Apadamtase Alfa: First Approval

Apadamtase Alfa: First Approval

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 No...

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Veröffentlicht in:Drugs (New York, N.Y.) N.Y.), 2024-04, Vol.84 (4), p.467-472
1. Verfasser: Heo, Young-A.
Format: Artikel
Sprache:eng
Schlagworte:
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
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Beschreibung
Zusammenfassung: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.
ISSN:0012-6667
1179-1950
DOI:10.1007/s40265-024-02007-6