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Learn more about achondroplasia and VOXZOGO1

Expand your collection of resources to learn why VOXZOGO has been chosen by the families of over 5,000 infants and children with achondroplasia around the world.1,2

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Meet Amrit Bhangoo, MD, pediatric endocrinologist

Watch as Dr. Bhangoo discusses the limited window for endochondral bone growth, the need for multidisciplinary care, and his thoughts on starting VOXZOGO early and staying on treatment until growth plates close.1,3-5

Resources to help guide you and your patients

Downloadable PDFs to start the conversation

VOXZOGO at a glance

A resource for caregivers to learn more about VOXZOGO.

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How to talk to your child about VOXZOGO

A guide to help caregivers discuss treatment with their child.

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Pediatrician’s guide to VOXZOGO

An overview of achondroplasia and VOXZOGO for pediatricians.

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Treatment with VOXZOGO

An in-depth look at VOXZOGO for healthcare professionals.

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Connect your patients to local care with the Achondroplasia Doctor Finder

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References:

  1. VOXZOGO [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2024.
  2. Data on file [1]. BioMarin Pharmaceutical Inc; 2025.
  3. Mackie EJ, Tatarczuch L, Mirams M. The skeleton: a multi-functional complex organ: the growth plate chondrocyte and endochondral ossification. 
J Endocrinol. 2011;211(2):109-121.
  4. Savarirayan R, Ireland P, Irving M, et al. International Consensus Statement on the diagnosis, multidisciplinary management and lifelong care of individuals with achondroplasia. Nat Rev Endocrinol. 2022;18(3):173-189.
  5. 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.
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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).