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Drug

Vosoritide

Approved medicine
ClassAtrial natriuretic peptide receptor B binding agentResearchOngoing clinical research

Also known as Voxzogo.

RxNorm2586354ChEMBLCHEMBL3707276ATCM05BX07UNII7SE5582Q2P
10
Active clinical trials
NPR2
Primary target
1
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vosoritide in Children With Hypochondroplasia

Clinical trial2026-08-01Primary completion · ClinicalTrials.gov

A Multicenter, Randomized, Operationally Seamless Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia

Clinical trial2026-07-06Results expected Q2 2029 · ClinicalTrials.gov

A Phase 2, Randomized, Controlled, Multicenter Study of Vosoritide in Children With Idiopathic Short Stature

Clinical trial2026-06-01Primary completion · ClinicalTrials.gov

A Phase 2, Randomized, Multicenter, Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment

Clinical trial2026-05-11Results expected Q1 2027 · ClinicalTrials.gov

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Vosoritide in Infants and Young Children With Hypochondroplasia, Aged 0 to < 36 Months

Clinical trial2026-03-31Results expected Q2 2028 · ClinicalTrials.gov

A Randomized, Controlled, Open-label Clinical Trial With an Open-label Extension to Investigate the Safety of Vosoritide in Infants and Young Children With Achondroplasia at Risk of Requiring Cervicomedullary Decompression Surgery

Clinical trial2026-03-13Results expected Q4 2027 · ClinicalTrials.gov

A Proof of Concept Study to Evaluate the Safety and Efficacy of Voxzogo (Vosoritide) for the Treatment of Growth Deficits in MPS IVA and VI

Clinical trial2026-03-10Results expected Q4 2028 · ClinicalTrials.gov

Vosoritide for Selected Genetic Causes of Short Stature

Clinical trial2025-08-14Primary completion · ClinicalTrials.gov

A Phase 3, Open-Label, Long-Term Extension Study to Evaluate the Safety and Efficacy of Vosoritide in Children With Hypochondroplasia

Clinical trial2025-07-18Results expected Q4 2040 · ClinicalTrials.gov

Vosoritide for Treatment of Short Stature in Girls With Turner Syndrome

Clinical trial2024-06-24Results expected Q3 2026 · ClinicalTrials.gov

Approval: Voxzogo (EMA)

Regulatory2021-08-26EMA

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Vosoritide
Aliases & brands
Voxzogo
RxNorm CUI
2586354
ChEMBL ID
CHEMBL3707276
ATC codes
M05BX07
UNII
7SE5582Q2P
Primary mechanism
Atrial natriuretic peptide receptor B binding agent
Regulatory jurisdictions
ema

Pharmacology & targets

1 target

Known molecular targets and mechanisms supported by curated pharmacology databases.

Atrial natriuretic peptide receptor 2
NPR2 · P20594
BINDING AGENTAtrial natriuretic peptide receptor B binding agent

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2021-08-26
Latest approval
2021-08-26
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2021-08-26Approval
Approval: Voxzogo (EMA)
Indication: Voxzogo is indicated for the treatment of achondroplasia in patients 4 months of age and older whose epiphyses are not closed. The diagnosis of achondroplasia should be confirmed… Show full indication

Voxzogo is indicated for the treatment of achondroplasia in patients 4 months of age and older whose epiphyses are not closed. The diagnosis of achondroplasia should be confirmed by appropriate genetic testing.

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

10 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
10
registered trials across all phases
10
Active studies
6
Recruiting
3
Late-stage (III+)
PHASE DISTRIBUTIONn = 10
Phase 1 / 21Phase 26Phase 2 / 31Phase 32

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

A Phase 3, Open-Label, Long-Term Extension Study to Evaluate the Safety and Efficacy of Vosoritide in Children With Hypochondroplasia

Enrolling by invitationPH IIIBioMarin Pharmaceuticaln=1402040

A Multicenter, Randomized, Operationally Seamless Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia

RecruitingPH II/IIIBioMarin Pharmaceuticaln=1602029

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vosoritide in Children With Hypochondroplasia

Active, not recruitingPH IIIBioMarin Pharmaceuticaln=802026
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.