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FDA grants fast track designation to caplacizumab for rare blood disorder
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The FDA granted fast track designation to caplacizumab for the treatment of acquired thrombotic thrombocytopenic purpura, according to the drug’s manufacturer.
Caplacizumab (Ablynx), a first-in-class anti-von Willebrand factor nanobody, blocks the interaction of ultra-large von Willebrand factor multimers with platelets, which results in the formation and accumulation of micro-clots. These clots cause severe thrombocytopenia, tissue ischemia and organ dysfunction in acquired thrombotic thrombocytopenic purpura, a rare and life-threatening blood-clotting disorder.
The phase 2 TITAN study evaluated the efficacy and safety of caplacizumab in combination with plasma exchange in 75 patients with acquired thrombotic thrombocytopenic purpura.
Patients treated with caplacizumab demonstrated a 39% reduction in time to platelet count normalization (P = .005), and a 71% reduction in thrombotic thrombocytopenic purpura recurrence compared with placebo.
Post-hoc analyses showed a clinically meaningful lower proportion of patients in the treatment arm experienced one or more major thromboembolic events or died (11.4% vs. 43.2%), and fewer patients were refractory to treatment (5.7% vs. 21.6%).
The randomized, double-blind, placebo-controlled phase 3 HERCULES study, designed to evaluate the efficacy and safety of caplacizumab when administered in addition to the standard-of-care, is ongoing.
“The designation of fast track status by the FDA is recognition of the high unmet medical need in patients with acquired thrombotic thrombocytopenic purpura and the potential for caplacizumab to improve outcomes in this very severe disease,” Robert K. Zeldin, MD, chief medical officer at Ablynx, said in a company-issued release.
“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 acquired thrombotic thrombocytopenic purpura.”