Vosoritide (Voxzogo)_Dosage, benefits & legal status
Also known as Voxzogo, BMN 111, BMN-111, DE-24
A 39-residue CNP analog with two extra amino acids that resist the enzyme neprilysin, injected once a day while growth plates are open. In the phase 3 trial, children aged 5 to 14 grew 1.57 cm a year faster than on placebo.
Common vial size is what vendors typically sell, not a recommendation. Enter your own vial in the calculator.
What is Vosoritide (Voxzogo)?
Plain language first. The technical label stays, but it never stands alone.Overview
Children with achondroplasia grow slowly because an overactive receptor, FGFR3, brakes bone growth in their growth plates, and vosoritide counters that brake with a daily injection. In the 121-child phase 3 trial (ages 5 to 14), growth was 1.57 cm a year faster than on placebo after 52 weeks, and the gain held at 2 years. It works only while growth plates are open, and its effect on final adult height is not yet established; the FDA is due to decide on full approval by February 28, 2027.
Vosoritide is a recombinant 39-residue analog of C-type natriuretic peptide, made in E. coli by BioMarin: the 37 C-terminal residues of human CNP-53 with an added N-terminal Pro-Gly that resists neutral endopeptidase (neprilysin). It activates NPR-B on growth-plate chondrocytes, raising cGMP and reducing ERK1/2 signaling in the MAPK pathway held on by the constitutively active FGFR3 that causes achondroplasia. With a half-life of 21 to 28 minutes it is injected daily, unlike navepegritide (Yuviwel), a weekly prodrug that releases native-sequence CNP-38.
What it's used for
A once-daily injection under the skin for children with achondroplasia whose growth plates are still open, to increase how fast they grow. The US sets no lower age limit; the EU approves it from 4 months.
How does Vosoritide (Voxzogo) work?
What it does in the body, and where.Vosoritide stands in for CNP, the body's own counterweight to the growth-plate brake that is stuck on in achondroplasia.
- 01The stuck brakeAchondroplasia comes from an FGFR3 receptor that is constantly active in growth-plate cartilage. It keeps the MAPK (ERK1/2) pathway running and so slows the multiplying and maturing of cartilage cells that makes bones longer.
- 02The counter-signalVosoritide binds NPR-B, a receptor that is also an enzyme (guanylyl cyclase B), and raises cGMP inside the cartilage cells. cGMP turns down ERK1/2 signaling, so the cells multiply and mature again and bones lengthen.
- 03Daily pulsesThe Pro-Gly added at its front end protects it from the enzyme neprilysin, but it still clears fast: it peaks about 15 minutes after an injection and has a half-life of 21 to 28 minutes. Daily injections give a pulse of signal each day rather than constant exposure.
A schematic of the compound's mechanism, not a diagnostic image.
What else is it used for?
Each group carries its own evidence level.Approved means regulators have cleared it for that purpose. Investigational means it is in trials. Early research means it is being studied and is not established.
Phase 3, ages 5 to 14 (111-301)
FDA-approved- Human trial121 children: growth velocity 1.57 cm/year above placebo at 52 weeks (95% CI 1.22 to 1.93); height standard deviation score up 0.28 more.
- No dataThe registry's arm means (1.71 against 0.13) differ from the label's (1.40 against -0.17) because of a different model; the difference between arms is essentially the same (about 1.58 against 1.57).
Under age 5 (111-206)
FDA-approved- Human trial64 children randomized from 4.4 months to under 5 years: height Z-score 0.30 above placebo at 52 weeks (95% CI 0.07 to 0.54), at 30 mcg/kg a day under age 2 and 15 mcg/kg from age 2.
Long-term height
FDA-approved- Human observationChildren who started after age 5: mean height 10.60 cm above natural-history comparisons after 6 years and 13.59 cm after 8 years (p<0.0001).
- No dataNot final adult height and not randomized; observed adult height data sit in the full-approval application and are not yet public.
Hypochondroplasia (CANOPY-HCH-3)
Investigational- Human trial81 children aged 3 to under 18 randomized (41 against 40): growth velocity change 1.95 against -0.39 cm/year, a difference of 2.33 cm/year (95% CI 1.85 to 2.82); standing height 2.35 cm more than on placebo.
- Human trialAny adverse event 87.8% against 72.5%, with none of grade 3 or higher and no stops for side effects.
