Apadamtase Alfa: First Approval

• Published in: Drugs (New York, N.Y.) Vol. 84; no. 4; pp. 467 - 472
• Main Author: Heo, Young-A.
• Format: Journal Article
• Language: English
• Published: Cham Springer International Publishing 01.04.2024; Springer Nature B.V
• Subjects: ADAM protein; ADAMTS13 Protein - metabolism; AdisInsight Report; Agreements; Anemia; Anemia, Sickle Cell - drug therapy; Antigens; Congenital diseases; Disease prevention; Drug Approval; Enzyme Replacement Therapy; Enzymes; Humans; Hypoxia; Intellectual property; Internal Medicine; Medicine; Medicine & Public Health; Mortality; Pediatrics; Pharmacokinetics; Pharmacology/Toxicology; Pharmacotherapy; Pharmacy; Plasma; Purpura; Purpura, Thrombotic Thrombocytopenic - drug therapy; Recombinant Proteins - therapeutic use; Sickle cell anemia; Sickle cell disease; Thrombocytopenia; Thrombocytopenic purpura; Thrombospondin; Thrombotic thrombocytopenic purpura; United States; United States; United States > US; Japan
• ISSN: 0012-6667; 1179-1950
• DOI: 10.1007/s40265-024-02007-6

Apadamtase alfa (ADAMTS13, recombinant-krhn; ADZYNMA), a human recombinant form of a disintegrin and metalloproteinase with thrombospondin motifs 13 (ADAMTS13), is being developed by Takeda under license from KM biologics for thrombotic thrombocytopenic purpura (TTP) and sickle cell disease. On 9 November 2023, apadamtase alfa was approved in the USA for prophylactic and on-demand enzyme replacement therapy (ERT) in paediatric and adult patients with congenital TTP. Apadamtase alfa is under regulatory review for congenital TTP in the EU and Japan, and is under clinical development for immune-mediated TTP in several countries worldwide. Clinical development of apadamtase alfa for vaso-occlusive crisis related to sickle cell anaemia is underway in the USA. This article summarizes the milestones in the development of apadamtase alfa leading to this first approval in the USA for congenital TTP.