This website is intended for Healthcare Professionals practicing in the U.S.
Search

Expanding possibilities
with VOXZOGO

For children with achondroplasia1,2

See the Proof

Sarah and her daughter Corrina, 20 months old, on VOXZOGO since 8 months old

The only FDA-approved targeted therapy indicated from birth to increase linear growth in children with achondroplasia until growth plates close.1

Sustained
Growth

Linear growth was maintained over baseline in children aged 5 to 15 years treated with VOXZOGO over 4 years1,3

Established History

Over 10 years of clinical trial experience in achondroplasia and 3,800+ infants and children prescribed worldwide1,4,5

Widespread Coverage

97% of insured patients have secured coverage and 96% of eligible families with commercial insurance paid $0 out of pocket6,7*†

International consensus guidelines support early initiation of VOXZOGO2

Learn More

BioMarin provided funding for the International consensus guidelines on the implementation and monitoring of vosoritide therapy in individuals with achondroplasia, including honoraria to participants. BioMarin was not involved in the selection of the guidelines development group, defining the guidelines scope, the voting process, analysis of the results, or preparation of the submitted manuscript. Please see Acknowledgements section of publication for additional detail.2

Connect With a BioMarin Representative

Register to have a BioMarin representative contact you.

Contact Us

Hear from families of children who have achondroplasia

These children are receiving VOXZOGO as part of a clinical trial.

Play

Eve, 3 years old

Play

Shawn, 8 years old

Play

Miller, 13 years old

Play

Annika, 14 years old

Meet the families who have experienced the impact of VOXZOGO

View Patient Stories 

*BioMarin RareConnections™ Data on File. VOXZOGO patients included are eligible VOXZOGO patients who have enrolled in BioMarin RareConnections and are on commercial therapy.6,7
†Terms and Conditions apply. Valid only for patients with commercial prescription insurance coverage who have a valid prescription for an FDA-approved indication and who meet additional eligibility criteria. Not valid for prescriptions reimbursed, in whole or in part, by any federal, state, or government-funded insurance programs (for example, Medicare, Medicare Advantage, Medigap, Medicaid, VA, DoD, or TRICARE) or where prohibited by law or by the patient’s health insurance provider. If at any time a patient begins receiving prescription drug coverage under any federal, state, or government-funded healthcare program, the patient will no longer be able to use the program and patient must notify BioMarin RareConnections at 1-866-906-6100 to stop participation. Patients residing in or receiving treatment in certain states may not be eligible for some or all of the program elements. Patients may not seek reimbursement for value received from the program from any third-party payers. Additional restrictions may apply. Offer subject to change or discontinuance without notice. This assistance offer is not health insurance. See BioMarin-copay-terms.com for full Terms and Conditions.

References:

  1. VOXZOGO [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2024.
  2. Savarirayan R, Hoover-Fong J, Ozono K, et al. International consensus guidelines on the implementation and monitoring of vosoritide therapy in individuals with achondroplasia. Nat Rev Endocrinol. 2025;21(5):314-324.
  3. Data on file [1]. BioMarin Pharmaceutical Inc; 2025.
  4. ClinicalTrials.gov. Search: Achondroplasia, BioMarin Pharmaceutical. Accessed April 23, 2025. https://www.clinicaltrials.gov/search?cond=Achondroplasia&term=BioMarin%20Pharmaceutical&viewType=Table
  5. Data on file[2]. BioMarin Pharmaceutical Inc; 2025.
  6. Data on file [3]. BioMarin Pharmaceutical Inc; 2023.
  7. Data on file [4]. BioMarin Pharmaceutical Inc; 2023.

Contact Us

INDICATION AND IMPORTANT SAFETY INFORMATION

Warnings and Precautions for Risk of Low Blood Pressure

Transient decreases in blood pressure were observed in clinical studies. Patients with significant cardiac or vascular disease and patients on anti-hypertensive medicinal products were excluded from participation in VOXZOGO clinical trials. To reduce the risk of a decrease in blood pressure and associated symptoms (dizziness, fatigue, and/or nausea), patients should be well hydrated, have adequate food intake, and drink approximately 8-10 ounces of fluid in the hour prior to VOXZOGO administration.

