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VOXZOGO®▼ (vosoritide)

Now listed on Pharmaceutical Benefits Scheme (PBS) from 1 May 2023.

Click here for PBS criteria

What is VOXZOGO®▼

▼This medicinal product is subject to additional monitoring in Australia. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse events at

VOXZOGO® (vosoritide) is indicated for the treatment of achondroplasia in patients 2 years of age and older whose epiphyses are not closed. The diagnosis of achondroplasia should be confirmed by appropriate genetic testing.

In achondroplasia, the endogenous C-type natriuretic peptide (CNP) cannot adequately regulate the overactive fibroblast growth factor receptor 3 (FGFR3) signaling.1,2

This affects the proliferation and terminal differentiation of growth plate chondrocytes, resulting in impaired endochondral bone growth and linear growth.3 VOXZOGO®, a biological CNP analog, acts as a positive regulator of endochondral bone growth, targeting overactive FGFR3 signaling and promoting chondrocyte proliferation and differentiation.

A girl dancing happily

VOXZOGO® mode of action

Watch this video to see how VOXZOGO® works to target overactive FGFR3 signaling.

Individuals with achondroplasia have overactive fibroblast growth factor receptor 3 (FGFR3) signaling1-3

Endogenous CNP levels cannot adequately regulate overactive FGFR3 signaling, resulting in impaired endochondral bone growth.1-2

VOXZOGO®, a CNP analog, works to increase endochondral bone growth

VOXZOGO® targets overactive FGFR3 signaling and promotes chondrocyte proliferation and differentiation.4

CNP: C-type natriuretic peptide; FGFR3: fibroblast growth factor receptor 3; MAPK: mitogen-activated protein kinase; NPR-B: natriuretic peptide receptor-B; RAF: rapidly accelerated fibrosarcoma proto-oncogene, serine/threonine kinase; RAS: rat sarcoma virus kinase.

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Clinical Data

VOXZOGO® (vosoritide) has been studied in 60 individuals aged 5-15 years over 1 year. The efficacy of once-daily, subcutaneous therapy demonstrated in the VOXZOGO® Phase 3 clinical trial.




Study Overview: Clinical Trial

The safety and efficacy of VOXZOGO® (vosoritide) were evaluated in patients with achondroplasia aged 5 to 15 years1,2

Clinical study overview1,2

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

VOXZOGO® Efficacy Data

The safety and efficacy of VOXZOGO® (vosoritide) were evaluated in patients with achondroplasia aged 5 to <18 years1,2

Statistically significant improvement in annualized growth velocity (AGV) was seen in patients aged 5 to <18 years1

Mean (SD) AGV results from a placebo-controlled clinical trial1-3

SD, standard deviation.
*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.
All randomized subjects. Two 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.
Baseline AGV was based on standing height at least 6 months prior to enrollment into the study.
§Two-sided P value.

Consistent improvement in AGV in favor of VOXZOGO® observed across all subgroups1,2

Difference in mean change from baseline in AGV at Week 52 by subgroup

*Data are based on 3 and 5 male patients taking VOXZOGO® and placebo, respectively, who were Tanner stage >|.
ANCOVA models were applied to determine the least squares mean change from baseline treatment difference at 52 weeks and 95% CIs. All models provide the treatment difference adjusted for the following baseline covariates: strata (male Tanner stage I, female Tanner stage I, male Tanner stage >I, female Tanner stage >I); age; annualized growth velocity; and height Z-score. Subgroup analyses were done by applying the ANCOVA model used for the primary analysis to each subgroup category. The vertical dashed line represents the change from baseline difference of 1.75 cm/year for which the study was powered.
AGV: annualized growth velocity; Z-score: height standard-deviation score.

VOXZOGO® improved height standard-deviation score (Z-score) compared to placebo1,2

Click here for PBS prescribing


VOXZOGO® Safety Data

The safety and efficacy of VOXZOGO® (vosoritide) were evaluated in patients with achondroplasia aged 5 to 15 years1,2

Adverse reactions that occurred in ≥5% of patients treated with VOXZOGO® and at a rate greater than placebo in study11,*

*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.

8 of 60 (13%) patients on VOXZOGO® experienced a transient decrease in blood pressure compared with 3 of 61 (5%) on placebo1

Decreases in blood pressure were identified during periods of frequent vital sign monitoring.

  • Median onset: 31 minutes (range, 18-120 minutes)
  • Median resolution: 31 minutes (range, 5-90 minutes)
  • 2 (3%) patients treated with VOXZOGO® each had one symptomatic episode of blood pressure with vomiting and/or dizziness compared with 0 patients on placebo
  • Patients with significant cardiac or vascular disease and patients on antihypertensive medicinal products were excluded from participation in VOXZOGO® clinical trials

97% of children on VOXZOGO® (58/60) remained on treatment during the 1-year clinical trial1,*

*One patient discontinued after 2 days due to pain from injections and one after 6 days due to fear of needles.


