VOXZOGO Powder and solvent for solution for injection Ref.[49897] Active ingredients: Vosoritide

Treatment with vosoritide should be initiated and directed by a physician appropriately qualified in the management of growth disorders or skeletal dysplasias.

Posology

Voxzogo is given as a daily subcutaneous injection. The recommended dose is based on the patient’s weight and is approximately between 15-30 μg/kg, where the higher dose is given to smallest children, see Table 1.

The dose can be administered using either mL graduated syringes or Unit (U) graduated syringes (see Table 1). The measurements for the Unit graduated syringes are equivalent to mL as follows: 0.1 mL = 10 Units. 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 in mL and Units (U) volumes:

Duration of treatment

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.

Missed dose

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.

Growth monitoring

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).

Special populations

Patients with renal or hepatic impairment

The safety and efficacy of vosoritide in patients with renal or hepatic impairment has not been evaluated.

Paediatric population

The safety and efficacy of Voxzogo in children aged less than 4 months of age is limited. Currently available data are described in sections 4.8, 5.1 and 5.2, but no recommendation on a posology can be made.

Method of administration

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:

• train caregivers on the preparation and subcutaneous injection of this medicinal product.
• 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 an adequate amount 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) (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:

3 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.

Vosoritide 0.4 mg powder and solvent for solution for injection

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.

Vosoritide 0.56 mg powder and solvent for solution for injection

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.

Vosoritide 1.2 mg powder and solvent for solution for injection

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:

• 10 vials of Voxzogo
• 10 pre-filled syringes of water for injections
• 10 individual single use needles (23 gauge, for reconstitution)
• 10 individual single use syringes (30 gauge, for administration)

Preparation of Voxzogo for subcutaneous injection

• The correct Voxzogo strength and correct pre-filled syringe of solvent (reconstitution volume) should be confirmed based on the patient’s body weight (see Table 1).
• All necessary ancillary supplies must be in place before starting.
  • Alcohol pads
  • Gauze or bandages
  • Sharps container
• The Voxzogo vial and solvent in a pre-filled syringe (water for injections) should be removed from the refrigerator and allowed to reach room temperature before reconstituting Voxzogo.
• The solvent needle must be attached to the solvent in the pre-filled syringe (water for injections).
• The entire solvent volume must be injected into the vial.
• The solvent in the vial should be gently swirled until the white powder is completely dissolved. The vial should not be shaken.
• The dosing volume of the reconstituted solution should be slowly withdrawn from the single use vial into a syringe.
• Once reconstituted this medicinal product is a clear, colourless to yellow liquid. The solution should not be used if discoloured or cloudy, or if particles are present.
• After reconstitution, Voxzogo can be held in the vial at a room temperature up to 25°C for a maximum of 3 hours. The medicinal product contains no preservative.
• For administration, the required dose volume must be extracted from the vial using the supplied administration syringe (see Table 1).
• Each vial and pre-filled syringe are for single use only.
• Only the administration syringe provided should be used.

Disposal

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.