FDA approves Takeda’s new enzyme replacement therapy for rare blood clotting disorder

Christophe Weber, Takeda CEO Shoko Takayasu
November 9, 2023
FDA approves Takeda’s new enzyme replacement therapy for rare blood clotting disorder
Zachary Brennan
Senior Editor
The FDA on Thursday approved Takeda’s Adzynma, an enzyme replacement therapy for adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP), which is a rare and potentially fatal blood clotting disorder.

The approval was based on data announced by Takeda in January from an interim Phase III analysis that found the therapy reduced instances of low blood platelet count for patients with cTTP compared to standard-of-care plasma-based therapies.

The FDA said the efficacy data included 46 patients randomized to receive six months of either Adzynma or plasma-based therapies, then crossed over to the other treatment for six months. Efficacy was demonstrated based on the incidence of TTP events and TTP manifestations, as well as the need for supplemental doses.

The most common side effects, according to FDA, include headache, diarrhea, migraine, abdominal pain, nausea, upper respiratory tract infection, dizziness and vomiting.

The therapy came to Takeda by way of Shire, which bought it from Baxalta. Takeda’s rare genetics and hematology lead Dan Curran previously told Endpoints News that cTTP affects about 300 to 400 people in the US.

Because Adzynma can help treat a rare pediatric disease, the approval comes with a priority review voucher for Takeda. The voucher is worth about $100 million on the open market.

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Zachary Brennan
Senior Editor
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