- RegulatoryNot approved for hypochondroplasia anywhere; a US application was submitted in 2026.
Headlines of a 13.6 cm height gain refer to children who started after age 5 being 13.59 cm taller after 8 years than untreated children in historical natural-history records; it is not final adult height and not a randomized result. The 21.7 to 26.4 cm final height gains also quoted are modeled projections for treatment from 6 months of age. Vosoritide does not raise adult height in people whose growth plates have closed, and whether it increases final adult height is not yet established: US approval is still accelerated, with a full-approval decision due February 28, 2027. The first FDA approval is often reported as covering children with achondroplasia in general; on November 19, 2021 it was limited to ages 5 and older, and the October 20, 2023 expansion removed the US age floor entirely, while the EU starts at 4 months. The trial dose of 15 mcg/kg is often quoted as the dose; the label uses fixed weight bands. It is not approved for hypochondroplasia, and that phase 3 trial randomized 81 children, not the 80 sometimes reported. The registry and label give different arm means for the phase 3 trial, but the difference is essentially the same, about 1.57 cm a year. It is a CNP analog with an altered sequence and is injected daily; navepegritide, injected weekly, is a prodrug of native-sequence CNP-38, though its US label also uses the CNP analog class name.
The published record
Papers and trials about Vosoritide (Voxzogo)
92 papers tagged human · 55 not reviews or lab · 10 clinical trial publications
18 registered trials · 3 with posted results
Furthest stage reached: Approved in the US
Counted from Europe PMC indexing, not read by a person; a human tag can land on reviews, commentary or lab papers; clinical trial publications is the narrower count.
At a glance
Four things to know about Vosoritide (Voxzogo)
Evidence
FDA-approved
Category
Growth hormone
Common vial
Varies
Half-life
About 21 to 28 minutes
How much, and do you cycle it?
The range, and whether you take breaks.0.096 to 0.8 mg once a day by body weight, from 3 kg up (label), once a day. Daily injections until the growth plates close FDA label
Given every day while the child can still grow; the label says to stop when the growth plates close.
How long does it stay in the body?
The half-life, and where the figure comes from.About 21 to 28 minutesFDA label
Mean 21.0 to 27.9 minutes; the EU gives 27.9 minutes (SD 9.9). Peaks about 15 minutes (median) after an injection under the skin. Exposure rises more than in proportion to dose from 7.5 to 30 mcg/kg; body weight affects it, but age (0.4 to 15 years), sex and race do not. Released CNP from weekly navepegritide has an apparent half-life of about 5.3 days, for comparison.
Source: Voxzogo US prescribing information, section 12.3; EU summary of product characteristics, section 5.2
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How is it given?
Dosing context and source notes.Supplied as a kit with its own diluent, and the label sets the volume, so there is nothing to work out here.
Label range: 96 up to 800 mcg per day. Voxzogo US prescribing information (DailyMed), section 2, Table 1
One injection under the skin once a day, at about the same time, at a fixed dose for each band of actual body weight: 0.096 mg at 3 kg, 0.12 mg at 4 kg, 0.16 mg at 5 kg, 0.2 mg at 6 to 7 kg, 0.24 mg at 8 to 11 kg, 0.28 mg at 12 to 16 kg, 0.32 mg at 17 to 21 kg, 0.4 mg at 22 to 32 kg, 0.5 mg at 33 to 43 kg, 0.6 mg at 44 to 59 kg, 0.7 mg at 60 to 89 kg and 0.8 mg at 90 kg or more. The label gives no mcg/kg dose: the trials used 15 mcg/kg a day, or 30 mcg/kg a day under age 2, and the bands work out to about 30 mcg/kg at 3 to 4 kg and under 9 mcg/kg at 90 kg. The child should have eaten enough and had 240 to 300 mL of fluid in the hour before each dose, to limit blood pressure drops. A missed dose can be given within 12 hours, otherwise it is skipped. Weight and growth are checked every 3 to 6 months, and treatment stops when the growth plates close. Not recommended when kidney function (eGFR) is below 60 mL/min/1.73 m2. Supplied as co-packs with a 0.4, 0.56 or 1.2 mg powder vial, a prefilled diluent syringe, a transfer needle and syringes; refrigerated, or at room temperature for up to 90 days, and the mixed solution is used within 3 hours. The dose is set by body weight, so the calculator cannot draw one.
