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VOXZOGO is FDA-approved to increase linear growth in children of all ages with achondroplasia and open growth plates1

Studied in children aged 4 months to <18 years1-4

*Duration of ≥6 months if aged ≥3 months at study entry; duration of ≥3 months if aged <3 months at study entry.3

†Participants completed at least 3 or 6 months of an observational run-in growth study (either in CANOPY ACH-OS [Study 901] or CANOPY ACH-2I [Study 206]) to establish their baseline annualized growth velocity.4,5

Data continue to be collected in ongoing clinical trials8

Children with achondroplasia on VOXZOGO can grow faster than without treatment1,9

Canopy ACH-3 (Study 301): Phase 3 pivotal trial in children aged 5 to 15 years (N=121)2,5

Mean (±SD) AVG at week 529

Study Design1,5,9

4 years of data were collected from VOXZOGO-treated patients aged 5 to 15 years across the CANOPY ACH-3 (Study 301) pivotal trial and CANOPY ACH-EXT (Study 302), the open-label extension study evaluating the long-term efficacy and safety profile of VOXZOGO.

Primary Endpoint1
Change from baseline in AGV at Week 52 compared to placebo. AGV is a measure of linear growth expressed as the change in height or length units over 1 year.


AGV, annualized growth velocity; CI, confidence interval; LS, least squares; SD, standard deviation.


*Improvement in AGV was consistent across all predefined subgroups, including sex, age, and Tanner stage.1
†All randomized subjects. 2 patients in the VOXZOGO group discontinued from the study before Week 52; the values for these 2 patients were imputed assuming baseline growth rate for the period with missing data.1
‡Difference in LS mean change from baseline. LS means were estimated from the ANCOVA (analysis of covariance) model, which included treatment, stratum defined by sex and Tanner stage, baseline age, baseline AGV, and baseline height Z-score.1

Additional preliminary data continue to be collected

VOXZOGO is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).1

The following information is being provided to inform healthcare providers on the ongoing assessment and experience of patients in the clinical trial program.

These data are not included in the US Prescribing Information and do not establish a clinical benefit or conclusions on efficacy. The analyses are preliminary and exploratory and should be interpreted cautiously.

Impact on final adult height has not been established and continues to be evaluated as studies are ongoing.

AGV by age in VOXZOGO-treated children was compared to a separate untreated control group10

Mean female AGV (±SD) by age10*

Mean male AGV (±SD) by age10*

Data Limitations

  • The VOXZOGO-treated and untreated groups are from different studies. Comparison of inter-study results should be interpreted with caution due to potential differences in study design and methods.10
  • The effect of VOXZOGO on final adult height has not been established and continues to be assessed. These preliminary results are not in the US label, reflect a cross-sectional mix of treatment durations, are descriptive, and may result from chance.10

Study Design
As a primary endpoint of CANOPY ACH-EXT (Study 302), AGV was calculated by age and sex in VOXZOGO-treated children (full analysis set; age 5 to <18 years; N=119) and plotted against the AGV of age- and sex-matched untreated children with ACH from an external ACH natural history study (CLARITY Study). For the VOXZOGO-treated children, AGV was derived for height assessments 12 ± 3 months apart and linked to a specific age integer considering the age at the midpoint of the 12-month interval.5,10

The mean (SD) VOXZOGO treatment duration at data cutoff was 4.0 (0.8) years (min: 1.7 years, max: 6.2 years).10

ACH, achondroplasia; AGV, annualized growth velocity; EXT, extension; SD, standard deviation.

