FDA approves first gene therapy for Duchenne muscular dystrophy
The U.S. Food and Drug Administration (FDA) has granted accelerated approval for Sarepta's first gene therapy for Duchenne muscular dystrophy. The drug will cost $3.2 million.A U.S. regulator has granted accelerated approval for Sarepta Therapeutics' first-of-its-kind gene therapy Elevidys to treat Duchenne muscular dystrophy (DMD), Reuters reported.
Elevidys delivers a functional copy of the dystrophin gene to the patient's muscle tissues. In DMD, mutations in this gene show characteristic muscle weakness and developmental delay. The disease mainly affects boys and is usually fatal.
Elevidus received accelerated FDA approval for outpatients ages 4 to 5 with a confirmed mutation in the dystrophin gene. Sarepta sought approval of the drug for all DMD patients who can walk.
The drug is in the final stages of a clinical trial to confirm efficacy. The first data are expected by December. Experts and analysts say the approval of Elevidus could open the door to more effective gene therapies against the disease. For example, Pfizer is developing its own version of gene therapy against DMD.
The therapy will cost $3.2 million wholesale, Sarepta CEO Douglas Ingram said. Currently, the most expensive drug in the world is a gene therapy against hemophilia from the Australian drugmaker CSL at $3.5 million.