Source: European Medicines Agency (EU) Revision Year: 2021 Publisher: BioMarin International Limited, Shanbally, Ringaskiddy, County Cork, P43 R298, Ireland
Voxzogo 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.
Treatment with vosoritide should be initiated and directed by a physician appropriately qualified in the management of growth disorders or skeletal dysplasias.
It is important to initiate treatment in children as young as possible.
The volume of vosoritide 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 15 µg/kg body weight. For practicality reasons and to account for weight-related PK changes (see section 5.2), the following dosing is recommended.
Table 1. Single dose volumes by body weight:
| Body weight (kg | Vosoritide 0.4 mg solvent (water for injections): 0.5 mL concentration: 0.8 mg/mL | Vosoritide 0.56 mg solvent (water for injections): 0.7 mL concentration: 0.8 mg/mL | Vosoritide 1.2 mg solvent (water for injections): 0.6 mL concentration: 2 mg/mL |
|---|---|---|---|
| Daily injection volume (mL) | |||
| 10-11 | 0.30 ml | ||
| 12-16 | 0.35 ml | ||
| 17-21 | 0.40 ml | ||
| 22-32 | 0.50 ml | ||
| 33-43 | 0.25 ml | ||
| 44-59 | 0.30 ml | ||
| 60-89 | 0.35 ml | ||
| ≥90 | 0.40 ml | ||
Treatment with this medicinal product should be stopped upon confirmation of no further growth potential, indicated by a growth velocity of <1.5 cm/year and closure of epiphyses.
If a dose of vosoritide is missed, it can be administered within 12 hours. If more than 12 hours have passed since the original dosing schedule, the missed dose should NOT be administered. Patients/caregivers should be advised to continue with the next scheduled dose the following day.
Patients should be monitored and assessed regularly every 3-6 months to check body weight, growth and physical development. Dose should be adjusted according to the patient’s body weight (see Table 1).
The safety and efficacy of vosoritide in patients with renal or hepatic impairment has not been evaluated.
The safety and efficacy of Voxzogo in children aged less than 2 years has not yet been established. Currently available data are described in sections 4.8, 5.1 and 5.2, but no recommendation on a posology can be made.
Voxzogo is for subcutaneous single use only. This medicinal product must be administered within 3 hours of reconstitution.
Prior to injecting, a healthcare professional should:
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, this medicinal product should be injected at approximately the same time each day.
For instructions on reconstitution of the medicinal product before administration, see section 6.6.
In clinical trials, doses of vosoritide were explored up to 30 μg/kg/day. Two patients received up to 3 times the recommended daily dose of 15 μg/kg/day for up to 5-weeks. No signs, symptoms or adverse reactions associated with the higher than intended dose were observed.
In the event a patient takes more than they should, the patient should contact their healthcare professional.
Unopened vials:
2 years
Reconstituted solution:
Chemical and physical stability has been demonstrated for 3 hours at 25°C.
From a microbiological point of view, unless the method of reconstitution precludes the risk of microbial contamination, the solution should be used immediately.
If not used immediately, Voxzogo must be administered within 3 hours of reconstitution (see section 4.2).
Store in a refrigerator (2°C–8°C). Do not freeze.
Store in the original package in order to protect from light.
Voxzogo may be stored at room temperature below 30°C for a single period up to 90 days, but not beyond the expiry date. Do not return Voxzogo to refrigerator after storage at room temperature.
For storage conditions after reconstitution of the medicinal product, see section 6.3.
Powder: 2 mL vial (glass) with rubber stopper (bromobutyl) and white flip cap.
Solvent: Pre-filled syringe (glass) with plunger (bromobutyl) and tip cap with a luer lock and tamper evident seal containing 0.5 mL of water for injections.
Powder: 2 mL vial (glass) with rubber stopper (bromobutyl) and magenta flip cap.
Solvent: Pre-filled syringe (glass) with plungers (bromobutyl) and tip cap with a luer lock and tamper evident seal containing 0.7 mL of water for injections.
Powder: 2 mL vial (glass) with rubber stopper (bromobutyl) and grey flip cap.
Solvent: Pre-filled syringe (glass) with plungers (bromobutyl) and tip cap with a luer lock and tamper evident seal containing 0.6 mL of water for injections
Each carton contains:
Any unused medicinal product or waste material should be disposed of in accordance with local requirements.
All needles and syringes should be disposed of in a sharps disposal container.
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