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The first targeted therapy for achondroplasia: a 39-residue CNP analogue that adds about 1.6 cm a year of growth in children.
Also known as: BMN 111 · BMN-111 · Voxzogo
Part of the Growth hormone & IGF peptides cluster
Overview
Vosoritide (Voxzogo, BMN 111) is a 39-amino-acid analogue of C-type natriuretic peptide — the 37 C-terminal residues of human CNP with an added N-terminal Pro-Gly — with a longer half-life than the endogenous hormone. By activating natriuretic peptide receptor B it offsets the over-active FGFR3 signalling that stunts bone growth in achondroplasia; in the phase 3 trial it increased annualised growth velocity by 1.57 cm/year versus placebo. Approved by the EMA in August 2021 and by the FDA in 2021.
Source & context
Biological / chemical source: Synthetic engineered peptide (BioMarin)
Primary research or clinical context: Approved (EMA, August 2021; FDA, 2021) for achondroplasia in children; investigational in hypochondroplasia, Noonan syndrome and other genetic short-stature conditions
What vosoritide is
Vosoritide is a synthetic analogue of C-type natriuretic peptide (CNP), a signalling peptide that promotes growth of long bones at the growth plate. It comprises the 37 C-terminal amino acids of human CNP with proline and glycine added at the N-terminus, giving a 39-residue peptide with a longer half-life than the endogenous hormone. Developed by BioMarin as BMN 111 and sold as Voxzogo, it was approved by the European Medicines Agency in August 2021 for achondroplasia, the most common genetic skeletal dysplasia, and by the US FDA the same year — the first targeted therapy for the condition (IUPHAR record; Jones et al., Cureus 2025).
Sequence and structure
The IUPHAR one-letter sequence is PGQEHPNARKYKGANKKGLSKGCFGLKLDRIGSMSGLGC. A disulfide bridge between cysteines 23 and 39 closes the ring that natriuretic peptides need for receptor binding; the chip display above shows the linear chain. The N-terminal Pro-Gly extension is the engineering: native CNP is cleared within minutes by neutral endopeptidase, and the extension protects the peptide enough for once-daily subcutaneous injection. Molecular formula and mass are given in the structure block from the PubChem record.
Mechanism: countering FGFR3
Achondroplasia is caused by activating variants in the fibroblast growth factor receptor 3 gene (FGFR3), whose over-active signalling suppresses chondrocyte proliferation and endochondral ossification — the process by which cartilage becomes bone at the growth plate. CNP acts through natriuretic peptide receptor B to inhibit the same intracellular pathway from the other direction, so a CNP mimic with a usable half-life can restore growth-plate activity. Vosoritide is that mimic; it does not touch FGFR3 itself but neutralises its downstream effect (IUPHAR mechanism record; Savarirayan et al., Lancet 2020).
Phase 3: growth velocity
The pivotal trial randomised 121 children with achondroplasia aged 5 to under 18, at 24 sites in seven countries, to once-daily subcutaneous vosoritide or placebo for 52 weeks after a six-month baseline growth study. The adjusted mean difference in annualised growth velocity was 1.57 cm/year in favour of vosoritide (95% CI 1.22–1.93). Adverse events occurred in 98% of both groups; none of the serious adverse events was considered treatment-related and there were no deaths. The authors noted that effects on final adult height and the harms of long-term therapy were not yet known (Savarirayan et al., Lancet 2020).
| Group | Baseline | Week 52 | Week 104 |
|---|---|---|---|
| Randomised to vosoritide | 4.26 cm/year | 5.39 cm/year | 5.52 cm/year |
| Crossed over from placebo at week 52 | — | 3.81 cm/year | 5.43 cm/year |
Longer-term and younger children
In the open-label extension, all participants received 15 μg/kg daily; growth velocity in the original vosoritide group rose from 4.26 cm/year at baseline to 5.52 cm/year at week 104, with no new adverse effects (Savarirayan et al., Genetics in Medicine 2021). Compared with untreated children from an external natural-history cohort, treated children gained an additional 5.75 cm of height over three years, with growth velocity similar to the average-stature population before puberty and an improved upper-to-lower body segment ratio (Savarirayan et al., Med 2025). A separate phase 2 trial extended treatment to children under five, in sequential age cohorts down to 0–5 months (Savarirayan et al., Lancet Child & Adolescent Health 2024).
