First therapy for very rare blood clotting disorder gets US greenlight

Blood cells

Takeda’s Adzynma has become the first recombinant protein product approved in the US for prophylactic or on-demand enzyme replacement therapy (ERT) for congenital thrombotic thrombocytopenic purpura (cTTP).

A very rare, inherited blood clotting disorder, cTTP is caused by a disease-causing mutation in the ADAMTS13 gene, which is responsible for making an enzyme, also named ADAMTS13, that regulates blood clotting. A deficiency in this enzyme causes blood clots to form in the small blood vessels throughout the body.

Adzynma is a purified recombinant form of the ADAMTS13 enzyme that works by providing a replacement for the low levels of the deficient enzyme in patients with cTTP. For prophylactic ERT, Adyznma is administered to help reduce the risk of disease symptoms.

“The FDA remains deeply committed in our efforts to help facilitate the development and approval of safe and effective therapies for patients with rare diseases,” said Peter Marks, Director of the FDA’s Center for Biologics Evaluation and Research. “Without treatment, cTTP is ultimately fatal. Today’s approval reflects important progress in the development of much-needed treatment options for patients affected by this life-threatening disorder.”

New possibilities for cTTP patients

The safety and effectiveness of Adzynma were demonstrated in a global study evaluating prophylactic and on-demand ERT with Adzynma compared to plasma-based therapies in patients with cTTP.

“In recent decades, significant progress has been made to better understand the link between ADAMTS13 deficiency and cTTP, ultimately leading to this moment where we finally have an FDA-approved treatment option for patients living with this rare disease,” said Spero Cataland, Professor of Internal Medicine at the Wexner Medical Center at The Ohio State University, co-director at the US Thrombotic Microangiopathy Alliance (USTMA) and Adzynma clinical trial investigator.

“Adzynma provides patients with a treatment option that replaces their deficient ADAMTS13 enzyme and offers a favourable efficacy and safety profile and reduced administration time and volume compared to current plasma-based therapies. Today marks a significant achievement, providing new possibilities for the cTTP patient community.”

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