Vosoritide
Vosoritide is an FDA-approved C-type natriuretic peptide analog. Achondroplasia is caused by gain-of-function variants in FGFR3 that over-activate the MAPK pathway and suppress chondrocyte proliferation at the growth plate. Vosoritide binds natriuretic peptide receptor B, raising cyclic GMP and inhibiting MAPK signalling downstream of FGFR3, which relieves that suppression.
For research use only.
Key Facts
- CAS
- 1480724-61-5
- Molecular Weight (MW)
- 4103 g/mol
- Half-life
- Mean half-life of 21.0 to 27.9 minutes (Voxzogo label)
- FDA status
- Approved
- Evidence level
- FDA Approved
- Human dose established
- Yes
- Administration Route
- Subcutaneous
- Frequency
- Once daily, at about the same time each day
- Last updated
- Nov 7, 2026
- Reviewed
- Nov 7, 2026
Research summary
Analog of C-type natriuretic peptide approved to increase linear growth in children with achondroplasia who have open epiphyses.
Research evidence
- Regulatory review (FDA label)1 reviewed source
- Controlled human trialsNone in reviewed sources
- Early or uncontrolled human studiesNone in reviewed sources
- Lab & animal studiesNone in reviewed sources
How it works
Each step shows whether the sources Peptide Library has reviewed include that kind of research. Counts are reviewed sources, not every study ever published. Reviews and programme summaries that describe human use are shown as "reported", not as trials, and registered trials without published results are listed but not counted. A higher step means research has progressed further; it does not mean a compound works better or is safer. Compounds whose evidence has not been reviewed yet show "Not reviewed" instead of a guess.
Peptide Library's Vosoritide profile summarises 5 cited studies listed in the Science section, the compound's regulatory status, and research-protocol parameters reported for it. It is a research reference, not medical advice.
Vosoritide dosage evidence
Vosoritide has an FDA-approved human dosage as Voxzogo, to increase linear growth in children with achondroplasia whose growth plates are still open, under accelerated approval. It is given once daily by subcutaneous injection at a dose set by body weight band, from 0.096 mg at 3 kg to 0.8 mg at 90 kg or more, after adequate food and about 240–300 mL of fluid.
| Human dosing established | Yes |
|---|---|
| Route | Subcutaneous |
| Frequency | Once daily, at about the same time each day |
| Duration studied | Until the growth plates close |
| Titration | No escalation: the dose follows the child's actual body weight band and is re-checked every 3 to 6 months. |
| Half-life | Mean half-life of 21.0 to 27.9 minutes (Voxzogo label) |
| Evidence level | FDA Approved |
| Last reviewed | 2026-11-07 |
Research-reported dosing
Doses as studied, one row per regimen. Different trials used different regimens; they are listed separately rather than averaged.
| Study / context | Population | Dose | Route | Frequency | Duration | Outcome studied | Source |
|---|---|---|---|---|---|---|---|
| FDA label — Voxzogo, achondroplasiaFDA-approved label · FDA prescribing information | Children with achondroplasia and open epiphyses | By weight band: 0.096 mg at 3 kg, 0.12 mg at 4 kg, 0.16 mg at 5 kg, 0.2 mg at 6–7 kg, 0.24 mg at 8–11 kg, 0.28 mg at 12–16 kg, 0.32 mg at 17–21 kg, 0.4 mg at 22–32 kg, 0.5 mg at 33–43 kg, 0.6 mg at 44–59 kg, 0.7 mg at 60–89 kg, 0.8 mg at 90 kg or more | Subcutaneous, rotating sites | Once daily | Stopped once the growth plates close | Annualised growth velocity: in the label's 52-week placebo-controlled study (121 children aged 5 to 14 given 15 mcg/kg daily), the treatment difference was 1.57 cm/year | VOXZOGO (vosoritide) for injection — FDA prescribing information, DailyMed |
Where sources disagree
- Voxzogo's approval is accelerated, based on annualised growth velocity; continued approval may depend on confirmatory trials. The label's phase 3 study used 15 mcg/kg once daily, and the label's weight-band table gives fixed doses in milligrams.
- The label warns of transient drops in blood pressure, which is why each dose follows adequate food and about 240–300 mL of fluid in the hour before. Children with significant heart or vascular disease and those on blood pressure medicines were excluded from the trials.
