SRPT $169 +35% Elevidys is among the most expensive treatments in the world with a list price of $3.2 million.
Sales of the therapy came in at $200.4 million in 2023 and are expected to rise to $1.07 billion this year, as per LSEG data.
DMD is an inherited progressive muscle-wasting disorder that affects an estimated one-in-3,500 male births worldwide, according to the U.S.-based National Organization for Rare Disorders.
Elevidys' could now be given to about 13,000 patients, or nearly 90% of U.S. patients, said BMO Capital analyst Kostas Biliouris.
The decision also follows concerns around an "uphill battle" to secure traditional approval after the therapy failed to meet the main goal in a key confirmatory late-stage trial.
A top official of the agency, Peter Marks, pushed for the "no strings attached" label for Elevidys overruling recommendations, RBC markets analyst Luca Issi said. FDA staff had recommended declining the expansion, citing a lack of benefit to certain patients, documents show.
The global treatment market for DMD - including gene therapies and other drugs - is expected to grow to $11.47 billion by 2034, according to ResearchandMarkets.com.
