RPB

Vosoritide

Voxzogo, BMN 111, CNP analogue, modified C-type natriuretic peptide (Pro-Gly-CNP-37)

Growth hormone axis
Awaiting medical reviewEN ES DE FR PT ZH+ Follow for updates
Note on this profile. Vosoritide is an approved prescription medicine for children with achondroplasia, not a research-only compound. This page explains the published science. It is sometimes mentioned online in the context of adult height, for which there is no evidence and no biological plausibility once growth plates have closed. Community/self-directed use is essentially none documented.

1What it is

C-type natriuretic peptide (CNP) is a natural hormone related to the heart's ANP and BNP but made locally in cartilage and blood-vessel lining rather than the heart. In the growth plate — the cartilage zone at the ends of children's bones where lengthening happens — CNP is the "go" signal that balances the "stop" signal from the receptor FGFR3. Achondroplasia, the most common form of disproportionate short stature (about 1 in 25,000 births), is caused by a single FGFR3 mutation that leaves the stop signal permanently on. Mice with extra CNP are rescued from the condition, which pointed to CNP as a treatment.

Natural CNP lasts about two minutes in blood, far too short to be a drug. BioMarin added two amino acids to the front of the molecule to block the degrading enzyme neprilysin, producing vosoritide, which lasts long enough for one injection a day to reach the growth plate. It was the first medicine approved for the underlying biology of achondroplasia, in Europe in 2021 and the US later that year. A longer-acting competitor, navepegritide (TransCon CNP, brand Yuviwel, Ascendis), a weekly prodrug of CNP, received approval in 2026 †, and vosoritide met its phase 3 endpoint in the related condition hypochondroplasia in May 2026.

Community/self-directed use: essentially none documented. Online discussion of CNP analogues for adult height has no basis — once the growth plates fuse after puberty the drug has nothing to act on.

2How it's thought to work

In achondroplasia, mutant FGFR3 constantly activates the RAF–MEK–ERK (MAPK) cascade in growth-plate chondrocytes, slowing their proliferation and preventing them from enlarging (hypertrophy), so bones lengthen slowly.
Vosoritide binds NPR-B, a guanylyl cyclase receptor. The cGMP it generates activates protein kinase G II, which phosphorylates and inhibits RAF-1, cutting the MAPK signal at a point downstream of FGFR3.
Chondrocyte proliferation and hypertrophy resume and bone growth accelerates toward, though not to, the rate of unaffected children.
The same receptor exists in blood vessels, which is why transient blood-pressure drops and dizziness occur.
It works only where growth plates are open; it has no effect on bone length once they have fused.
A C-type natriuretic peptide analogue. CNP signalling holds FGFR3 down, and it is overactive FGFR3 that closes the growth plate early in achondroplasia; lifting that brake lets the cartilage columns go on lengthening.Schematic — not to scale

3What the research shows

Growth velocity in children with achondroplasia aged 5–18

A phase 3, double-blind, placebo-controlled trial (n=121; 52 weeks) found annualised growth velocity increased by 1.57 cm/year more on vosoritide 15 µg/kg/day than placebo (from ~4 cm/year to ~5.6 cm/year), with improved height Z-scores and no worsening of body proportions (Savarirayan 2020, Lancet). This is the pivotal trial. [2]
Graded B (open-label extensions)

Durability over years

Phase 2 participants treated for up to 5+ years maintained increased growth velocity, with cumulative height gains of several centimetres versus untreated natural-history controls (Savarirayan 2019, NEJM phase 2; Savarirayan 2021, Genet Med extension). Final adult height data are not yet available. [1][3]
Graded A/B

Infants and young children

A phase 2 randomised trial in children under 5 (n=75) showed increased growth velocity and no new safety signals, supporting the 2023 US label expansion to all ages (Savarirayan 2024, Lancet Child Adolesc Health). Early treatment is of particular interest because foramen magnum stenosis (narrowing of the skull base, a serious complication of achondroplasia) develops in infancy. [4]
Graded A (positive, 2026)

Hypochondroplasia

CANOPY-HCH-3 (n=80, ages 3–17; 52 weeks) reported annualised growth velocity 2.33 cm/year higher than placebo; BioMarin planned a US supplemental filing in Q3 2026 (company announcement May 2026, publication pending). [7]
Graded B (early)

Other short-stature conditions

Phase 2 studies in idiopathic short stature, Noonan syndrome, Turner syndrome and SHOX deficiency have reported growth-velocity signals; none is approved.
Not gradednot yet known

Final adult height, proportions, complications (spinal stenosis, sleep apnoea, foramen magnum)

These matter more to families than centimetres per year and remain the key unanswered questions.

4Reported side effects and interactions

Literature-reported

injection-site reactions (redness, swelling, hives) in ~85%, mostly mild and brief; vomiting (~27%); arthralgia (joint pain); transient decreases in blood pressure with dizziness, nausea or fainting (about 1 in 8 children had at least one such episode in the pivotal trial; most within 90 minutes of injection); fatigue; raised alkaline phosphatase (expected with bone growth). The label advises eating a meal or snack and drinking fluids about 30 minutes before injection to reduce blood-pressure episodes. No treatment-related serious adverse events were reported in the pivotal trial. Anti-drug antibodies occur in a minority without apparent loss of effect.

