Sanofi scores a snap review for a rare disease drug, another win for the comeback players in R&D
Just a few months after spotlighting promising results from a small study for one of its rare disease drugs, the Sanofi team behind another one of their rare disease drugs is boasting that they snagged a snap review at the FDA — the kind that usually presages a quick OK.
The drug is sutimlimab, a C1s inhibitor which they picked up in their Bioverativ acquisition. The drug is used to treat cold agglutinin disease, where the immune system runs amok attacking healthy red blood cells.
Anemia and “profound fatigue” are common side effects, noted Sanofi R&D chief John Reed, who heralded the agency’s priority review status.
Researchers reported at ASH that 13 of the 24 patients in the small study met the composite endpoint, with “62.5% (n=15) of patients achieving a hemoglobin ≥ 12 g/dL or an increase of at least 2 g/dL and 71% (n=17) of patients remaining transfusion-free after week 5.”
The FDA action date is November 13, but as there’s no other drug approved here, Sanofi may not have to wait that long.
The drug may not move the dial in a big way on revenue, but any successes in R&D are likely to get some big cheers. Reed and Sanofi have continued to steer a more independent path than in years past — when they relied on Regeneron for its biggest advances. And FDA approvals are a tangible sign of competence, the kind of validation the pharma giant still badly needs.