Vosoritide
drugOn this page
Also known as BMN 111BMN-111VosoritidaVoxzogo
Summary
Vosoritide (CHEMBL3707276) is an approved protein (ATC M05BX07) targeting NPR2; indicated across 4 conditions including bone disorder and achondroplasia.
At a glance
- Status: Approved (max clinical phase 4)
- Modality: Protein
- ATC class: M05BX07
- Targets: 1 (NPR2)
- Indications: 4 conditions
- Clinical trials: 14
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL3707276 |
| Name | Vosoritide |
| Type | Protein |
| Max phase | 4 |
| ATC | M05BX07 |
Also known as: BMN 111, Bmn 111, BMN-111, Vosoritida, Vosoritide, Voxzogo, VOSORITIDE
Targets
Targets
Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).
| Gene | Target | Action | pAffinity | Cancer dependency | UniProt |
|---|---|---|---|---|---|
| NPR2 | Guanylyl cyclase-B | Agonist | P20594 |
Bioactivity
No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).
Target pathways
Aggregated over 1 target gene(s): NPR2.
Top Reactome pathways
3 total, by targets touching each:
| Pathway | Targets | Genes |
|---|---|---|
| Muscle contraction | 1 | NPR2 |
| Cardiac conduction | 1 | NPR2 |
| Physiological factors | 1 | NPR2 |
Dominant GO biological processes
| GO term | Targets |
|---|---|
| MAPK cascade | 1 |
| cumulus cell differentiation | 1 |
| vasculogenesis | 1 |
| lymph vessel development | 1 |
| endochondral ossification | 1 |
| startle response | 1 |
| blood vessel remodeling | 1 |
| chondrocyte differentiation | 1 |
| growth plate cartilage development | 1 |
| cGMP biosynthetic process | 1 |
| vacuole organization | 1 |
| receptor guanylyl cyclase signaling pathway | 1 |
| epidermal growth factor receptor signaling pathway | 1 |
| chemical synaptic transmission | 1 |
| spermatogenesis | 1 |
Indications & clinical
Indications
4 indications (2 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).
| Indication | Trial phase | MONDO | EFO |
|---|---|---|---|
| bone disorder | 4 | MONDO:0005381 | EFO:0004260 |
| achondroplasia | 4 | MONDO:0007037 | MONDO:0007037 |
2 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.
Clinical trials
Total trials: 14.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE2 | 9 |
| PHASE3 | 4 |
| PHASE1 | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT03424018 | PHASE3 | ACTIVE_NOT_RECRUITING | An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia |
| NCT06455059 | PHASE3 | ACTIVE_NOT_RECRUITING | Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia |
| NCT07073014 | PHASE3 | ENROLLING_BY_INVITATION | Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia |
| NCT03197766 | PHASE3 | COMPLETED | A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia |
| NCT02724228 | PHASE2 | ACTIVE_NOT_RECRUITING | A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH) |
| NCT04219007 | PHASE2 | ACTIVE_NOT_RECRUITING | Vosoritide for Selected Genetic Causes of Short Stature |
| NCT04554940 | PHASE2 | ACTIVE_NOT_RECRUITING | A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia |
| NCT05849389 | PHASE2 | RECRUITING | Vosoritide for Short Stature in Turner Syndrome |
| NCT06382155 | PHASE2 | RECRUITING | A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature |
| NCT06668805 | PHASE2 | RECRUITING | A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment |
| NCT07126262 | PHASE2 | RECRUITING | A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months |
| NCT02055157 | PHASE2 | COMPLETED | A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia |
| NCT03583697 | PHASE2 | COMPLETED | A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia |
| NCT01590446 | PHASE1 | COMPLETED | A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers |
Clinical evidence (CIViC)
No CIViC predictive evidence (expected for non-precision-medicine drugs).
Pharmacology
Pharmacogenomics
No PharmGKB pharmacogenomic data curated for this drug.
Related molecules
Related molecules
No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).
Related Atlas pages
- Genes: NPR2
- Diseases: bone disorder, achondroplasia