How do you store it?
Before mixing, and after.Follow the storage on the product label.
Vosoritide (Voxzogo) is supplied as a finished product, so its label sets the storage. Pens, prefilled syringes, implants, tablets and ready-made solutions are used as supplied. Kits that include a powder are mixed just before the dose and usually used straight away, not kept for weeks. Many injectable products go in the fridge and must not be frozen, while others are kept at room temperature, so check the pack or ask the pharmacist.
What are the side effects and cautions?
Known cautions and warnings for this compound.Solubility, computed: 31% charged residues, 33% hydrophobic. Water usually sufficient.
A first-pass rule from peptide manufacturers' guidance. It does not account for disulfide bridges, salt form or how the powder was dried, and it predicts what should be easy rather than what will happen in your vial.
Who should avoid it
- None on the US label
- Not recommended when kidney function (eGFR) is below 60 mL/min/1.73 m2
- Not studied in children with significant heart or blood-vessel disease or on blood pressure medicines, who were excluded from the trials
- No use once the growth plates have closed: treatment stops when they close
- Effect of liver impairment unknown
Stop and get help
- Dizziness, feeling faint, fainting, fatigue or nausea after a dose, possible signs of low blood pressure
Common effects
- Injection-site redness 75% against 69% on placebo, swelling 62% against 36% and hives 25% against 10% (ages 5 and older, 60 against 61 children)
- Vomiting 27% against 20%, joint pain 15% against 7%, decreased blood pressure 13% against 5%, and dizziness and diarrhea 10% against 3% each
- Raised alkaline phosphatase 17% against 7%; under age 5, injection-site reactions in 86% and rash in 28%
- Antibodies to the drug in 35% of children 5 and older and 33% of those under 5, none of them neutralizing
Where this page says nothing is established, that means nobody has studied it, not that a compound is safe.
Is Vosoritide (Voxzogo) approved?
And where the numbers on this page come from.Yes. Vosoritide (Voxzogo) is FDA approved and available on prescription in the United States. It is also approved in the European Union, Japan, Brazil, and Australia.
- Approval statusFDA-approved
- RouteUnder the skin (daily injection)
- Checked17 October 2026
- Approved outside the USEuropean Union, Japan, Brazil, Australia: EU authorization on August 26, 2021, first from age 2 and since late 2023 from 4 months, with diagnosis confirmed by genetic testing; Brazil approved it in 2021; Japan's approval for children of all ages with open growth plates was announced on June 21, 2022; Australia approves it for all ages with open epiphyses. BioMarin reports more than 5,000 children treated in more than 50 countries; UK, Canadian, Chinese and Indian approval dates are not established. FDA accelerated approval since November 19, 2021.This does not change the United States position above.
- DoseFrom the drug label. The manufacturer tested this range and a regulator reviewed it.
- Vial sizeFrom what vendors typically list; not a recommendation.
- SourcesVoxzogo US prescribing information (revised 11/2024)DailyMed, VoxzogoDrugs@FDA, NDA 214938Original Voxzogo label (2021)FDA supplement approval letter, S-002 (2023)FDA GSRS substance record, vosoritide (UNII 7SE5582Q2P)EMA, Voxzogo EPARVoxzogo EU summary of product characteristicsBioMarin, Japan approval (June 21, 2022)Savarirayan R et al., Lancet 2020;396:684-692 (phase 3)Savarirayan R et al., Lancet Child and Adolescent Health 2024;8:40-50 (under 5)Dauber A et al., NEJM Evidence 2026 (CANOPY-HCH-3)BioMarin, full-approval application accepted (July 13, 2026)BioMarin, first quarter 2026 results
What has happened recently
BioMarin discovered vosoritide in-house as BMN 111, and the EU granted orphan designation on January 24, 2013. The EU authorized Voxzogo on August 26, 2021, for children 2 and older whose epiphyses are not closed, extended to 4 months and older after a positive opinion from the European Medicines Agency's medicines committee (CHMP) on September 15, 2023; it is a standard, not conditional, authorization. The FDA granted accelerated approval on November 19, 2021 (NDA 214938, a new molecular entity with orphan status) for children 5 and older with open epiphyses, and on October 20, 2023 widened it to all pediatric patients with open epiphyses, with no lower age limit. Japan's approval for children of all ages with open growth plates was announced on June 21, 2022. The confirmatory final adult height study was due to finish in November 2025, with its report in August 2026; BioMarin submitted a supplemental application for full approval in April 2026, its acceptance was announced on July 13, 2026, and the FDA decision date is February 28, 2027. A hypochondroplasia application was submitted in the US in 2026; that use is not approved anywhere.
