Dive Brief:
- A drug from Sanofi's recent Ablynx acquisition, Cablivi, has gained approval in the EU for episodes of acquired thrombotic thrombocytopenic purpura (aTTP) in adults. The approval hinged on data from the Phase 2 TITAN and Phase 3 Hercules clinical trials.
- Genzyme, Sanofi's specialty care global business unit, "will work with relevant local authorities to make Cablivi available to patients in need in countries across Europe," the company said in a statement.
- Cablivi has been accepted for priority review in the U.S. with a target action date of Feb. 6, 2019.
Dive Insight:
Sanofi picked up Belgian drugmaker Ablynx earlier this year for $4.8 billion, beating its competitor Novo Nordisk to the deal. The key asset in the acquisition was lead drug Cablivi (caplacizumab), which slots neatly in Sanofi's plans to build up its rare blood disorders business.
In that same week, Sanofi also picked up Biogen spin-out Bioverativ in an $11.6 billion deal, adding two longer-acting marketed drugs for hemophilia, Eloctate (antihemophilic Factor VIII, Fc fusion protein) and Aprolix (coagulation Factor IX, Fc fusion protein), as well as a portfolio of earlier-stage drugs for hemophilia, sickle cell disease and beta thalassemia.
Cablivi is a bivalent anti-vWF nanobody that has orphan drug designations in Europe, the U.S., Switzerland and Japan for aTTP, a life-threatening, autoimmune blood clotting disorder.
In aTTP, clots form in small blood vessels throughout the body, leading to very low platelet count, destruction of red blood cells, ischemia and organ damage, especially in the brain and heart.
Patients with aTTP are currently treated with daily plasma exchange and immunosuppressant drugs, but episodes still occur. These are linked with a 20% mortality rate, and most deaths happen within a month of diagnosis.
"The approval of Cablivi provides an important addition to the standard-of-care treatment for patients with aTTP in Europe because it can significantly reduce time to platelet count normalization and induce a clinically meaningful reduction in recurrences," said Marie Scully, a hematology professor at University College London Hospitals, U.K., in a Sept. 3 statement from Sanofi.
The move toward other specialist areas comes as Sanofi deals with a battering in the diabetes market. The French pharma's diabetes drugs sales are falling in both the U.S. and Europe, with year-over-year declines reported in the second quarter.