FDA Approves New Option for Rare Blood Disorder

The FDA has approved Cablivi (caplacizumab-yhdp) injection, the first therapy specifically indicated (in combination with plasma exchange and immunosuppressive therapy) for the treatment of adults with acquired thrombotic thrombocytopenic purpura (aTTP).

The efficacy of the drug was examined in a clinical trial involving 145 patients randomly assigned to receive either Cablivi or placebo. Overall, platelet counts improved faster among those treated with Cablivi compared with placebo. Cablivi was also associated with lower total number of patients with either aTTP-related death or recurrence of aTTP during treatment, or at least 1 treatment-emergent major thrombotic event.

Common side effects include bleeding of the nose or gums and headache.

—Michael Potts

Reference:

FDA. FDA approves first therapy for the treatment of adult patients with a rare blood clotting disorder [press release]. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm630851.htm.