Ablynx’ caplacizumab receives fast track designation from FDA

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The fast track designation would allow caplacuzimab to enter the market 6 to 10 months earlier

Ablynx today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for caplacizumab, the Company’s first-in-class anti-von Willebrand factor (vWF) Nanobody® being developed for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP).

The FDA’s Fast Track programme is designed to facilitate the development and expedite the review of drugs that treat serious conditions and fill an unmet medical need. Drugs that receive this designation are eligible for more frequent interactions with the FDA, priority review and rolling review of the Biologics License Application (BLA). The purpose of this FDA programme is to get important new drugs to the patient earlier. aTTP is a life-threatening, autoimmune blood clotting disorder manifested by extensive clot formation in small blood vessels throughout the body, leading to thrombocytopenia, ischemia and widespread organ damage especially in the brain and heart. Currently no products have been authorised for the treatment of aTTP and despite standard-of-care treatment with plasma exchange (PEX) and immunosuppressive therapy, patients remain at risk for thrombotic complications, recurrences and death. The potential of caplacizumab to address this unmet need has been demonstrated in the Phase II TITAN study which supports the Marketing Authorisation Application (MAA) submitted to the European Medicines Agency (EMA) in February 2017. Caplacizumab is currently being further evaluated in the randomised, double-blind, placebo-controlled Phase III HERCULES study. Results from this Phase III study will be reported in the second half of 2017 and are expected to further support the MAA, as well as a planned BLA filing in the United States in 2018. Dr Robert K. Zeldin, Chief Medical Officer at Ablynx, commented: “The designation of Fast Track status by the FDA is recognition of the high unmet medical need in patients with aTTP and the potential for caplacizumab to improve outcomes in this very severe disease. We look forward to continuing to work with the FDA and accelerating the development of caplacizumab as potentially the first therapeutic specifically indicated for the treatment of aTTP.”