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Acquired TTP Drug Wins Approval

WASHINGTON — Caplacizumab (Cablivi), an antibody drug targeting von Willebrand factor, is now approved for acquired thrombotic thrombocytopenic purpura (aTTP), the FDA said Wednesday.

The drug is to be given in combination with plasma exchange and immunosuppressive treatment, the agency said, and is the first product approved specifically for aTTP. Made by Sanofi’s Ablynx unit, caplacizumab was approved in Europe last September.

Acquired TTP develops as a consequence of other conditions including HIV infection, pregnancy, systemic lupus erythematosus, infections, or malignancies. It can also be secondary to surgery, bone marrow transplantation, or cancer chemotherapy, the FDA explained. The condition is marked by severe platelet deficiency and thrombotic events.

Approval was based primarily on a 145-patient randomized trial, in which patients receiving caplacizumab showed significantly faster recovery in platelet counts than those assigned to placebo. The drug was also associated with fewer aTTP-related deaths and treatment-emergent thrombotic events. Recurrence of aTTP during treatment plus a 4-week follow-up period was 13% with caplacizumab versus 38% with placebo, the FDA said.

“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, MD, acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, in a statement announcing the approval. “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.”

Common adverse effects seen with the drug include headache and bleeding from the nose or gums.