This website is intended for residents of the U.S. interested in achondroplasia or VOXZOGO

Results with VOXZOGO

Children with achondroplasia grew faster on VOXZOGO than without treatment

In a clinical trial of children aged 5 to 15 years, the impact of VOXZOGO on linear growth was measured over 1 year.

Growth rate before and after 1 year of treatment

Bar chart showing greater annual growth rate with VOXZOGO vs placebo after 1 year
Bar chart showing greater annual growth rate with VOXZOGO vs placebo after 1 year

Improvement in linear growth rate over baseline (from when treatment was started) was maintained over 4 years in an ongoing study.

At year 4, the rate of growth was 1.88 in/yr.

97% of children (58/60) stayed on VOXZOGO during the first year.

*The difference when accounting for group characteristics, including sex and Tanner stage, treatment, baseline age, baseline growth rate, and baseline height Z-score.
These results are from the long-term follow-up study of the same clinical trial (known as an open-label extension), which did not have a placebo group, and caregivers, patients, and doctors were aware that all children were given VOXZOGO.
One child discontinued due to injection pain, and one child discontinued due to fear of needles.

Annika at ages 6 and 14, illustrating growth progress before and after taking VOXZOGO
Annika at ages 6 and 14, illustrating growth progress before and after taking VOXZOGO

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The impact of VOXZOGO continues to be studied

VOXZOGO is approved under accelerated approval based on an improvement in growth rate after 1 year of treatment. Compared to untreated patients in the study, body proportionality did not worsen at 1 year.

Because achondroplasia is a condition that affects multiple aspects of physical development, additional studies on the impact of VOXZOGO over a longer period of time are ongoing, including its effects on final adult height and body proportionality.

Graphic comparing clinical trial results between 4 months to
Graphic comparing clinical trial results between 4 months to

*Visit clinicaltrials.gov for the complete list of the trials’ additional study measures (NCT03583697, NCT03989947, NCT03197766, NCT03424018).

10+ years of clinical trial experience in achondroplasia

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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.