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Vosoritide

Aliases: Voxzogo

Last verified: 2026-09-23

Approved drugStrong evidencePeptidesMedium interest

The short version

A C-type natriuretic peptide (CNP) analogue — the first drug to increase bone growth in achondroplasia (the most common dwarfism) in children.

Identity & type

Molecular type
analog
Origin
A C-type natriuretic peptide (CNP) analogue — the first drug to increase bone growth in achondroplasia (the most common dwarfism) in children.

Mechanism of action

NPR-B agonism → suppression of hyperactive FGFR3 signaling → normalized chondrogenesis.

Dosing & routes

Official / clinical context

Approved drug — indications, dosing and warnings are defined by the official label. Weight-based SC daily while growth plates open.

Regulator-approved labeling and published study designs — never a recommendation.

Community-reported practice

Weight-based SC daily while growth plates open. Voxzogo: measurable extra growth (~1.5 cm/yr more) in children with achondroplasia; parents report good tolerability.

Unverified self-reports. Not medical advice. Not endorsement.

Expected effects (community)

Voxzogo: measurable extra growth (~1.5 cm/yr more) in children with achondroplasia; parents report good tolerability.

Protocol — official vs community

Official / label

Voxzogo, for achondroplasia in children with open epiphyses: weight-based SC once daily — 0.24 mg/kg for 15 to <60 kg, and a fixed 15 mg for ≥60 kg — at a consistent time each day, after a light meal.

  1. 01Day 1 (initiation): dose escalation in supervised steps (0.05 mg/kg → 0.1 mg/kg → final dose) to test tolerability
  2. 02After ≥12 months: may increase to 0.28 mg/kg in inadequate responders who tolerated therapy

Duration: Daily until growth plates close (end of puberty); phase 3 added ~1.57 cm/year of growth versus placebo.

CNP analogue that counteracts overactive FGFR3 signaling in the growth plate. Transient hypotension right after injection is the characteristic adverse effect — hence the light meal and consistent-timing instructions.

Community (anecdotal)

Structured protocol data for this entry is coming.

Human evidence

Phase III: +1.57 cm/year extra growth vs placebo.

Preclinical evidence

The preclinical phase was completed and submitted to regulators as part of registration; pharmacology and toxicology details are part of the official label.

Known risks

  • Injection-site reactions, transient hypotension.characterized

Teal: characterized in clinical/labeling contexts. Amber: theoretical or reported outside controlled settings.

Unknowns & evidence gaps

  • Real-world data outside controlled trials

Frequently asked questions

References