Rare Daily Staff
The European Commission has granted marketing authorization to Sanofi’s Cablivi for the treatment of adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), a rare blood-clotting disorder.
Cablivi is the first therapeutic specifically indicated for the treatment of aTTP, a life-threatening, autoimmune disorder characterized by extensive clot formation in small blood vessels throughout the body. The condition leads to severe low platelet count, loss of red blood cells through destruction, restricted blood supply to parts of the body, and widespread organ damage especially in the brain and heart.
Despite the current standard-of-care treatment, consisting of daily plasma exchange and immunosuppression, episodes of aTTP are still associated with a mortality rate of up to 20 percent, with most deaths occurring within 30 days of diagnosis.
Cablivi is the company’s first Nanobody-based medicine to receive approval and the first newly approved product that will be part of Sanofi Genzyme’s Rare Blood Disorders franchise. Ablynx, which Sanofi acquired earlier this year, developed Cablivi.
“The approval of Cablivi provides new hope for people diagnosed with aTTP, who to date have faced a very difficult disease with limited treatment options,” said Bill Sibold, executive vice president and head of Sanofi Genzyme. “This approval is the next step towards our goal of becoming the leading rare blood disorders company in the industry. We are excited about the opportunities to continue to expand our rare blood disorders business and to help many people with very serious diseases.”
The approval of Cablivi in the European Union is based on the phase 2 TITAN and phase 3 HERCULES studies in 220 adult patients with aTTP. The efficacy and safety of Cablivi in addition to standard-of-care treatment, daily PEX and immunosuppression, were demonstrated in these studies.
In the HERCULES study, treatment with Cablivi in addition to standard-of-care resulted in a significantly shorter time to platelet count response, the study’s primary endpoint; a significant reduction in aTTP-related death, recurrence of aTTP, or at least one major thromboembolic event during study drug treatment; and a significantly lower number of aTTP recurrences in the overall study period. Treatment with Cablivi resulted in a clinically meaningful reduction in the use of plasma exchange and length of stay in the intensive care unit and the hospital, compared to the placebo group.
In clinical trials, Cablivi demonstrated a safety profile, consistent with its mechanism of action. The most frequently reported adverse reactions were epistaxis, headache and gingival bleeding. No deaths were reported during study drug treatment in the Cablivi group in the TITAN and HERCULES studies, while for the placebo group, two deaths were reported in the TITAN study and three deaths in the HERCULES study.
The U.S. Food and Drug Administration has accepted for priority review the Biologics License Application for Cablivi for treatment of patients 18 years of age and older experiencing an episode of aTTP. The FDA is expected to issue a decision by February 6, 2019.
September 5, 2018
Photo: Bill Sibold, executive vice president and head of Sanofi Genzyme