Adzynma FDA Approval History
Last updated by Judith Stewart, BPharm on Nov 16, 2023.
FDA Approved: Yes (First approved November 9, 2023)
Brand name: Adzynma
Generic name: ADAMTS13, recombinant-krhn
Dosage form: Lyophilized Powder for Injection
Company: Takeda Pharmaceutical Company Limited
Treatment for: Thrombotic Thrombocytopenic Purpura
Adzynma (ADAMTS13, recombinant-krhn) is a human recombinant “A disintegrin and metalloproteinase with thrombospondin motifs 13” (rADAMTS13) indicated for prophylactic or on demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura.
- Congenital thrombotic thrombocytopenic purpura (cTTP) is an ultra-rare, chronic blood clotting disorder associated with acute events and debilitating chronic symptoms or thrombotic thrombocytopenic purpura (TTP) manifestations, which can include thrombocytopenia, microangiopathic hemolytic anemia, headache and abdominal pain.
- cTTP is caused by a deficiency in the ADAMTS13 enzyme. ADAMTS13 is a von Willebrand factor (VWF) cleaving protease, and a deficiency results in the accumulation of ultra-large VWF multimers in the blood causing uncontrolled platelet aggregation and adhesion
- Adzynma is a recombinant form of the ADAMTS13 protein that works in the treatment of cTTP by replacing the deficient ADAMTS13 enzyme.
- The FDA approval of Adzynma was supported by the totality of the evidence provided by data from the Phase 3 trial in cTTP as well as data from the continuation trial. In the Phase 3 trial, patients received Adzynma or plasma-based therapy for months 1-6 (period 1), crossing over to the alternate treatment for months 7-12 (period 2), and all patients received Adzynma for months 13-18 (period 3). No patient experienced an acute TTP event while receiving Adzynma, and no subacute TTP events were reported in patients receiving Adzynma during comparison periods 1 and 2, compared to five subacute TTP events in four patients receiving plasma-based therapies. In the continuation period (period 3), two patients receiving Adzynma experienced two subacute events.
- Adzynma is administered by intravenous infusion.
- Warnings and precautions associated with Adzynma include hypersensitivity reactions and the potential to develop antibodies which could potentially result in a decreased or lack of response to rADAMTS13.
- Common adverse reactions include headache, diarrhea, migraine, abdominal pain, nausea, upper respiratory tract infection, dizziness, and vomiting.
Development timeline for Adzynma
| Date | Article |
|---|---|
| Nov 9, 2023 | Approval FDA Approves Adzynma (ADAMTS13, recombinant-krhn) Enzyme Replacement Therapy for the Treatment of Congenital Thrombotic Thrombocytopenic Purpura (cTTP) |
Further information
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