Beyond achondroplasia
Because the FGFR3–CNP axis is shared by related growth disorders, vosoritide is being tested more widely. A single-arm phase 2 trial in 26 children with hypochondroplasia — a milder FGFR3 disorder with no approved treatment — measured growth velocity after a six-month observation period and 12 months of daily injections (Dauber et al., EClinicalMedicine 2024). ClinicalTrials.gov also lists recruiting phase 2 trials in Noonan syndrome, idiopathic short stature and selected genetic causes of short stature, and a phase 2/3 comparison with a longer-acting successor, BMN 333. International consensus guidelines on implementing and monitoring vosoritide therapy were published in 2025 (Savarirayan et al., Nature Reviews Endocrinology 2025).
Relation to other peptides on this site
Vosoritide is the growth cluster's only bone-directed peptide: the others act on the growth hormone–IGF axis (sermorelin, tesamorelin, CJC-1295, ipamorelin), which is not the pathway broken in achondroplasia. Its molecular family — the natriuretic peptides ANP, BNP and CNP — is otherwise represented on this site by nesiritide-type cardiovascular peptides. It is a supervised paediatric medicine; nothing here is guidance on its use.
Sequence
One-letter sequence commonly cited for Vosoritide (educational; isoforms and modifications may differ):
PGQEHPNARKYKGANKKGLSKGCFGLKLDRIGSMSGLGC
Residues plotted ~100° apart around an α-helix — clustering of one color reveals an amphipathic face.
Structure & chemistry
Molecular identity & reference confidence
Source-supported reference
Reference values are taken from the cited molecular record or curated profile. A database match is not independent laboratory verification; modifications, counter-ions, and formulations can change the measured mass.
- Molecular form
- The 37 C-terminal residues of human C-type natriuretic peptide with Pro-Gly added at the N-terminus; disulfide bridge between residues 23 and 39
- Species
- Not established by this molecular identity audit
- Termini (N / C)
- Not specified / Not specified
Average reference mass
Reference policy updated: 2026-10-05
- IUPHAR/BPS source snapshot · 2026-10-01
- PubChem molecular record · 2026-10-01
- Molecular formula
- C176H290N56O51S3
- Average reference mass
- ≈ 4,103
- Receptors
- Natriuretic peptide receptor B (NPR-B / guanylyl cyclase B), the receptor for endogenous CNP
- Backbone
- The 37 C-terminal residues of human C-type natriuretic peptide with Pro-Gly added at the N-terminus; disulfide bridge between residues 23 and 39
- Status
- Approved: EMA, August 2021; US FDA, 2021 (achondroplasia). Orphan drug status in both jurisdictions
Disulphide bridge between residues 23 and 39 (IUPHAR record); N-terminal PG extension confers resistance to neutral endopeptidase and a longer duration of action than native CNP.
- PubChem CID: 119058036
- IUPHAR ligand: 9068
- CAS RN: 1480724-61-5
- UNII: 7SE5582Q2P
Chemistry values: curated profile, PubChem, IUPHAR/BPS Guide to Pharmacology · fetched 2026-10-01.
Clinical development snapshot
ClinicalTrials.gov lists 17 registered studies with “Vosoritide” as an intervention (snapshot 2026-10-01). A registry footprint is not evidence of efficacy or approval.
| NCT | Title | Phase | Status | Updated |
|---|---|---|---|---|
| NCT07441876 | Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia | Phase 3 | recruiting | 2026-09-24 |
| NCT06668805 | A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hor | Phase 2 | active not recruiting | 2026-09-23 |
| NCT07073014 | Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia | Phase 3 | enrolling by invitation | 2026-09-09 |
| NCT06382155 | A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature | Phase 2 | recruiting | 2026-05-22 |
| NCT07126262 | A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months | Phase 2 | recruiting | 2026-03-31 |
| NCT04219007 | Vosoritide for Selected Genetic Causes of Short Stature | Phase 2 | active not recruiting | 2026-03-18 |
Vosoritide — reconstitution calculator
Enter how much peptide is in the vial and how much diluent (e.g. bacteriostatic water) is added. The tool returns the resulting solution concentration — and, for any amount you type, the volume it corresponds to. This is educational arithmetic, not a dosing recommendation.
