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| Stem definition | Drug id | CAS RN |
|---|---|---|
| humanized origin | 5308 | 915810-67-2 |
| Molecule | Description |
|---|---|
|
Synonyms:
|
In patients with aTTP, levels of a substance called von Willebrand factor are increased. von Willebrand factor acts on platelets to cause them to stick together and form blood clots. Caplacizumab, the active substance in Cablivi, is a nanobody (a small antibody) which has been designed to attach to von Willebrand factor in a way that stops it acting on platelets. This reduces platelets sticking together and forming clots in blood vessels and, as a result, platelet levels in the blood rise because they are no longer taken up to form clots.
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| Dose | Unit | Route |
|---|---|---|
| 10 | mg | P |
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| Date | Agency | Company | Orphan |
|---|---|---|---|
| Aug. 30, 2018 | EMA | ABLYNX NV | |
| Feb. 6, 2019 | FDA | ABLYNX NV | |
| Sept. 26, 2022 | PMDA | SANOFI K.K. |
| MedDRA adverse event term | Likelihood ratio | Likelihood ratio threshold | Patients taking drug having adverse event | Patients taking drug not having adverse event | Patients not taking drug having adverse event | Patients not taking drug not having adverse event |
|---|---|---|---|---|---|---|
| ADAMTS13 activity decreased | 491.00 | 45.57 | 56 | 1440 | 142 | 90626809 |
| Product use in unapproved indication | 377.83 | 45.57 | 156 | 1340 | 336080 | 90290871 |
| ADAMTS13 activity abnormal | 230.45 | 45.57 | 23 | 1473 | 0 | 90626951 |
| Epistaxis | 116.55 | 45.57 | 46 | 1450 | 85079 | 90541872 |
| Platelet count decreased | 98.55 | 45.57 | 47 | 1449 | 136922 | 90490029 |
| Off label use | 61.05 | 45.57 | 82 | 1414 | 1102170 | 89524781 |
| Injection site bruising | 56.84 | 45.57 | 24 | 1472 | 52283 | 90574668 |
| MedDRA adverse event term | Likelihood ratio | Likelihood ratio threshold | Patients taking drug having adverse event | Patients taking drug not having adverse event | Patients not taking drug having adverse event | Patients not taking drug not having adverse event |
|---|---|---|---|---|---|---|
| ADAMTS13 activity decreased | 176.43 | 42.20 | 21 | 476 | 131 | 42620707 |
| Product use in unapproved indication | 145.38 | 42.20 | 60 | 437 | 184396 | 42436442 |
| ADAMTS13 activity abnormal | 103.69 | 42.20 | 10 | 487 | 0 | 42620838 |
| Platelet count decreased | 48.46 | 42.20 | 26 | 471 | 138555 | 42482283 |
| Platelet count abnormal | 44.74 | 42.20 | 10 | 487 | 3639 | 42617199 |
| MedDRA adverse event term | Likelihood ratio | Likelihood ratio threshold | Patients taking drug having adverse event | Patients taking drug not having adverse event | Patients not taking drug having adverse event | Patients not taking drug not having adverse event |
|---|---|---|---|---|---|---|
| ADAMTS13 activity decreased | 596.51 | 35.70 | 71 | 1936 | 258 | 110587034 |
| Product use in unapproved indication | 508.65 | 35.70 | 215 | 1792 | 447162 | 110140130 |
| ADAMTS13 activity abnormal | 297.73 | 35.70 | 30 | 1977 | 0 | 110587292 |
| Epistaxis | 137.19 | 35.70 | 60 | 1947 | 130312 | 110456980 |
| Platelet count decreased | 104.78 | 35.70 | 60 | 1947 | 230383 | 110356909 |
| Platelet count abnormal | 88.76 | 35.70 | 21 | 1986 | 6261 | 110581031 |
| Injection site bruising | 71.87 | 35.70 | 29 | 1978 | 51234 | 110536058 |
| Off label use | 69.72 | 35.70 | 108 | 1899 | 1500077 | 109087215 |
| Gingival bleeding | 45.13 | 35.70 | 16 | 1991 | 19823 | 110567469 |
| Contusion | 41.28 | 35.70 | 34 | 1973 | 233327 | 110353965 |
| Injection site pain | 38.05 | 35.70 | 29 | 1978 | 177893 | 110409399 |
| Platelet count increased | 37.23 | 35.70 | 15 | 1992 | 26319 | 110560973 |
None
| Source | Code | Description |
|---|---|---|
| ATC | B01AX07 | BLOOD AND BLOOD FORMING ORGANS ANTITHROMBOTIC AGENTS ANTITHROMBOTIC AGENTS Other antithrombotic agents |
| CHEBI has role | CHEBI:50249 | anticoagulant |
| 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 | ANTIBODY BINDING | Kd | 8.51 | IUPHAR | DRUG LABEL |
| ID | Source |
|---|---|
| 2R27AB6766 | UNII |
| 4038197 | VANDF |
| 4038198 | VANDF |
| C3713057 | UMLSCUI |
| CHEBI:754365 | CHEBI |
| CHEMBL2109624 | ChEMBL_ID |
| DB06081 | DRUGBANK_ID |
| D11160 | KEGG_DRUG |
| 9511 | INN_ID |
| C585343 | MESH_SUPPLEMENTAL_RECORD_UI |
| 9123 | IUPHAR_LIGAND_ID |
| 784411004 | SNOMEDCT_US |
| 789447000 | SNOMEDCT_US |
| 36265 | MMSL |
| 36266 | MMSL |
| d09171 | MMSL |
| 017896 | NDDF |
| 2110604 | RXNORM |
None