New Products, Rare Diseases

Burnishing its rare disease drug ambitions, Sanofi picks up fast FDA OK for Cablivi — courtesy of a $4.8B buyout

Following up on last fall’s European approval for caplacizumab as a new treatment for a rare blood disease, Sanofi today followed up with the key to the big market in the US. The FDA has come through with an OK based on Phase III and Phase II data for acquired thrombotic thrombocytopenic purpura (aTTP).

The drug will now hit the market as Cablivi — at a cost of $270,000 per course — after getting a priority review at the agency, which is quick at whisking through new drugs when none are approved for a condition like this.

Richard Padzur

For Sanofi $SNY, today’s approval on top of the earlier news regarding the Phase III success for its cancer therapy isatuximab marks an unusual winning streak for Sanofi, which has been bedeviled by internal setbacks for years. It also highlights Sanofi’s plan to become a leading company in the rare disease drug space, one of the hottest sectors in biopharma. 

Sanofi picked up the then late-stage drug — buying Ablynx for $4.8 billion — when it was already close to the goal line, the lead program for the pipeline of nanobodies that Sanofi hopes to steer to more approvals.

This disease is ultra rare and deadly. Patients suffer blood clots in the small arteries that can lead to strokes, brain damage and death. The drug works by tackling the binding process of von Willebrand factor and platelets.

“Patients with aTTP endure hours of treatment with daily plasma exchange, which requires being attached to a machine that takes blood out of the body and mixes it with donated plasma and then returns it to the body. Even after days or weeks of this treatment, as well as taking drugs that suppress the immune system, many patients will have a recurrence of aTTP,” said Richard Pazdur, acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “Cablivi is the first targeted treatment that inhibits the formation of blood clots. It provides a new treatment option for patients that may reduce recurrences.


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