Preferred Label : Apadamtase Alfa;
NCIt synonyms : Recombinant a Disintegrin and Metalloproteinase with a Thrombospondin Type 1 Motif
Member 13; Recombinant Human ADAMTS13; Recombinant ADAMTS13; Recombinant a Disintegrin and Metalloproteinase with Thrombospondin Motifs 13; Recombinant Human ADAMTS-13; rADAMTS13;
NCIt definition : A recombinant human form of a disintegrin and metalloproteinase with thrombospondin
motifs 13 (ADAMTS13) that may be used as a replacement therapy for ADAMTS13 with indirect
anti-thrombotic and anti-thrombocytopenic activities. Upon administration, apadamtase
alfa acts similarly as endogenous ADAMTS13 and mediates the cleavage of von Willebrand
factor (VWF) at the Tyr1605-Met1606 bond in the A2 domain. The cleavage of ultralarge
(UL) VWF multimers into smaller forms helps regulate the size of VWF multimers, preventing
the binding of platelets to UL VWF and the resulting thrombotic thrombocytopenic purpura
(TTP). ADAMTS13 plays a key role in the proteolysis of VWF multimers. VWF is involved
in platelet adhesion and blood coagulation.;
UNII : U5SFU33XUX;
CAS number : 2086325-24-6;
Molecule name : BAX-930; TAK 755; BAX 930; TAK-755; SHP-655; SHP 655;
NCI Metathesaurus CUI : CL1382136;
Origin ID : C169784;
UMLS CUI : C5139849;
Currated CISMeF NLP mapping
- See also inter- (CISMeF)
- Semantic type(s)
- concept_is_in_subset
https://ansm.sante.fr/tableau-acces-derogatoire/tak-755
2025
false
false
false
France
French
Apadamtase Alfa
congenital thrombotic thrombocytopenic purpura
enzyme replacement therapy
ADAMTS13 Protein
purpura, thrombotic thrombocytopenic
child
adolescent
adult
drug information
technical report
---
https://www.has-sante.fr/jcms/p_3586231/fr/adzynma-adamts13r-purpura-thrombotique-thrombocytopenique-congenital-pttc
2025
false
false
false
France
ADAMTS13 Protein
Apadamtase Alfa
enzyme replacement therapy
congenital thrombotic thrombocytopenic purpura
adult
child
treatment outcome
insurance, health, reimbursement
orphan drug production
injections, intravenous
Congenital thrombotic thrombocytopenic purpura
evaluation of the transparency committee
purpura, thrombotic thrombocytopenic
---
https://www.has-sante.fr/jcms/p_3576108/fr/adzynma-apadamtase-alfa-purpura-thrombotique-thrombocytopenique-congenital-pttc-pediatrie
2025
false
false
false
France
child
treatment outcome
insurance, health, reimbursement
Apadamtase Alfa
guidelines for drug use
apadamtase alfa and cinaxadamtase alfa
Cinaxadamtase Alfa
enzyme replacement therapy
ADAMTS13 protein, human
injections, intravenous
evaluation of the transparency committee
purpura, thrombotic thrombocytopenic
congenital thrombotic thrombocytopenic purpura
---
https://www.has-sante.fr/jcms/p_3495284/fr/tak-755-apadamtase-alfa-purpura-thrombotique-thrombocytopenique-congenital-pttc
2024
false
false
false
France
enzyme replacement therapy
congenital thrombotic thrombocytopenic purpura
adolescent
adult
enzymes
evaluation of the transparency committee
purpura, thrombotic thrombocytopenic
Apadamtase Alfa
---