- Voxzogo comes as kits of single-dose powder vials (0.4, 0.56 and 1.2 mg), each with a prefilled syringe of sterile water: 0.5 mL for the 0.4 mg vial and 0.7 mL for the 0.56 mg vial give 0.8 mg/mL, and 0.6 mL for the 1.2 mg vial gives 2 mg/mL. One dose is taken per vial and the rest discarded. Vosoritide is a C-type natriuretic peptide analogue.
Sequence & molecular data
| Molecular formula | C176H290N56O51S3 |
|---|---|
| Molecular weight | 4103 g/mol |
| CAS number | 1480724-61-5 |
| PubChem CID | 119058036 |
| FDA UNII | 7SE5582Q2P |
Identifiers link to the public PubChem, FDA Substance Registration (UNII), ChEMBL and DrugBank records for the same entity.
Science
Mechanism of Action
Achondroplasia is caused by gain-of-function variants in FGFR3 that over-activate the MAPK pathway and suppress chondrocyte proliferation at the growth plate. Vosoritide binds natriuretic peptide receptor B, raising cyclic GMP and inhibiting MAPK signalling downstream of FGFR3, which relieves that suppression. Its modified N-terminus resists neutral endopeptidase degradation, giving a longer half-life than native CNP.
Studies
- Sustained growth-promoting effects of vosoritide in children with achondroplasia from an ongoing phase 3 extension study
- Vosoritide therapy in children with achondroplasia aged 3-59 months: a multinational, randomised, double-blind, placebo-controlled, phase 2 trial
- Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study
- Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial
- Vosoritide (Voxzogo) for Achondroplasia: A Review of Clinical and Real-World Evidence
Research statistics
100 PubMed publications · 49.5% human clinical research · 18 registered trials
Counts of published research through 2026-11-01, not evidence that it works. Publications by year, study types and trials · How publications are counted
Regulatory & research status
Vosoritide is an FDA-approved product for specific labelled indications. Approval does not extend to the research contexts described on this page, and approved products are prescription medicines.
| FDA status | Approved |
|---|---|
| Research status | Approved product (see FDA status) |
| Availability | Prescription-Only |
| WADA (sport) | Unknown / Not Listed |
| Library classification | FDA-Approved |
How FDA approval, compounding and research-only status differ
Benefits
—
Vosoritide side effects
No side effects listed yet for this entry.
Storage & stability
This compound is not supplied as a powder to be mixed, so the freeze-dried powder and reconstitution handling described for research peptides does not apply. Storage conditions are product-specific: follow the label or packaging of the product itself.
Vosoritide: frequently asked questions
What is Vosoritide?
Analog of C-type natriuretic peptide approved to increase linear growth in children with achondroplasia who have open epiphyses.
How does Vosoritide work?
Achondroplasia is caused by gain-of-function variants in FGFR3 that over-activate the MAPK pathway and suppress chondrocyte proliferation at the growth plate. Vosoritide binds natriuretic peptide receptor B, raising cyclic GMP and inhibiting MAPK signalling downstream of FGFR3, which relieves that suppression.
Is Vosoritide FDA approved?
Yes. Vosoritide is approved by the FDA, and approved products are prescription medicines. Approval is always for specific indications studied in clinical trials, so an approved status does not mean the compound is approved for every use it is discussed for. Research-chemical material sold under the same name is not the approved product.
What peptides are similar to Vosoritide?
Compounds most often compared with Vosoritide include Human Growth Hormone (Somatropin), Lanreotide, Lonapegsomatropin and Macimorelin. They are grouped by shared mechanism or research area rather than by equivalence, and their regulatory status and evidence base differ.
References
- Sustained growth-promoting effects of vosoritide in children with achondroplasia from an ongoing phase 3 extension study — Med, 2025External reference
- Vosoritide therapy in children with achondroplasia aged 3-59 months: a multinational, randomised, double-blind, placebo-controlled, phase 2 trial — Lancet Child Adolesc Health, 2024External reference
- Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study — Genet Med, 2021External reference
- Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial — Lancet, 2020External reference
- Vosoritide (Voxzogo) for Achondroplasia: A Review of Clinical and Real-World Evidence — Cureus, 2025External reference
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Compiled by Peptide Library Editorial · Reviewed Aug 30, 2026 · Updated Nov 7, 2026 · Version 2
This Vosoritide profile is compiled from published literature (5 cited studies linked above), public regulatory records and chemical registries. It describes what research reports; it is not medical advice, and nothing here is a dosing recommendation for humans. Peptide Library does not sell peptides. Editorial policy · How profiles are compiled · Report an error
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