NDASUBCUTANEOUSPOWDER
Approved
Nov 2021
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
7
Data completeness
3/8 — Basic
Drug data last refreshed 4h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
VOXZOGO (vosoritide) is a subcutaneous biologic approved in 2021 for achondroplasia, the most common form of dwarfism. It is a C-type natriuretic peptide analog that promotes bone growth by signaling through natriuretic peptide receptor B. The drug represents a first-in-class mechanism for a rare genetic skeletal disorder.
Peak16.1 years to LOE
As a peak-stage orphan drug with limited competition, VOXZOGO supports a focused commercial team operating in a specialized rare disease niche with long-term revenue stability.
Mechanism of Action
Mechanism of action data is being enriched from DailyMed and FDA sources. Check back soon for updated drug intelligence.
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
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 Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
Started Nov 2024
30 enrolled
Noonan 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.