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cTTP ERT Receives FDA Approval

January 17, 2024

February 2024

Adzynma, also known as ADAMTS13, recombinant-​krhn, received U.S. Food and Drug Administration (FDA) approval for the treatment of congenital thrombotic thrombocytopenic purpura (cTTP) in adult and pediatric patients. Adzynma is the first recombinant protein indicated for prophylactic and on-demand treatment of 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 R. Cataland, MD, professor of internal medicine at the Wexner Medical Center at The Ohio State University, co-director at the U.S. Thrombotic Microangiopathy Alliance, and clinical trial investigator for the treatment.

The treatment is a purified recombinant form of the ADAMTS13 enzyme. It works by replacing low levels of the enzyme in patients with cTTP, increasing ADAMTS13 activity by four to five times. For prophylactic purposes, the treatment is administered once every other week. It can also be administered once daily as on-demand enzyme replacement therapy (ERT) in individuals experiencing an acute event.

Approval was granted based on the results of a randomized, controlled, open-label, crossover phase III trial evaluating the safety, tolerability, pharmacokinetics, and efficacy of Adzynma. Results also found that common side effects included abdominal pain, nausea, dizziness, vomiting, migraine, headache, diarrhea, and upper respiratory tract infection.

Sources: FDA, November 9, 2023. Takeda, November 9, 2023.

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