Nulibry · 4.1 Therapeutic indications · 1

HOS-288035 · has-adverse-reaction-information · PDF-Seite 4

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 4 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 Complications associated with device site conditions 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.

HOS-288031 · has-contraindication-information · PDF-Seite 3

Hypersensitivity to the active substance or to any of the excipients listed in section 6.1.

HOS-288030 · has-indication-information · PDF-Seite 2

NULIBRY is indicated for the treatment of patients with molybdenum cofactor deficiency (MoCD) Type A.

HOS-288033 · has-interaction-information · PDF-Seite 4

No clinical drug-drug interaction studies have been performed with fosdenopterin. The likelihood of metabolism-based and transporter-based drug-drug interactions with fosdenopterin are minimal, and co-administration of other medicinal products is not likely to affect the pharmacokinetics of fosdenopterin (see section 5.2).

HOS-288034 · has-pregnancy-information · PDF-Seite 4

Pregnancy There are no or limited amount of data from the use of fosdenopterin in pregnant women. Animal studies are insufficient with respect to reproductive toxicity (see section 5.3). NULIBRY is not recommended during pregnancy and in women of childbearing potential not using contraception. Breastfeeding It is unknown whether fosdenopterin/metabolites are excreted in human milk. A risk to newborns/infants cannot be excluded. A decision must be made whether to discontinue breast-feeding or to discontinue from NULIBRY therapy taking into account the benefit of breast feeding for the child and the benefit of therapy for the woman. Fertility Fertility studies have not been conducted with fosdenopterin.

HOS-288032 · has-warning-information · PDF-Seite 3

Potential for photosensitivity Photosensitivity is a potential risk based on in vitro and in vivo animal studies, see section 5.3. 3 Fosdenopterin-treated patients or their caregivers must be advised that patients avoid or minimise exposure to direct sunlight and artificial UV light exposure (i.e., UVA or UVB phototherapy) and adopt precautionary measures (e.g., 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’.