Big things were in store Friday for Sarepta Therapeutics (NASDAQ:SRPT) as an expanded use approval opened up a bigger market in the U.S. for its gene therapy for patients with Duchenne muscular dystrophy (DMD).
On Thursday, the U.S. Food and Drug Administration granted traditional approval for the therapy, Elevidys, in patients four years and above who can walk, as well as an accelerated approval for those who cannot.
William Blair’s Tim Lugo estimated potential sales of Elevidys to reach $3 billion in 2025 and peak sales of $5 billion in 2027 with the expanded approval.
“Many patients below age four are not yet diagnosed but will age into the label, opening the DMD market to all current and future patients who will be eligible for treatment,” Lugo wrote in a note.
Sales of Elevidys came in at $200.4 million for 2023 and are expected to rise to $991.91 million 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.
Given the high demand and acceptable safety, analysts flagged manufacturing and supply concerns as the only limiting factors.
Elevidys’ addressable population is now about 13,000 patients, or nearly 90% of patients in the U.S., according to BMO Capital analyst Kostas Biliouris.
SRPT shares popped $46.20, or 37.4% to $169.70