In a 52-week, randomized, double-blind, placebo-controlled trial in 121 subjects with achondroplasia, subjects aged from 5.1 to 14.9 years, (Study 1) eight (13%) of 60 patients treated with VOXZOGO had a total of 11 events of transient decrease in blood pressure, compared to 3 (5%) of 61 patients on placebo, over a 52-week treatment period. The median time to onset from injection was 31 (18 to 120) minutes, with resolution within 31 (5 to 90) minutes in VOXZOGO-treated subjects. Two out of 60 (3%) VOXZOGO-treated patients each had one symptomatic episode of decreased blood pressure with vomiting and/or dizziness compared to 0 of 61 (0%) patients on placebo.

Adverse Reactions:
Adverse reactions that occurred in ≥5% of patients treated with VOXZOGO and at a rate greater than that of placebo in the phase 3 study are injection site reactions (including erythema, swelling, urticaria, pain, bruising, pruritus, hemorrhage, discoloration, and induration), vomiting, arthralgia, decrease in blood pressure, gastroenteritis, diarrhea, dizziness, ear pain, influenza, fatigue, seasonal allergy, and dry skin. VOXZOGO-treated patients had an increase in alkaline phosphatase levels (17%), and was noted as a laboratory abnormality.

Injection site reactions:In Study 1, injection site reactions occurred in 51 (85%) subjects receiving VOXZOGO and 50 (82%) subjects receiving placebo over a 52-week period of treatment. Patients receiving VOXZOGO experienced a total of 6983 events of injection site reactions, while patients receiving placebo experienced a total of 1776 events of injection site reactions, over a 52-week period, representing 120.4 events per patient/year exposure and 29.2 events per patient/year exposure, respectively. Two patients in the VOXZOGO arm discontinued treatment due to adverse events of pain and anxiety with injections.

Pediatric Patients 0 to <5 Years:The safety of VOXZOGO in pediatric patients 0 to <5 years with achondroplasia was evaluated in a 52-week randomized, double-blind, placebo-controlled study (Study 2). In this study, 64 patients from birth to <5 years of age were randomized to receive either a daily vosoritide dose with similar exposure to that characterized to be safe and effective in children with ACH aged ≥5 years old, or placebo. An additional 11 patients received open-label treatment as part of this study. The most common adverse reactions (>10%) reported in pediatric patients 0 to <5 years were injection site reactions (86%) and rash (28%). The overall safety profile of VOXZOGO in pediatric patients 0 to <5 years was similar to that seen in older pediatric patients.

Administration and Monitoring:
VOXZOGO is administered as a daily subcutaneous injection. Prior to use, instruct caregivers on proper preparation and administration of VOXZOGO, and ensure caregivers have demonstrated the ability to perform a subcutaneous injection.

Monitor and assess patient body weight, growth, and physical development regularly every 3-6 months. Adjust dosage according to the patient’s actual body weight. Permanently discontinue treatment with VOXZOGO upon confirmation of no further growth potential, indicated by closure of epiphyses.

Special Populations:

  • There are no available data on the use of VOXZOGO in pregnant women, or data on the presence of VOXZOGO in human milk, the effects on the breastfed infant, or the effects on milk production.
  • The influence of renal impairment on the pharmacokinetics of VOXZOGO has not been evaluated. No dosage adjustment is needed for patients with eGFR ≥60 mL/min/1.73 m2. VOXZOGO is not recommended for patients with eGFR <60 mL/min/1.73 m2.

You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to BioMarin at 1-866-906-6100.

Please see additional safety information in the full Prescribing Information.

VOXZOGO® (vosoritide) is indicated to increase linear growth in pediatric patients with achondroplasia and open growth plates.

  • This indication is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).