  1. VOXZOGO [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2021.
  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;396:684-692.
  3. Savarirayan R. Persistent and stable growth promoting effects of vosoritide in children with achondroplasia for up to 2 years: results from the ongoing phase 3 extension study. Oral presentation at: Endocrine Society’s Annual Meeting; November 21-24, 2021; Victoria, Australia.
  4. VOXZOGO® (vosoritide). Approved Product Information.
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PBS Prescribing

VOXZOGO® is now PBS listed from 1 May 2023 (Authority Required)



PBS information

  • 13275Q VOSORITIDE, vosoritide 400 microgram injection [10 vials] (&) inert substance diluent [10 x 0.5 mL syringes], 1 pack (VOXZOGO®)
  • 13274P VOSORITIDE, vosoritide 560 microgram injection [10 vials] (&) inert substance diluent [10 x 0.7 mL syringes], 1 pack (VOXZOGO®)
  • 13270K VOSORITIDE, vosoritide 1.2 mg injection [10 vials] (&) inert substance diluent [10 x 0.6 mL syringes], 1 pack (VOXZOGO®)

* Must be treated by a medical specialist, experienced in the management of achondroplasia; OR
* Must be treated by a paediatrician in consultation with a medical specialist experienced in the management of achondroplasia.

Clinical criteria – initial treatment:
* Patient must have a diagnosis of achondroplasia, confirmed by appropriate genetic testing. Appropriate genetic testing constitutes testing for FGFR3 gene mutation. AND
* Patient must not have evidence of growth plate closure demonstrated by at least one of the following:
i bilateral lower extremity X-rays (proximal tibia, distal femur). X-rays and dates (date commenced treatment and date of X-ray) must be documented in the patient’s medical records.
* In patients where puberty has not commenced, X-rays must be taken within 2 years of commencing treatment with vosoritide. Additional radiographic evidence is not required until patient has begun puberty.
* In patients where puberty has commenced, X-rays must be taken within 6 months of completing an authority application.
ii 1.5 cm/year as assessed over a period of at least 6 months.

* 30 vials + 5 repeats
* request the appropriate strength, based on the weight of the patient, according to the specified dosage in the approved Product Information (PI).

Continuing treatment:
* As above AND
* Patient must have received PBS subsidised vosoritide treatment previously.

Grandfather treatment (transition from non-PBS subsidised treatment) clinical criteria:
* As above AND
* Patient must have received non-PBS subsidised vosoritide treatment for this condition prior to 1 May 2023.
This grandfather restriction will cease to operate after 30 April 2024.

Request PBS prescribing codes
Approved Product Information


It is important to initiate treatment in children as young as possible. The volume of VOXZOGO® to be administered at the recommended dose is based on the patient’s weight and the vosoritide concentration (see Table 1). The usual dose is 15µg/kg body weight. For practicality reasons and to account for weight-related PK changes (see section 5.2 Pharmacokinetic Properties), the following dosing is recommended.

Table 1: Single dose volumes by body weight

Body weight affects VOXZOGO® clearance and volume of distribution. When treating with VOXZOGO®, it is essential to monitor growth, including height, body weight, and physical development, every 3-6 months

VOXZOGO® Dosing Calculator
VOXZOGO® Dosing and Administration Guide

Method of administration

VOXZOGO® is for subcutaneous single use in one patient only. Discard any residue. This medicinal product must be administered within 3 hours of reconstitution.
Prior to injecting, a healthcare professional should:

  • train caregivers on the preparation and subcutaneous injection of VOXZOGO®.
  • train caregivers and patients to recognise signs and symptoms of decreased blood pressure.
  • inform caregivers and patients what to do in the event of symptomatic decreases in blood pressure.

Patients and caregivers should be instructed to rotate sites for subcutaneous injections. Recommended injection sites on the body include the front middle of the thighs, the lower part of the abdomen except for 5 cm directly around the navel, top of the buttocks or the back of the upper arms. The same injection area should not be used on two consecutive days. VOXZOGO® should not be injected into sites that are red, swollen, or tender.

Patients should be well hydrated at the time of injection. It is recommended patients eat a light snack and drink a glass of fluid (e.g., water, milk, juice, etc.) about 30 minutes before injecting. This is to reduce the signs and symptoms of potential decreases in blood pressure (dizziness, fatigue and/or nausea) occurring (see section 4.4, Blood pressure effects).

If possible, VOXZOGO® should be injected at approximately the same time each day.

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