Armed with promising new data from a pre-planned interim analysis of a pivotal phase 3 study, Japan’s largest drugmaker Takeda says it aims to seek marketing authorisation for its TAK-755 enzyme replacement therapy.
TAK-755 is the first and only recombinant ADAMTS13 protein in development and is designed to treat congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood clotting disorder with a mortality rate of >90%, if left untreated. This condition also has significant unmet need, with the current standard of care being inefficient at restoring ADAMTS13, time consuming and costly.
‘The results of the trail are very encouraging’ says Daniel Curran, M.D., Head, Rare Genetics & Haematology Therapeutic Area Unit at Takeda, with data showing that enzyme replacement therapy TAK-755 reduced the incidence of thrombocytopenic events by 60%, when compared to the current standard of care. Meaning that Takeda plans to engage with global regulatory bodies with the aim of bringing TAK-755 to patients as quickly as possible, whilst also planning to submit the results of this interim analysis for presentation at an upcoming scientific event.