Sanofi files NDA for acquired thrombotic thrombocytopenic purpura in Japan.
Sanofi has submitted a new drug application in Japan for caplacizumab, a nanobody-based agent being developed for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP), a rare blood-clotting disorder.
Patients with aTTP see an excessive accumulation of von Willebrand factor (vWF), a protein in the blood involved in haemostasis, and platelet aggregation due to the decreased activity of ADAMTS13, a specific vWF-cleaving protease.
Caplacizumab targets vWF and is designed to block the interaction between vWF and platelets. In Japan, it has been granted orphan drug designation, which makes it subject to priority review.
The filing is supported by data from a global PIII study and a Japanese PII/III study. In the domestic trial covering Japanese aTTP patients, only one in 15 subjects in the per-protocol (PP) population who received caplacizumab experienced a recurrence, achieving the primary endpoint of the program (the percentage of patients in the PP population who had aTTP recurrences during the study period, with 20% set as the success threshold). The drug also showed a rapid normalization of platelet counts and biomarkers for organ injuries as well as a favourable tolerability profile, with no new safety signals specific to Japanese patients observed.
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