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European Commission authorises first-ever rare blood condition drug

Published on 04/09/18 at 11:02am

Sanofi have announced that the European Commission granted marketing authorisation for Cablivi (caplacizumab) for the treatment of adults experiencing an episode of acquired thrombotic thrombocytopaenic purpura (aTTP). The approval marks the first therapeutic specifically indicated for the disease.

“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,” stated Bill Sibold, who heads Sanofi Genzyme.

The French pharma giant Sanofi acquired the rights for the drug through the acquisition of Ablynx for €3.9 billion, earlier this year.

The authorisation was based on the Phase II TITAN trial and the Phase III HERCULES trial which demonstrated that cablivi was associated with a 74% reduction in the percentage of patients experiencing an aTTP related death.

According to Sanofi there are approximately 7,500 patients with aTTP symptoms in the United States, the European Union and Japan. The FDA, which has granted a fast track designation for the treatment of aTTPis now expected to come to a decisionby February 6th 2019.

Louis Goss

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