It appears likely that a new drug to treat amyotrophic lateral sclerosis (ALS) will get approval in the U.S. Regulators shared their thinking ahead of an advisory committee meeting.
The Food and Drug Administration committee is scheduled to meet Wednesday to discuss Biogen Inc.
BIIB,
and Ionis Pharmaceuticals Inc.’s
IONS,
tofersen as a treatment for a rare type of ALS that is associated with the superoxide dismutase 1 (SOD1) gene.
The FDA, in documents published Monday, suggests there is a clinical benefit for the drug, even though a Phase 3 clinical trial failed to meet the primary endpoint. The committee is made up of independent medical experts who are expected to make a recommendation about the drug to the FDA, which is not required to follow their advice but often does.
Fewer than 500 people in the U.S. have this form of ALS, and there are no approved treatments at this time, according to the FDA. The regulator last fall approved Amylyx Pharmaceuticals Inc.’s
AMLX,
new ALS therapy.
Wall Street analysts largely view the approval of tofersen as likely. However, most analysts say they don’t expect this drug to drive the kind of impact they’re looking for on Biogen’s business. Instead, all eyes remain on lecanemab, Biogen and Eisai’s
4523,
treatment for Alzheimer’s disease, which received accelerated approval in January. (Lecanemab’s brand name is Leqembi.)
“Based on the briefing documents, we see a greater chance of approval than not,” UBS analysts wrote in an investor note about tofersen on Monday. “However, clearly the primary driver of valuation and sentiment for [Biogen] remains the lecanemab full approval and launch.”
UBS predicts worldwide sales opportunity for this type of ALS drug is $300 million, while William Blair analysts estimate $230 million in peak sales for tofersen with this indication.
“We would view the potential approval of tofersen as a major advancement in treating drastically underserved patients and indicative of regulatory flexibility in a post-Billy Dunn era at the FDA Office of Neuroscience,” they wrote. “Given the totality of the data to-date and what appear to be favorable [advisory committee] briefing documents, we are optimistic heading into the [meeting].”
Dunn, a longtime neuroscience official at the FDA, recently left the agency. His departure was viewed as negative for companies with neuroscience drugs in development.
The FDA is set to make a decision on whether to grant approval to the drug by April 25.
