The US Food and Drug Administration (FDA) has approved Cablivi (caplacizumab-yhdp) as the first treatment for adult patients with a rare blood clotting disorder, acquired thrombotic thrombocytopenic purpura (aTTP).

The decision comes based on the efficacy of Cablivi, which was studied in a clinical trial of 145 patients in which the results of the trial demonstrated that platelet counts improved faster among patients treated with Cablivi, compared to placebo.

The drug received FDA Fast Track designation and was evaluated under Priority Review, which is reserved for medicines that represent significant improvements in safety or efficacy in treating serious conditions.

Treatment with Cablivi also resulted in a lower total number of patients with either aTTP-related death and recurrence of aTTP during the treatment period, or at least one treatment-emergent major thrombotic event (where blood clots form inside a blood vessel and may then break free to travel throughout the body).

“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, director of the FDA’s Oncology Centre of Excellence.

“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.”

Acquired thrombotic thrombocytopenic purpura is a rare and life-threatening disorder that causes blood clotting, where patients develop extensive blood clots in the small blood vessels throughout the body.