Tryngolza

Posology The recommended dose of olezarsen is 80 mg administered by subcutaneous injection once monthly. Missed dose If a dose is missed, Tryngolza should be administered as soon as possible. Dosing at monthly intervals should be resumed from the date of the most recently administered dose.

2). 2). Olezarsen has not been studied in patients with severe renal impairment or end-stage renal disease and should only be used in these patients if the anticipated clinical benefit outweighs the risk. 2). Olezarsen has not been studied in patients with moderate or severe hepatic impairment and should only be used in these patients if the anticipated clinical benefit outweighs the risk.

Paediatric population The safety and efficacy of this medicinal product in children and adolescents below 18 years of age have not yet been established. 1). Method of administration This medicinal product is intended for subcutaneous use only.

It should not be administered intramuscularly. Each pre-filled pen is for single use only. Patients and/or caregivers should be trained in the administration of this medicinal product in accordance with the comprehensive instructions for use provided at the end of the package leaflet.

This medicinal product should be administered into the abdomen or front of the thigh. The back of the upper arm can also be used as an injection site if a healthcare provider or caregiver administers the injection. It should not be injected into skin that is bruised, tender, red, or hard, into scars or damaged skin; the area around the navel should be avoided.

Some patients might not be responsive to the treatment after 6 months, in such a case the discontinuation of olezarsen should be considered on an individual basis by the prescribing physician. 6.

Summary of the safety profile In patients with FCS, the most commonly reported adverse reactions during treatment with olezarsen were injection site erythema (17%), headache (16%), arthralgia (15%), and vomiting (10%). Tabulated list of adverse reactions The safety data described below reflects exposure to olezarsen in 89 patients with FCS in clinical trials who received at least one dose of olezarsen.

Of these, 77 patients received at least 6 months of treatment and 65 patients received at least 12 months of treatment. The mean duration of treatment for these patients was 521 days (range: 28 to 1 080 days). Adverse reactions are listed according to MedDRA system organ class.

The frequency of adverse reactions is defined using the following convention: 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 the order of decreasing seriousness.

Table 1:

Adverse reactions System organ class Very common Common Immune system disorders Hypersensitivity Nervous system disorders Headache Gastrointestinal disorders Vomiting Musculoskeletal and connective tissue disorders Arthralgia Myalgia General disorders and administration site conditions Injection site erythema Injection site discolouration Chills Injection site pain Injection site swelling Description of selected adverse reactions Hypersensitivity Hypersensitivity has been observed with olezarsen.

Severe hypersensitivity reactions (including symptoms of bronchospasm, diffuse erythema, facial swelling, urticaria, chills, and myalgias) have been observed in 2 patients in clinical trials. In both patients the event was acute, required treatment, and resulted in treatment discontinuation.

8). If a serious hypersensitivity reaction occurs, Tryngolza must be discontinued immediately and appropriate therapy initiated. General Limited safety data exist for olezarsen use in FCS patients at the time of marketing authorisation.

While no serious risks of thrombocytopenia, hepatotoxicity, or renal toxicity were identified during clinical development, these adverse reactions have been observed with some antisense oligonucleotides and cannot be completely excluded.

Use in patients with low platelet counts Some patients with FCS are susceptible to platelet count variability over time as part of the natural history and progression of the disease. There are limited data available on the use of olezarsen in FCS patients with platelet count < 100 000/mm3.

4 Excipient with known effect Sodium This medicinal product contains less than 1 mmol sodium (23 mg) per 80 mg dose, that is to say essentially ‘sodium-free’.

1.