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June 23, 2023 – The Food and Drug Administration (FDA) has approved the first gene therapy for the treatment of Duchenne muscular dystrophy (DMD) in patients ages 4 through 5 years with a confirmed mutation in the DMD gene.
The FDA granted approval of Elevidys (delandistrogene moxeparvovec-rok) from Sarepta Therapeutics Inc. through an accelerated approval pathway, under which it may approve drugs for serious or life-threatening diseases where there is an unmet medical need.
About one in 3,300 boys is affected by DMD, a rare, serious genetic condition that worsens over time. DMD leads to weakness and wasting away of the muscles, with symptoms that include trouble walking and running, falling frequently, fatigue, learning difficulties, heart issues and breathing problems.
As the disease progresses, cardiac and respiratory issues can become life-threatening.
The disease occurs because of a defective gene that results in the absence of dystrophin — a protein that helps keep the body’s muscle cells intact.
Most current treatments for DMD address only symptoms, not the underlying cause.
Elevidys is a recombinant gene therapy that delivers 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 given to patients as a single intravenous dose.
“This is a very exciting approval for patients, families and clinicians in the Duchenne community,” said Garey H. Noritz, M.D., FACP, FAAP, chair of the AAP Council on Children with Disabilities and professor of pediatrics at The Ohio State University.
Read the full AAP article: “FDA approves first gene therapy for Duchenne muscular dystrophy.“
