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FDA Approved: First Recombinant Therapy for cTTP



Takeda’s ADZYNMA has received FDA approval for the treatment of congenital thrombotic thrombocytopenic purpura (cTTP), making it the

first and only approved recombinant therapy for this rare blood clotting disorder. cTTP is caused by a mutation in the ADAMTS13 gene that results in the deficient production of the ADAMTS13 enzyme, which is responsible for regulating blood clotting. People deficient in this enzyme develop blood clots in the small blood vessels, leading to acute and debilitating chronic symptoms, including severe bleeding episodes, strokes and damage to vital organs. Though cTTP is an “ultra rare” condition affecting less than 1,000 persons in the US, the mortality rate associated with untreated acute events is >90%.


ADZYNMA is a recombinant form of the ADAMTS13 protein that works by replacing the deficient ADAMTS13 enzyme. It is approved for use as a prophylactic and on-demand treatment of cTTP in adult and pediatric patients.


The FDA approval is backed by evidence from a Phase III trial including 46 patients with cTTP who received either ADZYNMA or a plasma-based therapy. In this trial, no acute events were reported in patients receiving ADZYNMA. The mean annualized event rate of thrombocytopenia, an important biomarker of disease activity, was 2.0 in the ADZYNMA arm versus 4.44 in the plasma-based therapy arm. ADZYNMA also demonstrated a favorable safety profile, and no patients receiving ADZYNMA developed neutralizing antibodies.


People living with cTTP are faced with serious, life-threating health challenges. The approval of ADZYNMA addresses an unmet need for people with cTTP, offering a novel treatment indicated specifically for their condition.


Written by Zaraa Malvat

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