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Bone & mineral

Vosoritide

Also called Voxzogo, BMN 111

Vosoritide is a lab-made version of a natural growth-plate signal called C-type natriuretic peptide (CNP). It is a daily injection that speeds up bone growth in children with achondroplasia, the most common form of dwarfism.

FDA-approvedApproved by the FDA for at least one use after large human trials.

All printable flashcards

Drawn from its published sequence: 39 residues, one bead each. Dashed arcs are ring bonds.

Overview

Vosoritide is a lab-made version of a natural growth-plate signal called C-type natriuretic peptide (CNP). It is a daily injection that speeds up bone growth in children with achondroplasia, the most common form of dwarfism.

How it works

Established: achondroplasia is caused by an overactive FGFR3 receptor that brakes growth-plate cartilage. Vosoritide binds the NPR-B receptor, which counteracts FGFR3's downstream MAPK signaling. This lets cartilage cells multiply and mature, so long bones grow faster. The two added amino acids make it resist breakdown, so it lasts long enough for once-daily dosing.

What research shows

  • Phase 3 RCT, 121 children aged 5-15, 52 weeks: annualized growth velocity 1.57 cm/year higher than placebo (95% CI 1.22-1.93) (FDA label).
  • The under-5 expansion was supported by phase 2 and long-term extension data, including pharmacokinetic data from age 4.5 months (FDA label; PES summary).
  • Whether faster growth leads to better final adult height or fewer complications has not yet been confirmed; this is why approval is accelerated (FDA label).

Doses studied in people

Study or labelDose usedSource
FDA label (Voxzogo), achondroplasia with open growth platesOnce-daily subcutaneous dose by body weight: e.g. 0.096 mg at 3 kg, 0.24 mg at 8-11 kg, 0.4 mg at 22-32 kg, up to 0.8 mg at ≥90 kg1

Dosing follows the weight table in the FDA label; not recommended when eGFR is below 60 mL/min/1.73 m².

Read this first: these are the amounts given to participants in published studies or written on official prescribing labels, reported for reference. They are not recommendations. Trial doses were chosen for specific patients under medical supervision, and a dose that is safe in one person can be harmful in another.

Weekly protocol

How the schedule looks week by week. Each chart says where it comes from: a prescribing label, a clinical trial, or a protocol commonly reported online.

Voxzogo label dosing

Prescribing label

One week

D1D2D3D4D5D6D7

Once daily · Subcutaneous injection

Dose by week

Weight-based: 0.096 mg (3 kg) up to 0.8 mg (90 kg or more)wk 1+
Course
Ongoing while growth plates are open
Note
Dose comes from the label's body-weight table (for example 0.24 mg at 8–11 kg, 0.4 mg at 22–32 kg). Not recommended if eGFR is below 60 mL/min/1.73 m².
Source
VOXZOGO (vosoritide) prescribing information (FDA, 2024)

Reference only, not a recommendation. Label and trial schedules were set for specific patients under medical supervision. Use the calculator only to understand the arithmetic.

Safety

  • Transient drops in blood pressure, with possible dizziness, fatigue or nausea; children should be well fed and hydrated before each dose (FDA label).
  • Common: injection-site redness (75% vs 69% placebo), swelling (62% vs 36%), vomiting (27% vs 20%), injection-site hives (25% vs 10%), decreased blood pressure (13% vs 5%); rash in 28% of children under 5 (FDA label).
  • Long-term effects on adult height and skeletal outcomes are still being studied (accelerated approval).

Report side effects of any drug or product to the FDA through MedWatch. For a possible poisoning in the US, call Poison Control at 1-800-222-1222.

Storage and stability

Refrigerate vials at 2-8°C; may be kept at room temperature for up to 90 days (do not return to the fridge); use within 3 hours of reconstitution; protect from light (FDA label).

General lab handling, not instructions for personal use. See Storage and stability.

History

Developed by BioMarin. The EU approved it in August 2021 and the FDA in November 2021 (accelerated approval) for children 5 and older with open growth plates. On October 20, 2023, the FDA expanded the indication to children of all ages with open growth plates. Continued approval depends on confirmatory data.

Common questions

Is vosoritide FDA-approved?

Yes. Voxzogo got accelerated approval in November 2021 for achondroplasia, the most common form of dwarfism, and was expanded in October 2023 to children of all ages with open growth plates 12.

Does vosoritide make children taller?

In a 52-week trial of 121 children aged 5 to 15, growth was 1.57 cm per year faster than placebo 1. Whether this leads to greater adult height is not yet confirmed 1.

Why is it an accelerated approval?

Approval was based on faster growth, and it is not yet confirmed that this leads to better adult height or fewer complications 1. Continued approval depends on confirmatory data 12.

What doses are on the FDA label?

The label sets a once-daily injection by body weight, for example 0.24 mg at 8 to 11 kg and up to 0.8 mg at 90 kg or more 1. This is label information, not advice.

What are the side effects?

Injection-site redness affected 75% vs 69% on placebo, and vomiting 27% vs 20% 1. Blood pressure can drop briefly, so children should be fed and hydrated before each dose 1.

How is vosoritide stored?

Vials are refrigerated at 2 to 8°C, may be kept at room temperature for up to 90 days, and are used within 3 hours of mixing 1.

Related compounds

Bone & mineralFDA-approvedFlashcard

Teriparatide

Forteo · rhPTH(1-34)

Teriparatide is a lab-made copy of the first 34 amino acids of human parathyroid hormone.

Sources

  1. VOXZOGO (vosoritide) prescribing information (2024) FDA label
  2. Pediatric Endocrine Society: Update to the Vosoritide (Voxzogo) indication in USA Regulatory
  3. KEGG DRUG: Vosoritide (D11190) Journal
  4. WADA 2026 Prohibited List Regulatory

Last reviewed 2026-10-25 · How we research · Found an error? See our corrections policy.