Sanofi grapples with rejection of potential first-in-class orphan drug, faults third-party manufacturing
A snap review didn’t lead to a quick OK for Sanofi’s sutimlimab. A day after the FDA’s supposed decision date on the orphan drug, the French pharma giant said it was handed a rejection.
The complete response letter cites “certain deficiencies identified by the agency during a pre-license inspection of a third-party facility responsible for manufacturing,” Sanofi wrote. Regulators apparently didn’t take any issue with the clinical data or safety.
Picked up in the $11.6 billion Bioverativ acquisition, the drug is a C1s inhibitor targeting cold agglutinin disease — a rare disorder where the immune system runs amok attacking healthy red blood cells. Patients can suffer from severe anemia and life-threatening thrombotic events.
Just months before it was acquired Bioverativ, a Biogen spinout originally focused on hemophilia, had dished out $825 million to buy out the original developer of the drug. True North Therapeutics had already scored breakthrough therapy designation through the backing of OrbiMed Advisors, Kleiner Perkins Caulfield & Byers, MPM Capital, SR One and Baxter Ventures.
Sitting at the intersection of immunology, rare diseases and rare blood disorders, sutimlimab fits nicely within the kind of specialty care focus that CEO Paul Hudson laid out last year as he chopped the legacy pipeline in diabetes and cardio. R&D chief John Reed has also touted it as a first-in-class targeted therapy that “has the potential to change the treatment paradigm for CAD.”
It cleared a small pivotal study involving 24 patients, meeting the composite endpoint measuring hemoglobin and need for transfusion while improving other metrics of hemolysis, anemia and fatigue.
The durability portion of the study is ongoing — although Sanofi said it won’t factor into the current BLA.
“Satisfactory resolution of the observations by the third-party manufacturer is required before the BLA can be approved and Sanofi remains in close contact with the FDA and the third-party manufacturer to reach a resolution in a timely manner,” it wrote.