pharmaphorum November 10, 2023
Patients in the US with ultra-rare blood clotting disorder congenital thrombotic thrombocytopenic purpura (cTTP) have an FDA-approved therapy for the first time, after Takeda got the green light for its Adzynma drug.
cTTP is a subtype of thrombotic thrombocytopenic purpura (TTP), a rare and life-threatening disorder in which blood clots form in the small blood vessels throughout the body. It is caused by a deficiency in the ADAMTS13 protease enzyme.
Symptoms typically develop in infancy or early childhood, but in some cases may develop in adulthood and may first manifest during pregnancy. Patients with cTTP can experience severe bleeding episodes, strokes, and damage to vital organs, which can be fatal if untreated.
Adzynma (formerly TAK-755) is a recombinant form of...
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