recombinant human n-acetylgalactosamine-6-sulfatase

HOS-096772 · has-adverse-reaction-information · PDF-Seite 5

Summary of the safety profile The assessment of adverse reactions is based on the exposure of 176 patients with MPS IVA, ages 5 to 57 years old to 2 mg/kg elosulfase alfa once a week (n=58), 2 mg/kg elosulfase alfa once every other week (n=59), or placebo (n=59) in a randomised, double-blind, placebo-controlled study. The majority of adverse reactions in clinical studies were IRs, which are defined as reactions occurring after initiation of infusion until the end of the day following the infusion. Serious IRs were observed in clinical studies and included anaphylaxis, hypersensitivity and vomiting. The most common symptoms of IRs (occurring in ≥ 10% of patients treated with Vimizim and ≥ 5% more when compared to placebo) were headache, nausea, vomiting, pyrexia, chills and abdominal pain. IRs were generally mild or moderate, and the frequency was higher during the first 12 weeks of treatment and tended to occur less frequently with time. Tabulated list of adverse reactions The data in Table 2 below describes adverse reactions from clinical studies in patients treated with Vimizim. 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) and not known (cannot be estimated from available data). Within each frequency grouping, adverse reactions are presented in order of decreasing seriousness. Table 2: Adverse reactions in patients treated with Vimizim MedDRA MedDRA Frequency System organ class Preferred term Immune system disorders Anaphylaxis Uncommon Hypersensitivity Common Nervous system disorders Headache Very common Dizziness Very common Respiratory, thoracic, and Dyspnoea Very common mediastinal disorders Gastrointestinal disorders Diarrhoea, vomiting, Very common oropharyngeal pain, upper abdominal pain, abdominal pain, nausea Musculoskeletal and connective Myalgia Common 5 tissue disorders Chills V

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HOS-096768 · has-contraindication-information · PDF-Seite 3

Life-threatening hypersensitivity (anaphylactic reaction) to the active substance or to any of the excipients listed in section 6.1 (see section 4.4).

HOS-096767 · has-indication-information · PDF-Seite 2

Vimizim is indicated for the treatment of mucopolysaccharidosis, type IVA (Morquio A Syndrome, MPS IVA) in patients of all ages.

HOS-096770 · has-interaction-information · PDF-Seite 4

No interaction studies have been performed.

HOS-096771 · has-pregnancy-information · PDF-Seite 4

Pregnancy There are no data on the use of Vimizim in pregnant women. Animal studies do not indicate direct or indirect harmful effects with respect to pregnancy or embryo- foetal development (see section 5.3). These studies however, are of limited relevance. As a precautionary measure, it is preferable to avoid the use of Vimizim during pregnancy, unless clearly necessary. Breast-feeding Available reproductive data in animals have shown excretion of elosulfase alfa in milk. It is not known whether elosulfase alfa is excreted in human breast milk, but systemic exposure via breast milk is not 4 expected. Due to lack of human data, Vimizim should only be administered to breast-feeding woman if the potential benefit is considered to outweigh the potential risk to the infant. Fertility No impairment of fertility has been observed in nonclinical studies (see section 5.3) with elosulfase alfa.

HOS-096769 · has-warning-information · PDF-Seite 3

Anaphylaxis and severe allergic reactions Anaphylaxis and severe allergic reactions have been reported in clinical studies. Therefore, appropriate medical support must be readily available when elosulfase alfa is administered. If these reactions occur, immediately stop the infusion and initiate appropriate medical treatment. The current medical standards for emergency treatment are to be followed. For patients who have experienced allergic reactions during infusion, caution should be exercised upon re-administration. 3 Infusion reactions Infusion reactions (IRs) were the most commonly observed adverse reactions in clinical studies. IRs may include allergic reactions. Patients should receive antihistamines with or without antipyretics prior to infusion (see section 4.2). Management of IRs should be based on the severity of the reaction and include slowing or temporary interruption of the infusion and/or administration of additional antihistamines, antipyretics, and/or corticosteroids. If severe IRs occur, immediately stop the infusion and initiate appropriate treatment. Re-administration after a severe reaction should be carried out with caution and close monitoring by the treating physician. Spinal/Cervical cord compression In clinical studies, spinal/cervical cord compression (SCC) was observed both in patients receiving Vimizim and patients receiving placebo. Patients should be monitored for signs and symptoms of SCC (including back pain, paralysis of limbs below the level of compression, urinary and faecal incontinence) and given appropriate clinical care. Sodium restricted diet This medicinal product contains 8 mg sodium per vial, equivalent to 0.4% of the WHO recommended maximum daily intake of 2 g sodium for an adult, and is administered in sodium chloride 9 mg/ml (0.9%) solution for infusion (see section 6.6). Sorbitol (E 420) This medicinal product contains 100 mg sorbitol in each vial which is equivalent to 40 mg/kg. Patients with hereditary fructose intolerance (HFI) must not be given this medicinal product unless strictly necessary. Babies and young children (below 2 years of age) may not yet be diagnosed with hereditary fructose intolerance (HFI). Medicinal products containing sorbitol/fructose given intravenously may be life- threatening. The treatment benefit to the child compared to the associated risks must be fully evaluated prior to treatment. A detailed history with rega

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