FDA approves first drug for rare inherited blood clotting disorder

The US Food and Drug Administration yesterday approved Japanese pharma major Takeda’s (TYO: 4502) Adzynma (ADAMTS13, recombinant-krhn; TAK-755), adding to the approval a day earlier of the firm’s bowel cancer drug Fruzaqla (fruquintinib).

Takeda says that Adzynma is the first recombinant (genetically engineered) protein product indicated for prophylactic (preventive) or on demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP), a rare and life-threatening blood clotting disorder.

Although the target population is small, the drug is administered chronically via once-weekly intravenous injections for prophylactic use, and once-daily for on-demand treatment.