BioMarin Pharmaceutical developed vosoritide (previously known as BMN 111) for children with achondroplasia, the most common form of dwarfism. Achondroplasia is an inherited autosomal-dominant chondrodysplasia caused by a gain-of-function mutation in fibroblast-growth-factor-receptor 3 (FGFR3). Vosoritide has been granted orphan drug designation in both the U.S. and Europe, and this drug participated in phase III clinical trials in children with achondroplasia. Vosoritide mimics C-type natriuretic peptide (CNP) and acts as a key regulator of longitudinal bone growth by downregulating the mitogen-activated protein kinase pathway, which is activated because of a FGFR3 gain-of-function mutation.
Originator
Approval Year
| Substance Class |
Protein
Created
by
admin
on
Edited
Sat Dec 16 08:10:42 GMT 2023
by
admin
on
Sat Dec 16 08:10:42 GMT 2023
|
| Protein Sub Type | |
| Sequence Type | COMPLETE |
| Record UNII |
7SE5582Q2P
|
| Record Status |
Validated (UNII)
|
| Record Version |
|
-
Download
| Name | Type | Language | ||
|---|---|---|---|---|
|
Official Name | English | ||
|
Common Name | English | ||
|
Common Name | English | ||
|
Common Name | English | ||
|
Common Name | English | ||
|
Common Name | English | ||
|
Code | English | ||
|
Brand Name | English | ||
|
Common Name | English | ||
|
Common Name | English |
| Classification Tree | Code System | Code | ||
|---|---|---|---|---|
|
FDA ORPHAN DRUG |
386012
Created by
admin on Sat Dec 16 08:10:42 GMT 2023 , Edited by admin on Sat Dec 16 08:10:42 GMT 2023
|
||
|
NCI_THESAURUS |
C307
Created by
admin on Sat Dec 16 08:10:42 GMT 2023 , Edited by admin on Sat Dec 16 08:10:42 GMT 2023
|
| Code System | Code | Type | Description | ||
|---|---|---|---|---|---|
|
CHEMBL3707276
Created by
admin on Sat Dec 16 08:10:42 GMT 2023 , Edited by admin on Sat Dec 16 08:10:42 GMT 2023
|
PRIMARY | |||
|
7SE5582Q2P
Created by
admin on Sat Dec 16 08:10:42 GMT 2023 , Edited by admin on Sat Dec 16 08:10:42 GMT 2023
|
PRIMARY | |||
|
DB11928
Created by
admin on Sat Dec 16 08:10:42 GMT 2023 , Edited by admin on Sat Dec 16 08:10:42 GMT 2023
|
PRIMARY | |||
|
100000181691
Created by
admin on Sat Dec 16 08:10:42 GMT 2023 , Edited by admin on Sat Dec 16 08:10:42 GMT 2023
|
PRIMARY | |||
|
7SE5582Q2P
Created by
admin on Sat Dec 16 08:10:42 GMT 2023 , Edited by admin on Sat Dec 16 08:10:42 GMT 2023
|
PRIMARY | |||
|
DE-24
Created by
admin on Sat Dec 16 08:10:42 GMT 2023 , Edited by admin on Sat Dec 16 08:10:42 GMT 2023
|
PRIMARY | |||
|
119058036
Created by
admin on Sat Dec 16 08:10:42 GMT 2023 , Edited by admin on Sat Dec 16 08:10:42 GMT 2023
|
PRIMARY | |||
|
10022
Created by
admin on Sat Dec 16 08:10:42 GMT 2023 , Edited by admin on Sat Dec 16 08:10:42 GMT 2023
|
PRIMARY | |||
|
Vosoritide
Created by
admin on Sat Dec 16 08:10:42 GMT 2023 , Edited by admin on Sat Dec 16 08:10:42 GMT 2023
|
PRIMARY | |||
|
2586354
Created by
admin on Sat Dec 16 08:10:42 GMT 2023 , Edited by admin on Sat Dec 16 08:10:42 GMT 2023
|
PRIMARY | |||
|
DTXSID001032751
Created by
admin on Sat Dec 16 08:10:42 GMT 2023 , Edited by admin on Sat Dec 16 08:10:42 GMT 2023
|
PRIMARY | |||
|
1480724-61-5
Created by
admin on Sat Dec 16 08:10:42 GMT 2023 , Edited by admin on Sat Dec 16 08:10:42 GMT 2023
|
PRIMARY | |||
|
C152918
Created by
admin on Sat Dec 16 08:10:42 GMT 2023 , Edited by admin on Sat Dec 16 08:10:42 GMT 2023
|
PRIMARY |
| From | To |
|---|---|
| 1_23 | 1_39 |
| Related Record | Type | Details | ||
|---|---|---|---|---|
|
|
TARGET -> AGONIST |
Reduces the activity of fibroblast growth factor receptor 3.
|
| Related Record | Type | Details | ||
|---|---|---|---|---|
|
|
ACTIVE MOIETY |
|
| Name | Property Type | Amount | Referenced Substance | Defining | Parameters | References |
|---|---|---|---|---|---|---|
| MOL_WEIGHT:SEQUENCE(CALCULATED) | CHEMICAL |
|
||||
| Molecular Formula | CHEMICAL |
|