Food and Drug Administration's (FDA) approval includes traditional approval for ambulatory patients aged four and older, as well as accelerated approval for non-ambulatory patients. Despite regulatory delays and questions about its effectiveness, Elevidys has been approved for broader use.
In its late-stage trial, Elevidys did not significantly improve motor function in patients aged four to seven compared to a placebo at 52 weeks. However, it met all secondary goals in the trial, with no new safety concerns reported.
Debra Miller, founder and CEO of CureDuchenne, a nonprofit organization for patient support and Duchenne muscular dystrophy (DMD) research, noted the difficulty in tracking progress through walk tests and endurance measures in patients. "From a parent's standpoint, you could watch your child deteriorate and hope for something better, or you can take advantage of something now," Miller said.
DMD affects approximately one in 3,500 male births worldwide, leading to progressive weakening of skeletal and heart muscles. Patients typically have a life expectancy of up to 25 years, according to the Cleveland Clinic.
Sarepta's CEO, Douglas Ingram, stated that the company is prepared to scale up its manufacturing capacity in response to the expanded approval. Continued approval for use in non-ambulatory patients may depend on the results of a confirmatory trial.
It is important to note that Elevidys is not recommended for patients with specific mutations in the DMD gene. Sarepta has also partnered with Roche for the commercialization of Elevidys outside the United States.