This website is intended for residents of the U.S. interested in achondroplasia or VOXZOGO
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Achondroplasia affects more than height

Evelyn, 20 months old

Achondroplasia care begins with understanding

Whether you’re navigating a new diagnosis or you’re looking for additional resources along the way—learn more about the impact achondroplasia may have on your child’s health.

Bones grow for a limited time. In achondroplasia, endochondral bone growth is inhibited.

Inhibited Bone Growth

Inhibited bone growth can impact different aspects of physical development.

Impact on Growth

Specialized care near you

Connect with local doctors who have chosen to be part of the Achondroplasia Doctor Finder.

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“We knew there was a limited time frame on how much growth she really has, which helped us make our decision to treat early.”

– Julie, Eve’s mom

Deepen your understanding

Find additional information and resources about the impact of achondroplasia at the Inside Story.

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Approaching care with confidence— VOXZOGO treatment discussion guide for caregivers

Talking with your child’s doctor

Prepare for your child’s next appointment with help from this treatment discussion guide.

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Common questions about achondroplasia

What is achondroplasia?

Achondroplasia is the most common type of skeletal dysplasia, a rare genetic condition that can be inherited from parents or occur randomly. In achondroplasia, the growth of bones that develop from cartilage (called endochondral bones) is inhibited throughout the body due to an imbalance of cell signals.

How does achondroplasia affect my infant or child?

Achondroplasia can impact physical development throughout the body, including reduced and disproportionate growth. However, it does not affect mental cognition and, with proper care, individuals with achondroplasia can lead full lives.

What kind of care could my child with achondroplasia need?

In addition to their routine check-ups, your child may have unique needs that require additional consideration from their pediatrician or other specialists.

Discuss these needs with their pediatrician or find specialized care if necessary.

Is there treatment available for achondroplasia?

VOXZOGO is the first and only treatment FDA approved from birth for children with achondroplasia until growth plates close.

How can I connect with other parents or caregivers of a child with achondroplasia?

You can connect with a caregiver raising a child on VOXZOGO by signing up for the VOXZOGO Mentor Program.

You can also learn more about VOXZOGO from an experienced caregiver and medical professional by attending an educational event.

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.