The FDA has approved caplacizumab-yhdp injection (Cablivi, Ablynx) for the treatment of adults with acquired thrombotic thrombocytopenic purpura (aTTP), a rare, life-threatening disorder that causes blood clotting.
aTTP causes extensive blood clots throughout the body. The clots can cut off oxygen and blood supply to major organs and cause stroke and heart attacks that can lead to brain damage or death. Patients can develop the condition through cancer, HIV, pregnancy, lupus, or infections, or after having surgery, bone marrow transplantation, or chemotherapy.
The new drug is the first therapy specifically indicated for the disorder, in combination with plasma exchange and immunosuppressive therapy. Patients with aTTP undergo hours of treatment with daily plasma exchange, and many have a recurrence of aTTP, even after weeks of treatment. Caplacizumab-yhdp offers patients a new option that may reduce recurrences.
In a clinical trial of 145 patients, who were randomized to receive either caplacizumab-yhdp or placebo, platelet counts improved faster in the caplacizumab-yhdp group compared to the placebo group. Treatment with caplacizumab-yhdp also resulted in fewer patients with either aTTP-related death and recurrence of aTTP during the treatment period, or at least one treatment-emergent major thrombotic event.
Common side effects from caplacizumab-yhdp were bleeding of the nose or gums and headache. The drug’s prescribing information carries a warning about the risk of severe bleeding.
The FDA granted caplacizumab-yhdp priority review and orphan drug designations.
Source: FDA, February 6, 2019