Community-reported

none documented.

Theoretical. effects on bone age advancement (not seen so far); growth-plate hyperactivity leading to disproportion (not seen); unknown very long-term effects, since treatment spans a decade of childhood; cardiovascular effects of chronic natriuretic peptide signalling.

Interactions. none formally identified; the label advises caution with other blood-pressure-lowering medicines. Not combined with growth hormone or other CNP analogues in approved use.

Markers researchers commonly monitor

Per label and trial protocols:

MarkerWhy it's watched
Height, annualised growth velocity and height-for-age Z-scoremeasured every 3–6 months with a standardised stadiometer; the efficacy marker.
Body weightfor dose banding; adjusted as the child grows.
Body proportions (upper-to-lower segment ratio, arm span)to confirm growth is proportionate.
Bone age X-ray (hand/wrist) and confirmation of open epiphysesbefore starting and periodically; treatment stops at growth-plate closure.
Blood pressure and heart rateespecially around the first doses and in children with dehydration or heart disease.
Alkaline phosphataserises with bone growth; not a safety concern.
Sleep study, spinal and foramen magnum imagingstandard achondroplasia care, to see whether treatment changes complications over time.

Free for everyone. Safety information is never behind a paywall.

5Reconstitution and handling

Supplied as single-use vials of powder (0.4, 0.56 or 1.2 mg) with a pre-filled syringe of sterile water. Caregivers are trained to by adding the , swirling gently (not shaking), and injecting subcutaneously within 3 hours, rotating sites (abdomen, thigh, buttock, upper arm). Doses are set by weight band and adjusted at clinic visits as the child grows. The calculator is not applicable; dosing follows the label's weight table under specialist supervision.

6Commonly studied alongside

Navepegritide (TransCon CNP / Yuviwel), the once-weekly competitor; infigratinib (an oral FGFR3 inhibitor in phase 3 for achondroplasia); growth hormone (approved for achondroplasia in Japan only, modest effect); teriparatide and abaloparatide (profiles 121–122) only in the sense that they are the other bone-active peptides in this library. Limb-lengthening surgery is the non-drug comparator families consider.

Informational cross-links. Not stack recommendations.

7Regulatory status by region

RegionStatus (checked —)
United StatesFDA-approved (Voxzogo, Nov 2021, accelerated approval) for children ≥5 with achondroplasia and open epiphyses; expanded Oct 2023 to all ages with open epiphyses. Hypochondroplasia sNDA planned Q3 2026 †
European UnionEMA-authorised (Aug 2021) for achondroplasia from age 2; extended to 4 months and older (2023) †
United KingdomMHRA-licensed; NICE recommended (2023) for achondroplasia from age 2 †
AustraliaTGA-registered (2022) †; PBS listing †
South AfricaNot registered as of review date †
SportNot on the WADA Prohibited List; irrelevant to adult athletes as it acts only on open growth plates

8Open questions

  • Does faster childhood growth translate into meaningfully taller final adult height and, more importantly, fewer medical complications (spinal stenosis, foramen magnum compression, sleep apnoea, hearing loss)?
  • Durability and safety across a decade of daily injections.
  • Head-to-head performance against the weekly CNP prodrug and oral FGFR3 inhibitors.
  • Whether very early (neonatal) treatment prevents foramen magnum stenosis.

9References

  1. 1Savarirayan R et al. (2019). C-type natriuretic peptide analogue therapy in children with achondroplasia. NEJM. PubMed · DOI
  2. 2Savarirayan R et al. (2020). Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial. Lancet. PubMed · DOI
  3. 3Savarirayan 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. Genet Med. PubMed
  4. 4Savarirayan R et al. (2024). Vosoritide therapy in children with achondroplasia aged 3–59 months: a multinational, randomised, double-blind, placebo-controlled, phase 2 trial. Lancet Child Adolesc Health. PubMed · DOI
  5. 5Lorget F et al. (2012). Evaluation of the therapeutic potential of a CNP analog in a Fgfr3 mouse model recapitulating achondroplasia. Am J Hum Genet. PubMed · DOI
  6. 6Wendt DJ et al. (2015). Neutral endopeptidase-resistant C-type natriuretic peptide variant represents a new therapeutic approach for treatment of fibroblast growth factor receptor 3-related dwarfism. J Pharmacol Exp Ther. PubMed · DOI
  7. 7BioMarin (2026). Positive phase 3 results for Voxzogo in children with hypochondroplasia (CANOPY-HCH-3), press release 20 May 2026. PubMed
  8. 8BioMarin. Voxzogo (vosoritide) prescribing information, revised Oct 2023; EMA EPAR (2021). PubMed

References are being resolved to PubMed/DOI identifiers; links search PubMed for the citation until then.

† Being confirmed by our reviewers against current sources.

10Community

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Self-reported side effects Phase 2

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11Page history

30 Oct 2026Profile published — awaiting medical review

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