Reference form: The 37 C-terminal residues of human C-type natriuretic peptide with Pro-Gly added at the N-terminus; disulfide bridge between residues 23 and 39Average reference mass: 4,103 Da
Enter an amount to see the volume it corresponds to.
Educational concentration mathematics only. This tool does not recommend a dose, amount, or protocol, and nothing here is medical advice. Many peptides are not authorized by Health Canada for human use — see our reconstitution and bacteriostatic-water guides for context.
Change log
Dated observations from the data pipeline (registry flags, trial footprints, identifiers) and editorial reviews of this profile.
- IdentifiersCross-linked to IUPHAR ligand 9068 and PubChem CID 119058036.
- TrialsClinicalTrials.gov snapshot added: 17 registered studies, highest phase 3.
- EditorialProfile content reviewed and updated by the editorial team.
FAQ about Vosoritide
What is Vosoritide?+
Vosoritide (Voxzogo, BMN 111) is a 39-amino-acid analogue of C-type natriuretic peptide — the 37 C-terminal residues of human CNP with an added N-terminal Pro-Gly — with a longer half-life than the endogenous hormone. By activating natriuretic peptide receptor B it offsets the over-active FGFR3 signalling that stunts bone growth in achondroplasia; in the phase 3 trial it increased annualised growth velocity by 1.57 cm/year versus placebo. Approved by the EMA in August 2021 and by the FDA in 2021.
What Canadian product status is recorded for Vosoritide?+
Canadian product status for Vosoritide has not been verified in this profile. No verified record here does not mean no authorization exists. Status depends on the product, formulation and use; consult Health Canada's product records.
What is the typical length of Vosoritide?+
Vosoritide is commonly described as approximately 39 amino acids (C-type natriuretic peptide analogue).
What is vosoritide?+
A 39-amino-acid analogue of C-type natriuretic peptide (Voxzogo, BMN 111) that increases bone growth in children with achondroplasia. Approved by the EMA and FDA in 2021.
How does vosoritide work?+
It activates natriuretic peptide receptor B, which inhibits the intracellular pathway that over-active FGFR3 drives in achondroplasia, restoring chondrocyte activity at the growth plate.
How much extra growth does vosoritide give?+
In the phase 3 trial, 1.57 cm/year more than placebo over 52 weeks. Against untreated children, treated children gained an additional 5.75 cm over three years.
What is the sequence of vosoritide?+
PGQEHPNARKYKGANKKGLSKGCFGLKLDRIGSMSGLGC — the 37 C-terminal residues of human CNP with an N-terminal Pro-Gly, and a disulfide between residues 23 and 39.
Is vosoritide used for conditions other than achondroplasia?+
Not by approval. Phase 2 trials in hypochondroplasia, Noonan syndrome and other genetic short-stature conditions are under way as of September 2026.
Related peptides
References & further reading
- 1.Savarirayan R et al. (2020). Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial. Lancet
- 2.Savarirayan R et al. (2021). Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study. Genetics in Medicine
- 3.Savarirayan R et al. (2025). Sustained growth-promoting effects of vosoritide in children with achondroplasia from an ongoing phase 3 extension study. Med
- 4.Savarirayan R et al. (2024). Vosoritide therapy in children with achondroplasia aged 3-59 months. Lancet Child & Adolescent Health
- 5.Dauber A et al. (2024). Vosoritide treatment for children with hypochondroplasia: a phase 2 trial. EClinicalMedicine
- 6.Savarirayan R et al. (2025). International consensus guidelines on the implementation and monitoring of vosoritide therapy in individuals with achondroplasia. Nature Reviews Endocrinology
- 7.PubChem — Vosoritide (CID 119058036)