Evidence review: Open Peptide Project (verified against primary PubMed, ClinicalTrials.gov & FDA records) · Last checked 2026-10-27. ← Back to Evidence Library · Trial & Regulatory Tracker →

Vosoritide (Voxzogo): what the evidence says

Name: Vosoritide (Voxzogo)
Published: 2026-10-27

At a glance

What it is: a lab-made 39-amino-acid analogue of C-type natriuretic peptide (CNP) that counteracts the overactive FGFR3 signal slowing bone growth in achondroplasia. Daily injection. Made by BioMarin; FDA-approved Nov 2021 as Voxzogo. ^{R1, R2}

What the evidence says: in a 121-child phase 3 RCT, growth sped up by 1.57 cm per year vs placebo. ^R3 Extension data show the gain persisting over several years. ^R4 Approval is accelerated, based on growth speed; whether it raises final adult height is not yet known. ^R5

Biggest risks: temporary drops in blood pressure after injection, and injection-site reactions. ^{R5, R3}

Rating


Human Human evidence	Strong for growth velocity in achondroplasia (phase 3 RCT) ^R3 · Moderate for under-5s ^R6 · Limited in hypochondroplasia ^R7 · No human data in typical-height children or adults
Animal Animal / lab evidence	Not reviewed for this page
Regulatory	FDA-approved (NDA 214938, accelerated approval) · not on the 503A/503B lists or FDA's safety-risk page · not named on WADA's 2026 list ^{R1, R5, R8, R9, R10, R11}
Established human dose	Label, weight-based (see the FDA label). Study details lists what trials used

Evidence verdict: Faster growth in children with achondroplasia: Strong (FDA-approved under accelerated approval; phase 3 RCT) · Final adult height: unknown · Adults or height gain in typical children: No human data

This page reports what studies used. It is not dosing advice.

Legend: HUMAN data · ANIMAL / LAB data · UNKNOWN · UNVERIFIED: not yet confirmed against a primary source

About the evidence: BioMarin funded the pivotal trials. Annual growth velocity is a surrogate endpoint; adult height and health outcomes are still being followed. ^{R3, R5}

What is it?

CNP is the body's own signal that helps growth-plate cartilage grow. Vosoritide is a longer-lasting version. ^R2

Does it work?

Human Phase 3 (Savarirayan 2020): 121 children aged 5-14; annualized growth velocity +1.57 cm/year vs placebo over 52 weeks. ^{R3, R12}
Human Under 5 years (2024): height Z-score difference 0.25 (95% CI −0.02 to 0.53), not clearly significant. ^{R6, R13}
Human Extension (2025): about 5.75 cm more height over 3 years than untreated comparison children. ^{R4, R14}
Human Hypochondroplasia (phase 2): early growth-velocity data. ^{R7, R15}

Is it safe?

Human Label warning: low blood pressure; give with food and fluids. No contraindications listed. ^R5
Human Injection-site reactions were common in trials. ^R3 One child in the under-5 vosoritide group died of sudden infant death syndrome. ^R6

Gray-market "vosoritide": extra risks

A gray-market vial has no quality control and no guarantee of identity, strength or sterility. We test retail vials blind under the Open Peptide Standard; see /tests/. A passing test covers one batch on one date and does not mean the compound is safe. Giving it to children without open growth plates or without a diagnosis has never been studied.

Is it legal?

US (FDA): prescription only, for children with achondroplasia and open growth plates. ^{R5, R1}
Athletes: not named on WADA's 2026 list. ^R11

Study details

These are amounts researchers used, not recommendations.

Study	People / model	Route	Amount used	Follow-up	Reported	Citation
Savarirayan 2020 (phase 3)	121 children 5-14 with achondroplasia	SC daily	15 µg/kg	52 weeks	+1.57 cm/year vs placebo	PMID 32891212
Under-5 RCT 2024	Infants and children <5	SC daily	Age-based (per trial)	52 weeks	Z-score diff 0.25	PMID 37984383
Extension 2025	Children from earlier trials	SC daily	15 µg/kg	~3 years	+5.75 cm vs untreated	PMID 39740666

Full reference list (PubMed, CT.gov, FDA and WADA checked Oct 26, 2026)

^R1 Drugs@FDA, Voxzogo, BioMarin, approved Nov 19, 2021, application 214938 (openFDA drugsfda record checked Oct 26, 2026). https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=214938
^R2 Duggan S. Drugs. 2021. PMID 34694597
^R3 Savarirayan R, et al. Lancet. 2020. PMID 32891212
^R4 Savarirayan R, et al. Med. 2025. PMID 39740666
^R5 Voxzogo (vosoritide) for injection prescribing information, label published Nov 26, 2025 (DailyMed, checked Oct 26, 2026). https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=228e8560-04a4-4bb1-a81f-29531a9e4d27
^R6 Savarirayan R, et al. Lancet Child Adolesc Health. 2024. PMID 37984383
^R7 Dauber A, et al. EClinicalMedicine. 2024. PMID 38813446
^R8 FDA, 503A bulk drug substances categories list (updated May 14, 2026). https://www.fda.gov/media/94155/download
^R9 FDA, 503B bulk drug substances categories list (updated Mar 21, 2025). https://www.fda.gov/media/94164/download
^R10 FDA, Certain bulk drug substances for use in compounding that may present significant safety risks (content current Apr 22, 2026). https://www.fda.gov/drugs/human-drug-compounding/certain-bulk-drug-substances-use-compounding-may-present-significant-safety-risks
^R11 WADA. The 2026 Prohibited List, full list checked; compound not named. https://www.wada-ama.org/sites/default/files/2025-09/2026list_en_final_clean_september_2025.pdf
^R12 ClinicalTrials.gov NCT03197766, BioMarin, vosoritide phase 3 in achondroplasia ages 5-14, completed (CT.gov API v2, checked Oct 26, 2026). https://clinicaltrials.gov/study/NCT03197766
^R13 ClinicalTrials.gov NCT03583697, BioMarin, vosoritide in infants and young children, phase 2, completed (CT.gov API v2, checked Oct 26, 2026). https://clinicaltrials.gov/study/NCT03583697
^R14 ClinicalTrials.gov NCT03424018, BioMarin, open-label extension, phase 3 (CT.gov API v2, checked Oct 26, 2026). https://clinicaltrials.gov/study/NCT03424018
^R15 ClinicalTrials.gov NCT04219007, vosoritide in hypochondroplasia and other short-stature conditions, phase 2 (CT.gov API v2, checked Oct 26, 2026). https://clinicaltrials.gov/study/NCT04219007

Disclaimer

This page is for education and harm reduction, not medical advice or a recommendation to use Vosoritide (Voxzogo). It is a prescription drug for children with achondroplasia whose growth plates are open. Talk to your child's specialist, especially about low blood pressure. Report problems to FDA MedWatch (https://www.fda.gov/safety/medwatch).