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The safety of VOXZOGO has been rigorously evaluated

Adverse reactions reported in the 1-year phase 3 study are consistent with those seen in the long-term safety study.1,2

adverse reactions that occured in less than 5 percent of patients treated with VOXZOGO and at a percentage greater than placebo over 1 year.

*Includes adverse reactions occurring more frequently in the VOXZOGO arm and with a risk difference of ≥5% (ie, difference of >2 subjects) between treatment arms.
†Injection site reactions occurring more frequently in VOXZOGO-treated subjects than placebo.
‡Includes the preferred terms: gastroenteritis and gastroenteritis, viral.
§Includes the preferred terms: dizziness, presyncope, procedural dizziness, and vertigo.
||Includes the preferred terms: fatigue, lethargy, and malaise.

97% of children given VOXZOGO (58/60) remained on treatment during the phase 3 pivotal clinical trial1,3

One patient discontinued due to pain and one discontinued due to anxiety with injections.

Overall safety profile in patients <5 years was similar to that seen in older pediatric patients1

  • The safety of VOXZOGO in patients <5 years was evaluated in a 52-week randomized, placebo controlled, double-blind study1
  • The most common adverse reactions (>10%) reported in pediatric patients <5 years were injection site reactions (86%) and rash (28%)1

Transient decreases in blood pressure1

In the phase 3 study of children aged 5 to 15 years, the median time to onset of events from injection was 31 minutes (range, 18-120 minutes)1

VOXZOGO safety data.
  • 2 of 60 VOXZOGO-treated patients (3%) each had one symptomatic episode of blood-pressure decrease with vomiting and/or dizziness, compared with 0 of 61 patients (0%) on placebo1
  • 8 of 60 patients (13%) taking VOXZOGO had a total of 11 events, compared with 3 of 61 patients (5%) on placebo, who had 3 events1,3,*
  • Changes in blood pressure were identified predominantly during periods of frequent vital signs monitoring at clinical visits after dosing over 1 year of treatment1
  • No clinically significant cardiovascular changes were observed
    • 14 of 60 VOXZOGO-treated patients (23%) reported post-dose decreases in systolic blood pressure <70 mm Hg (+2x age), compared with 15 of 61 (25% on placebo)3
    • 10 of 60 VOXZOGO-treated patients (17%) reported post-dose decreases in diastolic blood pressure <40 mm Hg, compared with 6 of 61 (10%) on placebo3

References:

  1. VOXZOGO [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2023.
  2. 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. doi:10.1038/s41436-021-01287-7
  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. doi:10.1016/S0140-6736(20)31541-5
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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).