Hard on the heels of completing its €3.9 billion ($4.5 billion) Ablynx acquisition, Sanofi has won a European recommendation for the first product out of that deal.
On Friday, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended approval of Cablivi (caplacizumab) for the treatment of a rare autoimmune blood-clotting disorder called acquired thrombotic thrombocytopenic purpura (aTTP).
A bivalent anti-vWF nanobody, Cablivi is the first medicine based on Ablynx’s nanobody platform to receive a positive regulatory opinion, and if approved, it will also be the first drug specifically indicated for aTTP. Jefferies analysts have previously predicted it will become the standard of care for aTTP, with peak annual sales of $500 million.
“The benefits with Cablivi are its ability to reduce time to platelet count response, the recurrence rate of the disease, the number of days of plasma exchange, the volume of plasma used, and the length of hospitalization and intensive care unit stay,” the committee wrote in its opinion.
That efficacy profile was proved in the phase 3 HERCULES studies, and a three-year follow-up is currently in progress to further evaluate the drug’s long-term safety and efficacy.
Sanofi now expects a final marketing authorization from the European Commission in the coming months. As for the U.S., the drug is under an FDA “Fast Track” designation for the same indication, and Sanofi plans to file for approval by the end of this year.
Cablivi will fit into Genzyme’s new blood disorder franchise that also includes hemophilia assets gained from Sanofi’s $11.6 billion Bioverativ buyout and renewed agreement with Alnylam. Ablynx also has ALX-0171, a phase 2 nanobody designed to fight respiratory syncytial virus infection in infants.
As part of its refocusing effort championed by CEO Oliver Brandicourt, Sanofi is busy buying new products and is also jettisoning some noncore businesses at the same time. After first announcing its intent to unload the European generics business Zentiva back in 2015, Sanofi has just reached a final, €1.9 billion deal with buyer Advent.