*Adapted from Savarirayan R, et al. Med. 2025;6(5):100566.10

Backed by the longest clinical trial program in achondroplasia8

Body proportionality was measured over 5 years in a subset of children treated with VOXZOGO10

CANOPY ACH-3 (Study 301): After 1 year in the pivotal trial, the secondary endpoint of LS mean change from baseline in upper-to-lower body segment ratio was -0.02 in the placebo group (n=61) and -0.03 in the VOXZOGO group (n=58). The difference in LS mean change from baseline was -0.01 (95% CI: -0.05, 0.02; P=0.5).1,5,11

Mean (±SE) upper-to-lower body segment ratio10*

5-year Data Limitations

  • Due to potential differences in study design, care should be taken when interpreting the inter-trial comparison of the VOXZOGO-treated and untreated groups.10
  • The effect of VOXZOGO on final adult height and proportionality has not been established and continues to be assessed. These preliminary results are not in the US label, are not statistically powered, should be cautiously interpreted, and may result from chance.10

Study Design
As an exploratory endpoint of CANOPY ACH-EXT (Study 302), body proportionality was measured by upper-to-lower body segment ratio in a subset of VOXZOGO-treated children with assessments at age <11 (girls) or <12 years (boys); all children were age 5 to <18 years. Assessments beyond these ages are excluded from analysis.5,10,12†

Upper-to-lower body segment ratio of age-matched untreated children with ACH were also measured in a separate untreated control group; the untreated children were from CANOPY ACH-OS (Study 901) and the placebo arm of CANOPY ACH-3 (Study 301).5,10,12

ACH, achondroplasia; CI, confidence interval; LS, least squares; SE, standard error.

*Adapted from Savarirayan R, et al. Med. 2025;6(5):100566.10
†In average-stature children, average upper-to-lower body segment ratio is 1.7 at birth and decreases to 1.0 at 10 years old. In ACH, the ratio never reaches 1.0 but still declines from birth up to ~age 10 years in girls and 11 years in boys.10

References:

  1. VOXZOGO [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2024.
  2. Savarirayan R, Tofts L, Irving M, et al. Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial. Lancet. 2020;396(10252):684-692.
  3. Savarirayan R, Tofts L, Irving M, et al. Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial. Lancet. 2020;396(10252):684-692. Supplementary Appendix.
  4. Savarirayan R, Wilcox WR, Harmatz P, et al. Vosoritide therapy in children with achondroplasia aged 3-59 months: a multinational, randomised, double-blind, placebo-controlled, phase 2 trial. Lancet Child Adolesc Health. 2024;8(1):40-50.
  5. Data on file [1]. BioMarin Pharmaceutical Inc; 2025.
  6. ClinicalTrials.gov. Identifier: NCT03989947. Accessed April 23, 2025. https://www.clinicaltrials.gov/study/NCT03989947
  7. Savarirayan R, Tofts L, Irving M, et al. Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study. Genet Med. 2021;23(12):2443-2447.
  8. ClinicalTrials.gov. Search: Achondroplasia, BioMarin Pharmaceutical. Accessed April 23, 2025. https://www.clinicaltrials.gov/search?cond=Achondroplasia&term=BioMarin%20Pharmaceutical&viewType=Table
  9. Data on file [2]. BioMarin Pharmaceutical Inc; 2025.
  10. Savarirayan R, Irving M, Wilcox WR, et al. Sustained growth-promoting effects of vosoritide in children with achondroplasia from an ongoing phase 3 extension study. Med. 2025;6(5):100566.
  11. ClinicalTrials.gov. Identifier: NCT03197766. Accessed April 23, 2025. https://www.clinicaltrials.gov/study/NCT03197766
  12. Savarirayan R, Irving M, Wilcox WR, et al. Sustained growth-promoting effects of vosoritide in children with achondroplasia from an ongoing phase 3 extension study. Med. 2024;30:100566. Supplementary Appendix.
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INDICATION AND IMPORTANT SAFETY INFORMATION

Warnings and Precautions for Risk of Low Blood Pressure

Transient decreases in blood pressure were observed in clinical studies. Patients with significant cardiac or vascular disease and patients on anti-hypertensive medicinal products were excluded from participation in VOXZOGO clinical trials. To reduce the risk of a decrease in blood pressure and associated symptoms (dizziness, fatigue, and/or nausea), patients should be well hydrated, have adequate food intake, and drink approximately 8-10 ounces of fluid in the hour prior to VOXZOGO administration.

