VOXZOGO (vosoritide)
VOXZOGO (vosoritide) is a C-type natriuretic peptide (CNP) analog indicated to increase linear growth in pediatric patients with achondroplasia who have open epiphyses. The drug received accelerated approval based on demonstrated improvements in annualized growth velocity. Continued approval for this indication may be contingent upon the verification and description of clinical benefit in confirmatory trials.
How VOXZOGO Works
In patients with achondroplasia, endochondral bone growth is negatively regulated by a gain-of-function mutation in fibroblast growth factor receptor 3 (FGFR3). Vosoritide binds to natriuretic peptide receptor-B (NPR-B), which antagonizes FGFR3 downstream signaling by inhibiting the ERK1/2 and MAPK pathways. This inhibition promotes chondrocyte proliferation and differentiation, acting as a positive regulator of endochondral bone growth to increase skeletal growth.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2021-11-19
- Patent Cliff
- 2042
- Revenue
- $273M (Q4-2025)
- Routes
- SUBCUTANEOUS
- Dosage Forms
- POWDER
VOXZOGO Approval History
What VOXZOGO Treats
1 indicationsVOXZOGO is approved for 1 conditions since its original approval in 2021. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Achondroplasia
VOXZOGO Target & Pathway
ProTarget
VOXZOGO Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
MoA expansion candidates
Same target(s), different indications — where else is this mechanism being explored?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
Clinical Trial Registry
6 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT07126262 | 111-212 | Ph 2 | recruiting | A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months |
| NCT04219007 | Pro00013585 | Ph 2 | active not recruiting | Vosoritide for Selected Genetic Causes of Short Stature |
| NCT04554940 | 111-209 | Ph 2 | active not recruiting | A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia |
| NCT06455059 CANOPY-HCH-3 | 111-303 | Ph 3 | active not recruiting | Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia |
| NCT07073014 CANOPY-HCH-EXT | 111-308 | Ph 3 | enrolling by invitation | Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia |
| NCT05849389 | STUDY0000567 | Ph 2 | recruiting | Vosoritide for Short Stature in Turner Syndrome |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
VOXZOGO FDA Label Details
Indications & Usage
FDA Label (PDF)VOXZOGO is indicated for the treatment of Achondroplasia.
VOXZOGO Patents & Exclusivity
Patents (8 active)
Exclusivity
Pro Intelligence Preview
Deep insights for VOXZOGO
Revenue Insights
- • Q4-2025: $273M
- • Historical trend analysis
Patent Timeline
- • Cliff: 2042
- • 72 active patents
Trial Analysis
- • 6 total trials
- • Stage: Growth
Competitive Landscape
- • Competitor tracking
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment