Frequently Asked Questions

Instructions for Use Important Safety Information Prescribing Information Patient Information Healthcare Provider Website

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Frequently Asked Questions

Find quick answers to common questions

How can VOXZOGO help my child?

VOXZOGO is indicated to increase linear growth in children with achondroplasia aged 5 years and up 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 1 year of treatment compared with placebo. Talk to your child’s doctor about the potential side effects.

Learn about the side effects from the clinical trial.

How does VOXZOGO work?

Achondroplasia affects endochondral bone growth—this is bone growth that replaces cartilage with bone tissue as your child grows. A balance between fibroblast growth factor receptor 3 (FGFR3) and C-type natriuretic peptide (CNP) signaling pathways occurs within the cells in the growth plate. FGFR3 slows bone growth while CNP promotes bone growth.

Achondroplasia is caused by an imbalance between FGFR3 and CNP signaling pathways and leads to impaired bone growth. Learn how VOXZOGO targets the imbalance to promote growth.

How do I get my child started with VOXZOGO?

Once your doctor decides 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 can I find a local doctor for my child?

If you are interested in speaking with a healthcare provider who cares for children with achondroplasia, use the Achondroplasia Doctor Finder to find someone in your area.

How will I know how to administer VOXZOGO?

A healthcare provider will train you on how to give your child VOXZOGO at home. A BioMarin Clinical Coordinator is also available for injection training support and ongoing education.

In addition, we have resources, like videos and pamphlets, to help guide you throughout treatment.

Watch a step-by-step training video for how to inject VOXZOGO.

What is the age cutoff for starting my child on VOXZOGO?

VOXZOGO is indicated for increasing linear growth in children with achondroplasia aged 5 years and up with open growth plates. As long as growth plates are open, there is an opportunity for bone growth. In the clinical trial, some children who received VOXZOGO were up to 15 years old. Talk to your doctor who can determine potential for growth.

How long will my child take VOXZOGO?

VOXZOGO helps to improve annualized growth velocity in children with achondroplasia aged 5 years and older while their growth plates are open.

Regular checkups with your child’s prescribing doctor will be able to determine when their growth potential has been reached, and your child’s doctor will stop treatment when growth plates are closed. In the clinical trial, some children receiving VOXZOGO were up to 15 years old.

How do I get my child involved?

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 reach out to discuss your insurance coverage and help you navigate your financial options for gaining access to treatment. Learn how we help.

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

BioMarin is committed to providing resources and support throughout your treatment. In addition to understanding insurance and financial assistance, you’ll also have a Clinical Coordinator who will provide one-to-one product education and injection training tailored to your family’s needs.

Find out more about our Patient Support Program.

Watch Miller’s and Annika’s videos and learn about the impact of VOXZOGO on their daily lives.

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), 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 who are 5 years of age and older with open growth plates (epiphyses).
It is not known if VOXZOGO is safe and effective in children with achondroplasia under 5 years of age.
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.