FDA approves Sarepta muscular dystrophy drug

June 23, 2023

Rare disease drugmaker Sarepta Therapeutics announced this week that it has received accelerated approval from the FDA for SRP-9001, to be sold as Elevidys.

The one-time treatment is a gene therapy based on adeno-associated virus, now approved for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene. 

The drug's approval hinged on data from three ongoing clinical studies: SRP-9001-101, SRP-9001-102, and SRP-9001-103. The basis for accelerated approval primarily relied on data obtained from SRP-9001-102 and SRP-9001-103, with over 80 treated patients from all three studies contributing to the safety profile of the therapy.

During a conference call with investors, CEO Douglas Ingram stated that the therapy — which will be manufactured by Catalent — has a wholesale acquisition cost of $3.2 million. The price tag makes it the second most expensive prescription drug available currently, behind CSL Behring's Hemgenix. 

Ingram also expressed the significance of the approval by stating, "The approval of Elevidys is a landmark moment for Duchenne treatment. Elevidys is the first and only gene therapy authorized for Duchenne, and this approval brings us closer to our objective of introducing a treatment that has the potential to change the course of this degenerative disease."

Developed in collaboration with Roche, the drug facilitates the specific generation of functional parts of dystrophin, a protein crucial for DMD, genetic mutations affect the protein's structure, impairing its normal function and leading to various musculoskeletal symptoms. Although the disease is rare, it is life-threatening and affects around 1 in every 3,500-5,000 newborn boys annually.