US-based Sarepta Therapeutics, Inc. (NASDAQ: SRPT) has announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus-based gene therapy co-developed by Roche. This treatment is designed for ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene. This marks the first in vivo gene therapy to be fast-tracked for marketing worldwide.
Understanding Duchenne Muscular Dystrophy (DMD)
DMD is a rare and fatal X-linked degenerative neuromuscular disease, and it is one of the most common fatal genetic diseases. The pathogenesis of DMD is due to a mutation in the gene encoding dystrophin, leading to the loss or functional defect of dystrophin. Patients may experience inflammatory reactions shortly after birth, which can lead to muscle fibrosis, atrophy, and degeneration. The life expectancy of patients is typically less than 40 years due to respiratory and/or heart failure. Globally, there is one case of DMD in every 3,500 to 5,000 male infants.
Elevidys: A Single-Dose Gene Transfer Therapy
Elevidys (delandistrogene moxeparvovec-rokl) is a single-dose gene transfer therapy for intravenous infusion, designed to address the underlying cause of Duchenne muscular dystrophy by targeting the production of Elevidys micro-dystrophin in skeletal muscle. This one-time treatment is priced at USD 3.2 million, which is considered cost-effective when compared to the standard of care expenses for DMD, which range from USD 5 million to USD 13 million.-Fineline Info & Tech