In a 52-week, randomized, double-blind, placebo-controlled trial in 121 subjects with achondroplasia, subjects aged from 5.1 to 14.9 years, (Study 1) eight (13%) of 60 patients treated with VOXZOGO had a total of 11 events of transient decrease in blood pressure, compared to 3 (5%) of 61 patients on placebo, over a 52-week treatment period. The median time to onset from injection was 31 (18 to 120) minutes, with resolution within 31 (5 to 90) minutes in VOXZOGO-treated subjects. Two out of 60 (3%) VOXZOGO-treated patients each had one symptomatic episode of decreased blood pressure with vomiting and/or dizziness compared to 0 of 61 (0%) patients on placebo.

Adverse Reactions:
Adverse reactions that occurred in ≥5% of patients treated with VOXZOGO and at a rate greater than that of placebo in the phase 3 study are injection site reactions (including erythema, swelling, urticaria, pain, bruising, pruritus, hemorrhage, discoloration, and induration), vomiting, arthralgia, decrease in blood pressure, gastroenteritis, diarrhea, dizziness, ear pain, influenza, fatigue, seasonal allergy, and dry skin. VOXZOGO-treated patients had an increase in alkaline phosphatase levels (17%), and was noted as a laboratory abnormality.

Injection site reactions:In Study 1, injection site reactions occurred in 51 (85%) subjects receiving VOXZOGO and 50 (82%) subjects receiving placebo over a 52-week period of treatment. Patients receiving VOXZOGO experienced a total of 6983 events of injection site reactions, while patients receiving placebo experienced a total of 1776 events of injection site reactions, over a 52-week period, representing 120.4 events per patient/year exposure and 29.2 events per patient/year exposure, respectively. Two patients in the VOXZOGO arm discontinued treatment due to adverse events of pain and anxiety with injections.

Pediatric Patients 0 to <5 Years:The safety of VOXZOGO in pediatric patients 0 to <5 years with achondroplasia was evaluated in a 52-week randomized, double-blind, placebo-controlled study (Study 2). In this study, 64 patients from birth to <5 years of age were randomized to receive either a daily vosoritide dose with similar exposure to that characterized to be safe and effective in children with ACH aged ≥5 years old, or placebo. An additional 11 patients received open-label treatment as part of this study. The most common adverse reactions (>10%) reported in pediatric patients 0 to <5 years were injection site reactions (86%) and rash (28%). The overall safety profile of VOXZOGO in pediatric patients 0 to <5 years was similar to that seen in older pediatric patients.

Administration and Monitoring:
VOXZOGO is administered as a daily subcutaneous injection. Prior to use, instruct caregivers on proper preparation and administration of VOXZOGO, and ensure caregivers have demonstrated the ability to perform a subcutaneous injection.

Monitor and assess patient body weight, growth, and physical development regularly every 3-6 months. Adjust dosage according to the patient’s actual body weight. Permanently discontinue treatment with VOXZOGO upon confirmation of no further growth potential, indicated by closure of epiphyses.

Special Populations:

  • There are no available data on the use of VOXZOGO in pregnant women, or data on the presence of VOXZOGO in human milk, the effects on the breastfed infant, or the effects on milk production.
  • The influence of renal impairment on the pharmacokinetics of VOXZOGO has not been evaluated. No dosage adjustment is needed for patients with eGFR ≥60 mL/min/1.73 m2. VOXZOGO is not recommended for patients with eGFR <60 mL/min/1.73 m2.

You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to BioMarin at 1-866-906-6100.

Please see additional safety information in the full Prescribing Information.

VOXZOGO® (vosoritide) is indicated to increase linear growth in pediatric patients with achondroplasia and open growth plates.

  • This indication is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).