Carbaglu · 4.4 Special warnings and precautions for use · 1

HOS-093528 · has-adverse-reaction-information · PDF-Seite 4

Reported adverse reactions are listed below, by 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 the available data). Within each frequency grouping, undesirable effects are presented in order of decreasing seriousness. - Undesirable effects in N-acetylglutamate synthase deficiency Investigations Uncommon: increased transaminases Skin and subcutaneous tissue Common: increased sweating disorders Not known: rash - Undesirable effects in organic acidaemia Cardiac disorders Uncommon: bradycardia Gastrointestinal disorders Uncommon: diarrhoea, vomiting General disorders and Uncommon: pyrexia Administration site conditions Skin and subcutaneous tissue Not known: rash disorders 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 4 professionals are asked to report any suspected adverse reactions via the national reporting system listed in Appendix V.

HOS-093524 · has-contraindication-information · PDF-Seite 3

Hypersensitivity to the active substance or to any of the excipients listed in section 6.1 . Breast-feeding during the use of carglumic acid is contraindicated (see sections 4.6 and 5.3).

HOS-093523 · has-indication-information · PDF-Seite 2

Carbaglu is indicated in treatment of • hyperammonaemia due to N-acetylglutamate synthase primary deficiency. • hyperammonaemia due to isovaleric acidaemia. • hyperammonaemia due to methylmalonic acidaemia. • hyperammonaemia due to propionic acidaemia.

HOS-093526 · has-interaction-information · PDF-Seite 4

No specific interaction studies have been performed.

HOS-093527 · has-pregnancy-information · PDF-Seite 4

Pregnancy For carglumic acid no clinical data on exposed pregnancies are available. Animal studies have revealed minimal developmental toxicity (see section 5.3). Caution should be exercised when prescribing to pregnant women. Breast-feeding Although it is not known whether carglumic acid is secreted into human milk, it has been shown to be present in the milk of lactating rats (see section 5.3). Therefore, breast-feeding during the use of carglumic acid is contraindicated (see section 4.3).

HOS-093525 · has-warning-information · PDF-Seite 3

Therapeutic monitoring Plasma levels of ammonia and amino acids should be maintained within normal limits. As very few data on the safety of carglumic acid are available, systematic surveillance of liver, renal, cardiac functions and haematological parameters is recommended. Nutritional management Protein restriction and arginine supplementation may be indicated in case of low protein tolerance. 3 Use in patients with renal impairment The dose of Carbaglu must be reduced in patients with renal impairment (see section 4.2)