Sarepta Therapeutcis, a medical research and drug development company, has announced the approval of Elevidys, its gene therapy for Duchenne’s muscular dystrophy (DMD), by United Stated Food and Drug Administration (FDA). Elevidys is the first gene therapy approved for the treatment of DMD.
DMD is an X-linked, degenerative muscle disease generally manifesting in early childhood, eventually leading to effective paralysis due to muscle weakness. The life expectancy is around 26-years of age and there is currently no curative therapy available.
Elevidys (delandistrogene moxeparvovec-rokl) consists of modified adeno-associated virus (AAV) particles of serotype rh74, carrying a gene coding for micro-dystrophin, which is a shortened version of the dystrophin gene mutated in DMD patients. The intravenous infusion at the dose of 1.33 x 10^14 vector genomes/kg is a one-time treatment, with current data supporting effectiveness at least up to 4 years (SRP-9001-103).