Medication Monitor

Generic Name (Trade Name—Company)
February 7, 2019


FDA approves first drug therapy for adults with rare blood clotting disorder

FDA approved caplacizumab-yhdp injection, the first therapy specifically indicated, in combination with plasma exchange and immunosuppressive therapy, for treatment of adult patients with acquired thrombotic thrombocytopenic purpura (aTTP), a rare and life-threatening disorder that causes blood clotting.

Patients with aTTP develop extensive blood clots in the small blood vessels throughout the body. These clots can cut off oxygen and blood supply to the major organs and cause strokes and heart attacks that may lead to brain damage or death. Patients can develop aTTP because of conditions such as cancer, HIV, pregnancy, lupus or infections or after having surgery, bone marrow transplantation, or chemotherapy.

Common adverse effects reported by patients in clinical trials were bleeding of the nose or gums and headache. The prescribing information for caplacizumab-yhdp includes a warning to advise health care providers and patients about the risk of severe bleeding.

Health care providers are advised to monitor patients closely for bleeding when administering the drug to patients who currently take anticoagulants.