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approvedGrowth & Recovery

Vosoritide

Also known as: Voxzogo, BMN 111, Modified CNP-39

Vosoritide (Voxzogo) is a modified analog of C-type natriuretic peptide (CNP) approved by the FDA in 2021 for the treatment of achondroplasia in pediatric patients 5 years and older with open growth plates. It is the first and only targeted therapy for the most common form of dwarfism, addressing the underlying FGFR3-mediated growth inhibition rather than just managing symptoms. Vosoritide represents a landmark in rare disease treatment, offering children with achondroplasia the opportunity for improved linear growth.

3 cited references·4 researched benefits

Quick Answer

Vosoritide (Voxzogo) is a C-type natriuretic peptide analog FDA-approved in 2021 as the first targeted therapy for achondroplasia, the most common form of dwarfism. Administered as a daily subcutaneous injection, it counteracts the overactive FGFR3 signaling that inhibits bone growth by stimulating NPR-B receptors in growth plate chondrocytes. Clinical trials demonstrated an increase of approximately 1.57 cm/year in annualized growth velocity compared to placebo in children aged 5 and older.

Key Facts

Mechanism
Vosoritide is a 39-amino acid peptide engineered from CNP-53 with modifications to resist neutral endopeptidase (NEP) degradation, extending its half-life. It binds to natriuretic peptide receptor B (NPR-B) on growth plate chondrocytes, activating intracellular cGMP signaling via guanylyl cyclase. This cGMP pathway directly inhibits the RAF-MEK-ERK (MAPK) cascade that is constitutively overactivated by the gain-of-function FGFR3 mutation in achondroplasia. By dampening FGFR3 downstream signaling, vosoritide restores more normal chondrocyte proliferation and differentiation in the growth plate, promoting endochondral ossification and linear bone growth.
Research Status
approved
Half-Life
~30 minutes
Molecular Formula
C₁₇₂H₂₈₁N₅₅O₅₅S₂
Primary Use
Growth & Recovery
Table of Contents

Benefits

  • Increased annualized growth velocity — phase 3 trial showed +1.57 cm/year gain vs placebo over 52 weeks in children with achondroplasiastrong
  • Sustained growth improvement — open-label extension data show maintained increased growth velocity over multiple years of treatmentstrong
  • Improved body proportions — increases in sitting height and arm span, not just standing height, suggest more proportionate skeletal growthmoderate
  • Potential to reduce need for surgical limb-lengthening procedures and other orthopedic interventionspreliminary

Dosage Protocols

RouteDosage RangeFrequencyNotes
Subcutaneous injection15 mcg/kgOnce dailyWeight-based dosing at 15 mcg/kg body weight. Maximum single dose not specified but patients up to ~60 kg studied. Administered in the abdomen, thigh, or upper arm. Patients should eat a snack and drink fluids before injection to reduce risk of hypotension. Injection should be given at approximately the same time each day.

Medical disclaimer

Dosage information is provided for educational reference only. Always follow your prescriber's instructions and consult a qualified healthcare provider before starting any peptide protocol.

Side Effects

  • Injection site reactions — erythema, swelling, and pain at the injection site in approximately 35-70% of patientscommon
  • Transient blood pressure decrease — mild, self-resolving drops in blood pressure within 15-90 minutes of injectioncommon
  • Nausea and vomiting — reported in ~25% of patients, typically mild and transient post-injectioncommon
  • Dizziness — related to transient hypotensive effects, typically resolves within 1-2 hourscommon
  • Severe hypotension or syncope — rare but possible, particularly with dehydration; patients should be well-hydrated before injectionserious

Frequently Asked Questions

How effective is vosoritide for achondroplasia?
In the pivotal phase 3 trial (Study 111-301), children aged 5-18 receiving vosoritide grew an average of 1.57 cm/year faster than those on placebo over 52 weeks. Open-label extension data over 3+ years show sustained increased growth velocity of approximately 1.4-1.6 cm/year above pre-treatment baseline. While vosoritide does not normalize height to typical ranges, it meaningfully improves growth trajectory and may reduce the disproportionality that contributes to medical complications in achondroplasia.
What is the cost of Voxzogo treatment?
Voxzogo has a list price of approximately $320,000 per year in the United States, making it one of the more expensive rare disease therapies. The cost is weight-based and may vary. BioMarin offers patient assistance programs and works with insurance companies to improve access. Most major commercial insurers and some Medicaid plans cover Voxzogo following the FDA approval, though prior authorization is typically required.
Can adults with achondroplasia benefit from vosoritide?
Vosoritide is only approved for pediatric patients with open growth plates, as its mechanism of action requires active growth plate chondrocytes responsive to CNP signaling. Once growth plates fuse (typically by age 14-18), vosoritide cannot increase height. Adults with achondroplasia do not benefit from this therapy for height. However, research into CNP analogs for other adult conditions involving fibrotic or inflammatory pathways is ongoing.
Is vosoritide a cure for achondroplasia?
No. Vosoritide is a targeted treatment, not a cure. It does not alter the underlying FGFR3 genetic mutation that causes achondroplasia. It works by counteracting the downstream effects of the overactive FGFR3 signaling pathway while growth plates remain open. Treatment must be continued daily for ongoing benefit. Once discontinued, growth rate returns to pre-treatment baseline. Gene therapy approaches are in early research stages as potential curative strategies.

References

  1. 1
    Vosoritide therapy in children with achondroplasia: a randomized, double-blind, placebo-controlled, phase 3 trial(2020)PubMed ↗
  2. 2
    Long-term efficacy and safety of vosoritide in children with achondroplasia: 2-year results from an open-label extension study(2022)PubMed ↗
  3. 3
    C-type natriuretic peptide analog as therapy for achondroplasia(2015)PubMed ↗

Latest Research

Last updated: 2026-02-19