Nulibry

NULIBRY is to be administered only if the patient has a confirmed genetic diagnosis or presumptive diagnosis of MoCD Type A. Patients with a presumptive diagnosis of MoCD Type A need to have a genetic test to confirm the diagnosis of MoCD Type A.

NULIBRY must be discontinued if the MoCD Type A diagnosis is not confirmed by genetic testing. Treatment with NULIBRY is to be initiated and supervised in hospital by a healthcare professional experienced in the management of inborn errors of metabolism.

NULIBRY is a chronic substrate replacement therapy intended for long-term use. Posology Paediatric population less than 1 year of age (by gestational age) In patients less than one year of age, the recommended dose of NULIBRY is titrated based on gestational age.

40 mg/kg/day administered intravenously once daily. 90 mg/kg/day over a period of 3 months as shown in Table 1. 55 mg/kg/day administered intravenously once daily. 90 mg/kg/day over a period of 3 months as shown in Table 1. 90 mg/kg (based on actual body weight) administered intravenously once daily.

Missed dose If a dose is missed, the missed dose is to be administered as soon as possible. The next scheduled dose must be given at least 6 hours after the administration of the missed dose. Method of administration NULIBRY is for intravenous use only.

5 mL/min after reconstitution with 5 mL of sterile water for injection. Dose volumes below 2 mL may require syringe administration by slow intravenous push. 6. If deemed appropriate by a healthcare professional, NULIBRY may be administered at home by the patient’s caregiver.

If NULIBRY is administered by a caregiver/patient, the caregiver/patient must read and follow carefully the detailed “Instructions for the user” on the preparation, administration, storage, and disposal of NULIBRY provided in the carton.

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Summary of the safety profile The adverse drug reactions described in this section were evaluated in 11 patients with MoCD Type A. The most frequent (> 20%) adverse reaction observed during clinical trials were complications 5 associated with device, which have been attributed to the catheter and not to fosdenopterin.

No patients had to have their treatment discontinued due to adverse events. Tabulated list of adverse reactions Adverse drug reactions (ADRs) observed are listed below by MedDRA system organ class and by frequency: 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).

Table 2 presents the most common ADRs that occurred in patients treated with NULIBRY. Table 2 Adverse reactions reported by SOC/PT and frequency System Organ Class Very common (≥ 10%) General disorders and administration site conditions Complications associated with device Description of selected adverse reactions Catheter-related complications Eight of ten patients treated with NULIBRY experienced at least one device-related adverse event.

The events reported in more than one patient included complications associated with device (7 patients), device dislocation and catheter site infection (3 patients each), and catheter site extravasation, catheter site pain, central venous catheterization, catheter site discharge, device leakage, device occlusion, bacteraemia, sepsis, and vascular device infection (2 patients each).

Reporting of suspected adverse reactions Reporting suspected adverse reactions after authorisation of the medicinal product is important. It allows continued monitoring of the benefit/risk balance of the medicinal product. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system listed in Appendix V.

3. , use broad spectrum sunscreen with high sun protection factor, and wear clothing, a hat, and sunglasses that protects against sun exposure). Caregivers/patients must be advised to seek medical attention immediately if the patient develops a rash or if they notice symptoms of photosensitivity reactions (redness, burning sensation of the skin, blisters).

Physicians should consider Vitamin D supplementation due to the use of sunscreens and sun protective clothing and advise the caregiver/patients accordingly. Sodium content This medicinal product contains less than 1 mmol sodium (23 mg) per vial, that is to say essentially ‘sodium-free’.

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