Cablivi Approved to Treat Rare Blood-Clotting Disorder
February 6, 2019 – The U.S. FDA has approved Cablivi® (caplacizumab-yhdp), manufactured by Sanofi, for use in combination with plasma exchange to treat adult patients who have acquired thrombotic thrombocytopenic purpura (aTTP).
TTP is a serious, life-threatening condition that occurs in both an acquired form and a familial (inherited) form. The disease causes extensive blood clots in small blood vessels throughout the body. These clots can lead to low red blood cell counts, anemia, and reduced blood flow to vital organs. Potential complications include heart problems, jaundice, abnormal kidney function, abdominal pain, and neurological programs. Currently, TTP is believed to occur in about 3.7 out of every one million individuals per year worldwide. Acquired TTP may occur due to circumstances that include pregnancy, cancer, HIV infection, lupus, chemotherapy, surgery, infection, or bone marrow transplant.
Cablivi helps to inhibit the formation of blood clots by blocking the activity of von Willebrand factor proteins. In clinical studies, Cablivi reduced the incidence of aTTP-related death, aTTP recurrence, or at least one major thromboembolic event by 74% compared to placebo. Patients who received Cablivi had a 38% reduction in use of plasma exchange, a 65% reduction in length of intensive care unit stays, and a 31% reduction in length of hospital stays.
Recommended dosing for Cablivi is 11mg administered via intravenous injection prior to a plasma exchange. Following the plasma exchange, another 11mg should be given by subcutaneous (SC) injection. Daily SC injections of 11mg are continued throughout the plasma exchange period, and for another 30 days thereafter. If needed, treatment can be extended an additional 28 days. Patients and caregivers can administer Cablivi themselves once they have been trained in its proper use.
Sanofi plans to launch Cablivi late in the first quarter of 2019 at a wholesale acquisition cost (WAC) of $270,000 per treatment of a typical aTTP episode.