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What else is like Vosoritide (Voxzogo)?
Others in Growth hormone, side by side.If you're weighing Vosoritide (Voxzogo), it's worth reading Ipamorelin, CJC-1295 with DAC, and CJC-1295 no DAC (Mod GRF 1-29) in the same class.
A 39-residue CNP analog with two extra amino acids that resist the enzyme neprilysin, injected once a day while growth plates are open. In the phase 3 trial, children aged 5 to 14 grew 1.57 cm a year faster than on placebo.
- Dose
- 0.096 to 0.8 mg once a day by body weight, from 3 kg up (label)
- How often
- Once a day
A gentle shot that nudges your body to release its own growth hormone.
- Dose
- 0.2 up to 0.3 mg per dose (start low, increase slowly)
- How often
- Daily, often at night
A once-or-twice-weekly shot that keeps your growth hormone raised for days.
- Dose
- 1 up to 2 mg per dose (start low, increase slowly)
- How often
- 1-2x per week
A short-acting growth-hormone pulse, usually paired with ipamorelin.
- Dose
- 0.1 mg per dose
- How often
- Daily to 2x daily
Guides for this compound
- How to Reconstitute a Peptide
Mix peptide powder with bacteriostatic water, step by step.
- How to Read an Insulin Syringe
Read the units, convert to millilitres, and draw the exact dose.
- mcg vs mg: The Peptide Dosing Mistake to Avoid
Your vial says mg and your protocol says mcg. Here is the conversion and how to check it.
Sources
- Voxzogo US prescribing information (revised 11/2024)(2024)Indication for all children with open epiphyses under accelerated approval; phase 3 difference 1.57 cm/year (95% CI 1.22 to 1.93); blood pressure warning; half-life 21.0 to 27.9 minutes.
- DailyMed, Voxzogo(2025)Weight bands from 0.096 to 0.8 mg; adverse reactions against placebo; co-packs with a prefilled diluent syringe; 39 amino acids made in E. coli.
- Drugs@FDA, NDA 214938(2026)Approved November 19, 2021 (new molecular entity, standard review, orphan); expansion approved October 20, 2023; labeling supplement November 4, 2024.
- Original Voxzogo label (2021)(2021)First indication limited to children 5 and older with open epiphyses.
- FDA supplement approval letter, S-002 (2023)(2023)Expansion to all pediatric patients; confirmatory final adult height study (PMR 4134-2).
- FDA GSRS substance record, vosoritide (UNII 7SE5582Q2P)(2026)CAS 1480724-61-5; 39-residue sequence; disulfide Cys23 to Cys39; no modifications listed.
- EMA, Voxzogo EPAR(2026)EU authorization August 26, 2021; now from 4 months of age; orphan designation January 24, 2013.
- Voxzogo EU summary of product characteristics(2026)Diagnosis confirmed by genetic testing; half-life 27.9 minutes; under-5 height Z-score difference 0.30; injection-site reactions 85%.
- BioMarin, Japan approval (June 21, 2022)(2022)Approved in Japan for children of all ages whose growth plates are not closed.
- Savarirayan R et al., Lancet 2020;396:684-692 (phase 3)(2020)Randomized, double-blind, placebo-controlled phase 3 in 121 children aged 5 to 14.
- Savarirayan R et al., Lancet Child and Adolescent Health 2024;8:40-50 (under 5)(2024)Phase 2 placebo-controlled trial in children aged 3 to 59 months.
- Dauber A et al., NEJM Evidence 2026 (CANOPY-HCH-3)(2026)Hypochondroplasia, 81 children randomized: growth 2.33 cm/year faster than placebo (95% CI 1.85 to 2.82).
- BioMarin, full-approval application accepted (July 13, 2026)(2026)FDA decision due February 28, 2027; more than 5,000 children treated in more than 50 countries.
- BioMarin, first quarter 2026 results(2026)Children who started after age 5: 10.60 cm taller after 6 years and 13.59 cm after 8 years than natural-history comparisons.
Beyond these, 153 records are indexed. Browse the records