NDASUBCUTANEOUSPOWDER
Approved
Nov 2021
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
7
Data completeness
4/8 — Partial
VOXZOGO (vosoritide) by BioMarin Pharmaceutical. Approved for hypochondroplasia, achondroplasia. First approved in 2021.
Drug data last refreshed 20h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
VOXZOGO (vosoritide) is a subcutaneous CNP analog approved for achondroplasia and hypochondroplasia, rare genetic skeletal dysplasias caused by FGFR3 overactivation. The drug works by binding to natriuretic peptide receptor-B (NPR-B), inhibiting the MAPK pathway and promoting endochondral ossification to stimulate bone growth. It represents a mechanistic breakthrough in addressing the underlying pathophysiology of these conditions rather than managing symptoms.
Peak16.2 years to LOE
Peak-stage product with established market presence; team likely stabilized post-launch with focus on patient access and indication expansion rather than rapid scaling.
Mechanism of Action
Mechanism of action data is being enriched from DailyMed and FDA sources. Check back soon for updated drug intelligence.
Indications (2)
Clinical Trials (7)
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
Started Apr 2026
160 enrolled
Achondroplasia
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
Started Jul 2025
60 enrolled
Hypochondroplasia
Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia
Started Jun 2025
140 enrolled
Hypochondroplasia
A Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
Started Nov 2024
54 enrolled
Short Stature Homeobox- Containing Gene SHOX DeficiencyNoonan SyndromeTurner Syndrome
A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
Started Oct 2024
100 enrolled
Idiopathic Short Stature
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.