For laboratory and research use only. Not for human consumption.
Hormonal

Vosoritide

Also known as: Voxzogo, BMN 111, C-type natriuretic peptide analog, CNP analog

FDA Approved
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Last reviewed

Key Facts: Vosoritide

Category
Hormonal
FDA Status
FDA Approved
Clinical Status
FDA Approved (accelerated approval, 2021) - Voxzogo, to increase linear growth in pediatric patients with achondroplasia with open epiphyses. Also approved in the EU, Japan and Australia. sNDA for full achondroplasia approval under review (PDUFA 28 February 2027); sNDA for hypochondroplasia submitted 2026. WADA: not on the 2026 Prohibited List.
Banned in Sport (WADA)
Not on the WADA Prohibited List (2026).
Administration
Subcutaneous injection once daily (thighs, abdomen, buttocks, upper arms), after food and 240-300 mL of fluid
Typical Dose
About 15 mcg/kg once daily (label weight bands)
Frequency
Once daily
Duration
Daily until growth plates close, then permanently stopped
Also Known As
Voxzogo, BMN 111, C-type natriuretic peptide analog, CNP analog

How does Vosoritide work?

Achondroplasia comes from a gain-of-function mutation in FGFR3, a receptor that acts as a brake on cartilage cells in the growth plate. Vosoritide binds natriuretic peptide receptor B (NPR-B) and blocks FGFR3's downstream signal at the MAPK (RAF-1, ERK1/2) step. That releases the brake, so chondrocytes multiply and mature and bones lengthen. Native CNP is broken down in minutes; vosoritide is a 39-amino-acid analog built to resist neprilysin, though its half-life is still short (about 21 to 28 minutes per the label), which is why it is injected daily. Because natriuretic peptides relax blood vessels, a transient drop in blood pressure shortly after injection is the expected side effect.

What does the research show?

The pivotal phase 3 trial (Savarirayan and colleagues, Lancet 2020) randomized 121 children aged 5 and up to 15 mcg/kg daily or placebo for 52 weeks. Annualized growth velocity improved by 1.57 cm per year over placebo (95% CI 1.22 to 1.93). Injection site reactions were very common in both arms, and transient blood pressure drops occurred in 13% on vosoritide vs 5% on placebo. The label later expanded to younger children, based on a study in which infants and toddlers received the drug. Approval is accelerated, based on growth velocity, so long-term benefit is still being verified: BioMarin has an sNDA under FDA review for full approval in achondroplasia with adult height and body proportion data, with a target action date of 28 February 2027. In hypochondroplasia, the phase 3 CANOPY-HCH-3 trial (2026) met its endpoint with a 2.33 cm per year gain over placebo, was published in NEJM Evidence, and an sNDA has been submitted; that use is not approved yet. Competition arrived in February 2026, when the FDA granted accelerated approval to once-weekly navepegritide (Yuviwel, Ascendis) for children 2 and older. Honest take: a real, mechanism-based drug with consistent growth gains, but the size of the final adult height benefit and its effect on complications like spinal stenosis are still open questions.

Trial Progress:FDA Approved
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FDA

FDA Approval Studies

This peptide is FDA approved. Below are the key clinical trials that supported its approval.

Phase 3 vosoritide in achondroplasia (Study 1)

BioMarin Pharmaceutical • Phase 3

Approved 2021
Participants:

121

Duration:

52 weeks

Primary Endpoint:

Change in annualized growth velocity

Key Results:
  • ✓Growth velocity +1.57 cm/year vs placebo (95% CI 1.22 to 1.93)
  • ✓Baseline growth velocity about 4 cm/year in both arms
  • ✓Transient blood pressure drops in 13% vs 5% on placebo
View Study

What doses have been studied?

