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How VOXZOGO works

Support bone growth as early as birth

In people with achondroplasia, the FGFR3 cell signals are overactive, leading to inhibited bone growth throughout the body. VOXZOGO targets the cause of inhibited bone growth by mimicking the body’s natural cell signals to improve the balance of cell signaling.

Learn more about treatment with VOXZOGO.

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Unbalanced cell signaling showing overactive FGFR3 inhibiting bone growth outweighing CNP in achondroplasia without VOXZOGO

This graphic is an illustration of the cell signaling imbalance in achondroplasia and how VOXZOGO is designed to improve the balance. This graphic is not to scale and does not reflect a percentage growth.

CNP, C-type natriuretic peptide; FGFR3, fibroblast growth factor receptor 3.

Bones only grow for a limited time

VOXZOGO is the first and only treatment FDA approved from birth to increase linear growth*
for children with achondroplasia while they are still growing (before growth plates have closed).

*Linear growth, an increase in length or height, is one indicator of overall health and development.

Medical guidelines support starting VOXZOGO early, with treatment until bones stop growing.

BioMarin provided funding for the International consensus guidelines on the implementation and monitoring of vosoritide therapy in individuals with achondroplasia, including payment to participants. BioMarin was not involved in deciding the guidelines content, including who were involved in developing the guidelines. For details, please see the Acknowledgements section of the publication.

Young child with achondroplasia who is on VOXZOGO treatment, reaching into a kitchen drawer
Young child with achondroplasia who is on VOXZOGO treatment, reaching into a kitchen drawer

Ask your child’s doctor about starting VOXZOGO as early as possible
to help give them the most time possible to benefit from treatment.

Results with VOXZOGO

Backed by 10+ years of clinical trial experience in achondroplasia.

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Looking for more information about VOXZOGO?

Your local BioMarin representative can help you find answers.

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IMPORTANT SAFETY INFORMATION

What is the most important safety information about VOXZOGO?

  • VOXZOGO may cause serious side effects including a temporary decrease in blood pressure in some patients. To reduce the risk of a decrease in blood pressure and associated symptoms (dizziness, feeling tired, or nausea), patients should eat a meal and drink 8 to 10 ounces of fluid within 1 hour before receiving VOXZOGO.

What are the most common side effects of VOXZOGO?

  • The most common side effects of VOXZOGO include injection site reactions (including redness, itching, swelling, bruising, rash, hives, and injection site pain), high levels of blood alkaline phosphatase shown in blood tests, vomiting, joint pain, decreased blood pressure, and stomach ache. These are not all the possible side effects of VOXZOGO. Ask your healthcare provider for medical advice about side effects, and about any side effects that bother the patient or that do not go away.

How is VOXZOGO taken?

  • VOXZOGO is taken daily as an injection given under the skin, administered by a caregiver after a healthcare provider determines the caregiver is able to administer VOXZOGO. Do not try to inject VOXZOGO until you have been shown the right way by your healthcare provider. VOXZOGO is supplied with Instructions for Use that describe the steps for preparing, injecting, and disposing VOXZOGO. Caregivers should review the Instructions for Use for guidance and any time they receive a refill of VOXZOGO in case any changes have been made.
  • Inject VOXZOGO 1 time every day, at about the same time each day. If a dose of VOXZOGO is missed, it can be given within 12 hours from the missed dose. After 12 hours, skip the missed dose and administer the next daily dose as usual.
  • The dose of VOXZOGO is based on body weight. Your healthcare provider will adjust the dose based on changes in weight following regular check-ups.
  • Your healthcare provider will monitor the patient’s growth and tell you when to stop taking VOXZOGO if they determine the patient is no longer able to grow. Stop administering VOXZOGO if instructed by your healthcare provider.

What should you tell the doctor before or during taking VOXZOGO?

  • Tell your doctor about all of the patient’s medical conditions including
    • If the patient has heart disease (cardiac or vascular disease), or if the patient is on blood pressure medicine (anti-hypertensive medicine).
    • If the patient has kidney problems or renal impairment.
    • If the patient is pregnant or plans to become pregnant. It is not known if VOXZOGO will harm the unborn baby.
    • If the patient is breastfeeding or plans to breastfeed. It is not known if VOXZOGO passes into breast milk.
  • Tell your doctor about all of the medicines the patient takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

You may report side effects to BioMarin at 1-866-906-6100. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

Please see additional safety information in the full Prescribing Information and Patient Information.

What is VOXZOGO used for?

  • VOXZOGO is a prescription medicine used to increase linear growth in children with achondroplasia and open growth plates (epiphyses).
  • VOXZOGO is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials.