The FDA approved the first gene therapy for some pediatric patients with Duchenne muscular dystrophy, a rare genetic condition that weakens the body's muscles, on June 22.
The approval is relatively small: The drug, Elevidys, is indicated for 4- and 5-year-olds "with a confirmed mutation in the DMD gene who do not have a pre-existing medical reason preventing treatment with this therapy," the FDA said.
Elevidys is a single intravenous dose that was granted accelerated approval after a clinical trial found the drug increased a protein present in normal muscle cells among patients aged 4 and 5. It will cost $3.2 million, its drugmaker Sarepta Therapeutics said June 22.
Most therapies for DMD focus on symptoms of the disease that affects about 1 in every 3,300 males and kills most patients in their 20s or 30s, but Sarepta's newly approved medication is the first to target the condition's genetic cause.