This website is intended for residents of the U.S. interested in achondroplasia or VOXZOGO
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Frequently Asked Questions From Families Like Yours

Eve, 3, on VOXZOGO since 16 months old as part of a clinical trial

Find answers to questions that others have asked about VOXZOGO® (vosoritide).

How can VOXZOGO help my child?

VOXZOGO is indicated to increase linear growth in children of all ages with achondroplasia whose growth plates are still open. See the results in children receiving VOXZOGO in the clinical trial.

What should I know about VOXZOGO safety?

The safety profile of VOXZOGO has been studied over 3 years in multiple clinical trials. 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. However, these are not all the possible side effects of VOXZOGO.

To learn more, talk to your child’s doctor or read about possible side effects here.

How does VOXZOGO work?

Achondroplasia is caused by an imbalance between fibroblast growth factor receptor 3 (FGFR3) and C-type natriuretic peptide (CNP) signaling pathways. This imbalance leads to impaired bone growth. VOXZOGO acts like the body’s natural CNP to improve the balance and promote bone growth. Learn how VOXZOGO targets the imbalance to promote growth.

How do I get my child started with VOXZOGO?

Once you and your doctor decide that VOXZOGO is right for your child, you can follow these steps to get started or request to connect with a local BioMarin representative.

How do I get my child involved in their treatment routine?

Download the Talking to Your Child About VOXZOGO guide for guidance on how to get your child involved in treatment.

How do I know if my insurance covers VOXZOGO?

Once you and your doctor decide VOXZOGO is right for your child, BioMarin RareConnections™ will help you navigate the insurance process and identify financial assistance options you may be eligible for to access treatment. Learn how we help.

Are there support resources available to help my family while on treatment?

BioMarin is committed to providing product resources and support throughout your treatment. Your BioMarin RareConnnections Case Manager will provide personalized support to help you understand insurance and financial assistance options. Your Clinical Coordinator will provide one-on-one product education and injection training tailored to your family’s needs.

Find out more about our Patient Support Program.

Have additional questions?

Have a local BioMarin Representative connect with you.

Connect With a Rep

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.