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# Takeda’s Adzynma Approved by FDA
- URL: https://www.fdaweb.com/takedas-adzynma-approved-by-fda/
- Published: 2023-11-09T12:00:00.000Z
- Updated: 2026-09-14T14:15:44.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5155762

FDA has approved Takeda’s Adzynma, a recombinant protein product designed for prophylactic or on‑demand enzyme replacement therapy (ERT) in both adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP) — a rare life-threatening blood clotting disorder. cTTP is linked to a disease-causing mutation in the ADAMTS13 gene, responsible for producing the ADAMTS13 enzyme that regulates blood clotting, according to an FDA [news release](https://lnks.gd/l/eyJhbGciOiJIUzI1NiJ9.eyJidWxsZXRpbl9saW5rX2lkIjoxMDAsInVyaSI6ImJwMjpjbGljayIsInVybCI6Imh0dHBzOi8vY29udGVudC5nb3ZkZWxpdmVyeS5jb20vYWNjb3VudHMvVVNGREEvYnVsbGV0aW5zLzM3YTQ3YWIiLCJidWxsZXRpbl9pZCI6IjIwMjMxMTA5Ljg1NDI5NTkxIn0.ufj4pOAPP8E43FUG-EyZ7f6dPnKu3P4Am2Jztz6CMwc/s/1255931999/br/230465529539-l?ref=fdaweb.com). Enzyme deficiency triggers the formation of blood clots in small vessels across the body, and if left untreated, it can lead to severe bleeding episodes, strokes, and damage to vital organs.

Adzynma’s approval was based on data in 46 patients who were randomized to receive six months of treatment with either Adzynma or plasma‑based therapies (Period 1), then crossed over to the other treatment for six months (Period 2), FDA says. “The efficacy was demonstrated based on the incidence of thrombotic thrombocytopenic purpura (TTP) events, and TTP manifestations, as well as the incidence of the need for supplemental doses,” it says. “The efficacy of on‑demand ERT was evaluated based on the proportion of acute TTP events responding to Adzynma in both the prophylactic and the on-demand cohorts throughout the duration of the study. All acute and subacute TTP events resolved after treatment with either Adzynma or plasma‑based therapies.”

Common side effects associated with Adzynma include headache, diarrhea, migraine, abdominal pain, nausea, upper respiratory tract infection, dizziness, and vomiting, the agency says.