Apadamtase alfa
| Clinical data | |
|---|---|
| Trade names | Adzynma |
| Other names | ADAMTS13, recombinant-krhn; rADAMTS13 |
| AHFS/Drugs.com | Monograph |
| License data | |
| Routes of administration | Intravenous |
| ATC code |
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| Legal status | |
| Legal status | |
| Identifiers | |
| CAS Number | |
| DrugBank | |
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Apadamtase alfa, sold under the brand name Adzynma, is an enzyme replacement therapy used for the treatment of thrombotic thrombocytopenic purpura.[1] Apadamtase alfa is a human recombinant a disintegrin and metalloproteinase with thrombospondin motifs 13.[1] It is given by injection into a vein.[1]
The most common side effects include headache, diarrhea, migraine, abdominal pain, nausea, upper respiratory tract infection, dizziness and vomiting. During the clinical studies, no adverse events, including allergic reactions, were observed during the administration of Adzynma.[5]
Apadamtase alfa was approved for medical use in the United States in November 2023,[5][6] and in the European Union in August 2024.[3]
Medical uses
[edit]Apadamtase alfa is indicated for prophylactic (preventive) or on demand enzyme replacement therapy in people with congenital thrombotic thrombocytopenic purpura.[1][5]
History
[edit]The efficacy of apadamtase alfa in the prophylactic treatment of participants with congenital thrombotic thrombocytopenic purpura was evaluated in 46 participants who were randomized to receive six months of treatment with either apadamtase alfa or plasma based therapies (Period 1), then crossed over to the other treatment for six months (Period 2).[5] The efficacy was demonstrated based on the incidence of thrombotic thrombocytopenic purpura events, and thrombotic thrombocytopenic purpura manifestations, as well as the incidence of the need for supplemental doses.[5]
The efficacy of on demand enzyme replacement therapy was evaluated based on the proportion of acute thrombotic thrombocytopenic purpura events responding to apadamtase alfa in both the prophylactic and the on-demand cohorts throughout the duration of the study.[5] All acute and subacute thrombotic thrombocytopenic purpura events resolved after treatment with either apadamtase alfa or plasma based therapies.[5]
The US Food and Drug Administration (FDA) granted the application for apadamtase alfa priority review, fast track, and orphan drug designations.[5] The FDA granted approval of Adzynma to Takeda Pharmaceuticals U.S.A. Inc.[5]
Society and culture
[edit]Legal status
[edit]In May 2024, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization under exceptional circumstances for the medicinal product Adzynma, indicated for the treatment of ADAMTS13 deficiency in people with congenital thrombotic thrombocytopenic purpura (cTTP).[3][7] The applicant for this medicinal product is Takeda Manufacturing Austria AG.[3] The active substance of Adzynma is recombinant ADAMTS13 (rADAMTS13).[3] Apadamtase alfa was approved for medical use in the European Union in August 2024.[3][4]
References
[edit]- ^ a b c d e "Adzynma- apadamtase alfa kit". DailyMed. 9 November 2023. Retrieved 30 November 2023.
- ^ "Adzynma". U.S. Food and Drug Administration (FDA). 9 November 2023. Retrieved 30 November 2023.
- ^ a b c d e f "Adzynma EPAR". European Medicines Agency (EMA). 30 May 2024. Retrieved 1 June 2024.
- ^ a b "Adzynma Product information". Union Register of medicinal products. 2 August 2024. Retrieved 27 August 2024.
- ^ a b c d e f g h i "FDA Approves First Treatment for Patients with Rare Inherited Blood Clotting Disorder". U.S. Food and Drug Administration (Press release). 9 November 2023. Retrieved 30 November 2023.
This article incorporates text from this source, which is in the public domain.
- ^ "Takeda's Adzynma (ADAMTS13, recombinant-krhn) Approved by U.S. FDA as the First and Only Recombinant ADAMTS13 Enzyme Replacement Therapy for the Treatment of Congenital Thrombotic Thrombocytopenic Purpura (cTTP)" (Press release). Takeda Pharmaceuticals. 9 November 2023. Retrieved 30 November 2023 – via Business Wire.
- ^ "Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 27-30 May 2024". European Medicines Agency (Press release). 31 May 2024. Retrieved 13 June 2024.
External links
[edit]- "Apadamtase Alfa (Code C169784)". NCI Thesaurus. 30 October 2023.
