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The impact of inhibited bone growth

In achondroplasia, endochondral bone growth is inhibited throughout the body

Bones grow for a limited time. In achondroplasia, the growth of bones that develop from cartilage (called endochondral bones) is inhibited. This can impact physical development, including reduced and disproportionate growth.

See how physical development is tracked for children with achondroplasia.

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Endochondral bone growth is inhibited in achondroplasia—that’s over 90% of the body’s bones.

Endochondral bones highlighted in the human skeleton—in achondroplasia, endochondral bone growth is inhibited, affecting over 90% of the body's bones

The most rapid growth occurs in the first 2 years of life


By 2 years old, children with achondroplasia reach more than half of their adult height*

Bar chart showing endochondral bone growth timeline from birth to age 20, with greatest growth in the first 2 years of life
Bar chart showing endochondral bone growth timeline from birth to age 20, with greatest growth in the first 2 years of life

*Based on stature-for-age data (birth to 18 years old) from CLARITY, an achondroplasia natural history study comprising measurements from 549 males and 502 females with achondroplasia.

Bone growth is inhibited by imbalanced cell signals

Endochondral bone growth occurs in areas of the bones called growth plates and requires a balance of two cell signals—FGFR3 (which slows growth) and CNP (which promotes growth). In achondroplasia, the FGFR3 signals are overactive, causing an imbalance that inhibits bone growth.

Diagram showing FGFR3 and CNP signals in balance, resulting in bone growth in a person without achondroplasia

This graphic is an illustration of the cell signaling imbalance in achondroplasia and is not to scale.
CNP, C-type natriuretic peptide; FGFR3, fibroblast growth factor receptor 3.

See how VOXZOGO works

Treatment to help promote bone growth from birth.

Learn More
Achondroplasia growth curve chart— track your child's physical development

Tracking your child’s growth

Growth charts specific to children with achondroplasia are important when tracking physical development.

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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.