Takeda scores second FDA approval this week with green light for rare blood clotting disorder drug Adzynma

Fierce Pharma November 9, 2023
Fraiser Kansteiner

Thanks to a Takeda approval Thursday, patients now have a drug to tackle a rare inherited blood clotting disorder.

The U.S. FDA has given a green light to Takeda’s Adzynma, the first recombinant protein product as a preventative or on demand enzyme replacement therapy in adults and kids with congenital thrombotic thrombocytopenic purpura (cTTP).

According to the FDA, cTTP is a very rare inherited blood clotting disorder caused by a disease-causing mutation in the ADAMTS13 gene, which makes an enzyme responsible for regulating blood clotting. Patients with a deficiency in this enzyme create blood clots in the small blood vessels throughout the body. The disease is estimated to affect fewer than 1,000 people in the U.S.

While symptoms of cTTP...

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