What is Elevidys and how does it work?
A one-time $3.2 million infusion using an AAVrh74 vector to deliver a micro-dystrophin gene to muscle cells in boys with DMD — the first gene therapy for the disease and among the most expensive drugs ever sold at launch.
DMD is caused by mutations that prevent the body from producing dystrophin, a protein essential for muscle. It affects about 1 in 5,000 boys and causes progressive muscle loss, typically leading to death in early adulthood. Elevidys uses a modified adeno-associated virus (the AAVrh74 vector) to deliver a gene for a shortened version of dystrophin, called micro-dystrophin, to muscle cells. It is given once, by infusion. Sarepta set its wholesale price at $3.2 million, making it among the most expensive drugs ever sold. It generated $200.3 million in sales in 2023, its first year. Because the therapy uses high doses of viral vector, it carries risks common to AAV gene therapies, including immune reactions and liver injury.