FDA Approved·Accelerated FDA approval on growth velocity from a placebo-controlled phase 3 trial; adult height benefit still being confirmed

Typical Dosingⓘ

Community experience

Common Dose

About 15 mcg/kg once daily (label weight bands)

Range

0.096-0.8 mg per day depending on body weight

Frequency

Once daily

Dose follows the label's weight table and is updated as the child grows. Kids should eat and drink 240-300 mL of fluid in the hour before injection to blunt blood pressure drops. It is stopped for good once growth plates close. There is no adult use.

Research Dosingⓘ

Scientific studies

FDA-approved labeled dosing (prescription only, pediatric, weight-based)

Duration

Daily until growth plates close, then permanently stopped

Administration

Subcutaneous injection once daily (thighs, abdomen, buttocks, upper arms), after food and 240-300 mL of fluid

When and how is Vosoritide taken?

Best Time to Take

Same time each day, about an hour after a meal and fluids

Once daily, roughly the same time every day

Food Recommendation

Take with food

Why This Timing?

The label requires adequate food and 240-300 mL of fluid in the hour before injection to reduce the risk of low blood pressure, which peaks around 30 minutes after dosing.

What are the side effects of Vosoritide?

Not everyone experiences these effects. Individual responses vary based on dosage, duration, and personal factors.

  • ●Injection site reactions (redness, swelling, hives) in most patients
  • ●Transient low blood pressure, dizziness, usually within 30 minutes of injection
  • ●Vomiting
  • ●Rash
  • ●Joint pain
  • ●Gastroenteritis
  • ●Fatigue
  • ●Not recommended with eGFR below 60

References

Research This Peptide Further

Frequently Asked Questions

What does Vosoritide do?

Vosoritide is a modified version of C-type natriuretic peptide (CNP), a growth plate signal, and the first drug approved to target the root cause of achondroplasia, the most common form of dwarfism. Sold as Voxzogo by BioMarin, it got FDA accelerated approval in 2021 and is now labeled for children with achondroplasia and open growth plates. It adds about 1.5 cm of growth per year compared with placebo. Whether that adds up to meaningfully taller adults and better health is still being confirmed, and it is useless once growth plates close.

How does Vosoritide work?

Achondroplasia comes from a gain-of-function mutation in FGFR3, a receptor that acts as a brake on cartilage cells in the growth plate. Vosoritide binds natriuretic peptide receptor B (NPR-B) and blocks FGFR3's downstream signal at the MAPK (RAF-1, ERK1/2) step. That releases the brake, so chondrocytes multiply and mature and bones lengthen. Native CNP is broken down in minutes; vosoritide is a 39-amino-acid analog built to resist neprilysin, though its half-life is still short (about 21 to 28 minutes per the label), which is why it is injected daily. Because natriuretic peptides relax blood vessels, a transient drop in blood pressure shortly after injection is the expected side effect.

Is Vosoritide FDA approved?

Yes, Vosoritide is FDA approved. FDA Approved (accelerated approval, 2021) - Voxzogo, to increase linear growth in pediatric patients with achondroplasia with open epiphyses. Also approved in the EU, Japan and Australia. sNDA for full achondroplasia approval under review (PDUFA 28 February 2027); sNDA for hypochondroplasia submitted 2026. WADA: not on the 2026 Prohibited List.

Is Vosoritide banned in sport?

Not on the WADA Prohibited List (2026).

What are the side effects of Vosoritide?

Reported side effects include: Injection site reactions (redness, swelling, hives) in most patients, Transient low blood pressure, dizziness, usually within 30 minutes of injection, Vomiting, Rash, Joint pain. Individual responses vary based on dosage, duration, and personal health factors.

What is the typical dose of Vosoritide?

Community-reported common dose: About 15 mcg/kg once daily (label weight bands) (Once daily). Range: 0.096-0.8 mg per day depending on body weight. Administration: Subcutaneous injection once daily (thighs, abdomen, buttocks, upper arms), after food and 240-300 mL of fluid. Prescription drug. Dose set by prescriber per label. Not medical advice.

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Hormonal

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