Voxzogo

Treatment with vosoritide should be initiated and directed by a physician appropriately qualified in the management of growth disorders or skeletal dysplasias. 3 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). 1 mL = 10 Units. 2), the following dosing is recommended. 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.

4 Paediatric population The safety and efficacy of Voxzogo in children aged less than 4 months of age is limited. 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. ) about 30 minutes before injecting.

Summary of the safety profile The most common adverse reactions to vosoritide were injection site reactions (85%), vomiting (27%), and decreased blood pressure (13%). Tabulated list of adverse reactions Adverse reactions in patients treated with vosoritide are tabulated below.

Adverse reactions are listed below by MedDRA system organ class and by frequency. Frequencies are defined as very common (≥ 1/10); common (≥ 1/100 to < 1/10); uncommon (≥ 1/1 000 to < 1/100); rare (≥ 1/10 000 to < 1/1 000); very rare (< 1/10 000); not known (cannot be estimated from available data).

Within each frequency grouping, adverse reactions are presented in order of decreasing seriousness. 6 Table 2: Adverse reactions in patients treated with Voxzogo System organ class Very common Common Uncommon Nervous system disorders Syncope Pre-syncope Dizziness Vascular disorders Hypotensiona Gastrointestinal disorders Vomiting Nausea Skin and subcutaneous tissue disorders Hypertrichosis General disorders and administration site conditions Injection site reactionb Fatigue Investigations Increased alkaline phosphatase a.

Hypotension includes both asymptomatic and symptomatic adverse reactions. b. Injection site reactions include the preferred terms; injection site erythema, injection site reaction, injection site swelling, injection site urticaria, injection site pain, injection site bruising, injection site pruritus, injection site haemorrhage, injection site discolouration, and injection site induration.

Description of selected adverse reactions Hypotension In ACH study 111-301, patients aged ≥ 5 years, 13% of patients treated with vosoritide compared to 5% of patients treated with placebo, experienced events of decreases in blood pressure which were transient and resolved without intervention.

The median time to onset from injection was 31 (18 to 120) minutes with resolution within 31 (5 to 90) minutes. The reported events were identified predominantly during periods of frequent vital signs monitoring at clinical visits after dosing over a 52-week treatment period.

Traceability In order to improve the traceability of biological medicinal products, the name and the batch number of the administered medicinal product should be clearly recorded. Blood pressure effects Patients with significant cardiac or vascular disease and patients on anti-hypertensive medicinal products were excluded from participation in premarketing clinical trials.

8). Sodium This medicinal product contains less than 1 mmol sodium (23 mg) per unit volume, that is to say essentially ‘sodium-free’. 5

1.