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| Stem definition | Drug id | CAS RN |
|---|---|---|
| enzymes | 5851 | None |
| Molecule | Description |
|---|---|
|
Synonyms:
|
Adzynma is approved for the treatment of ADAMTS13 deficiency in adults and children with congenital thrombotic thrombocytopenic purpura (cTTP). Adzynma contains the active ingredient apadamtase alfa/cinaxadamtase alfa. rADAMTS13, the active substance, is a recombinant form of the endogenous ADAMTS13. ADAMTS13 is a plasma zinc metalloprotease that regulates the activity of von Willebrand factor (VWF) by cleaving large and ultra-large VWF multimers to smaller units and thereby reducing the platelet binding properties of VWF and its propensity to form microthrombi. rADAMTS13 is expected to reduce or eliminate the spontaneous formation of VWF-platelet microthrombi that leads to platelet consumption and thrombocytopenia in patients with cTTP.
|
| Dose | Unit | Route |
|---|---|---|
| 200 | U | P |
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| Date | Agency | Company | Orphan |
|---|---|---|---|
| Aug. 1, 2024 | EMA | Takeda Manufacturing Austria AG |
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| Source | Code | Description |
|---|---|---|
| ATC | B01AD13 | BLOOD AND BLOOD FORMING ORGANS ANTITHROMBOTIC AGENTS ANTITHROMBOTIC AGENTS Enzymes |
| CHEBI has role | CHEBI:75835 | anti-anaemic agent |
| Disease | Relation | SNOMED_ID | DOID |
|---|---|---|---|
| Thrombotic thrombocytopenic purpura | indication | 78129009 | DOID:10772 |
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| Target | Class | Pharos | UniProt | Action | Type | Activity value (-log[M]) | Mechanism action | Bioact source | MoA source |
|---|---|---|---|---|---|---|---|---|---|
| von Willebrand factor | Secreted | OTHER | UNKNOWN | DRUG LABEL |
| ID | Source |
|---|---|
| DB15164 | DRUGBANK_ID |
| C5669630 | UMLSCUI |
| CHEMBL5095472 | ChEMBL_ID |
| CHEMBL4594607 | ChEMBL_ID |
| 2406308-29-8 | SECONDARY_CAS_RN |
| 2086325-24-6 | SECONDARY_CAS_RN |
| 11803 | INN_ID |
| 10765 | INN_ID |
| 2670285 | RXNORM |
| 1366579000 | SNOMEDCT_US |
| 1380115007 | SNOMEDCT_US |
| 1380117004 | SNOMEDCT_US |
| D12570 | KEGG_DRUG |
| R765VT7PWP | UNII |
| 375207 | MMSL |
| 42236 | MMSL |
| d10146 | MMSL |
| CHEBI:759033 | CHEBI |
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