Cerdelga

Therapy with Cerdelga should be initiated and supervised by a physician knowledgeable in the management of Gaucher disease. Patient selection Before initiation of treatment with Cerdelga, patients must be genotyped for CYP2D6 to determine the CYP2D6 metaboliser status.

4). Posology Adults The recommended dose is 84 mg eliglustat twice daily in CYP2D6 IMs and EMs. The recommended dose is 84 mg eliglustat once daily in CYP2D6 PMs.

Paediatric population (from 6 to <18 years of age) weighing ≥ 15 kg Table 1:

Paediatric population (from 6 to <18 years of age) weighing ≥ 15 kg Weight CYP2D6 EMs and IMs CYP2D6 PMs ≥ 50 kg 84 mg twice daily 84 mg once daily 25 to < 50 kg 84 mg twice daily 42 mg once daily 15 to < 25 kg 42 mg twice daily 21 mg once daily Cerdelga is to be taken orally in children who can swallow intact capsule.

Missed dose If a dose is missed, the prescribed dose should be taken at the next scheduled time; the next dose should not be doubled. Elderly There is limited experience in the treatment of elderly with eliglustat. 2). 2) Paediatric population (<6 years of age) weighing <15 kg Safety and efficacy data of eliglustat are limited in paediatric patients below the age of 6 years.

There are no data to support the use of eliglustat in children weighing less than 15 kg. 1. Method of administration Cerdelga is to be taken orally. The capsules should be swallowed whole, preferably with water, and must not be crushed or dissolved.

The capsules may be taken with or without food. 5). Mixing the content of the capsule (eliglustat powder) into food or drinks has not been studied.

5% (for both cohorts) of paediatric patients from the ELIKIDS study. Overall, the safety profile of eliglustat in paediatric patients observed in clinical development setting was consistent with the established safety profile in adults.

Tabulated list of adverse reactions Adverse reactions are ranked by system organ class and 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)]).

Adverse reactions from long term clinical trial data reported in at least 4 patients are presented in Table 4. Within each frequency grouping, adverse reactions are presented in order of decreasing seriousness.

Table 4:

Tabulated list of adverse reactions System organ class Common Nervous system disorders Headache*, dizziness*, dysgeusia Cardiac disorders Palpitations Respiratory, thoracic and mediastinal disorders Throat irritation, cough Gastrointestinal disorders Dyspepsia, abdominal pain upper*, diarrhoea*, nausea, constipation, abdominal pain*, gastroesophageal reflux disease, abdominal distension*, gastritis, dysphagia, vomiting*, dry mouth, flatulence Skin and subcutaneous tissue disorders Dry skin, urticaria* Musculoskeletal and connective tissue disorders Arthralgia, pain in extremity*, back pain* General disorders and administration site conditions Fatigue * The incidence of the adverse reaction was the same or higher with placebo than with eliglustat in the placebo-controlled pivotal study.

6%). 7% (1/6) of the patients). Of 57 enrolled patients, 53 (93%, 48/51 in Cohort 1) experienced at least one treatment-emergent adverse event (TEAE) with no meaningful difference by age group, gender, or GD type. No patients permanently discontinued treatment due to TEAE.

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.

Patients with pre-existing cardiac conditions Use of eliglustat in patients with pre-existing cardiac conditions has not been studied during clinical trials. g. g. amiodarone, sotalol) antiarrhythmic medicinal products. 5 Patients with hepatic impairment and concomitant use with other medicinal products Concomitant use of eliglustat with CYP2D6 or CYP3A4 inhibitors in CYP2D6 EMs with mild hepatic impairment can result in further elevation of eliglustat plasma concentrations, with the magnitude of the effect depending on the enzyme inhibited and the potency of the inhibitor.

g. 2). 2). 1). For these patients, monitoring for further improvement or an alternative treatment modality should be considered. g. after 6 months with regular monitoring thereafter) should be performed for all disease domains to evaluate disease stability.

Reinstitution of enzyme replacement therapy or an alternative treatment modality should be considered in individual patients who have a sub-optimal response. Lactose Patients with rare hereditary problems of galactose intolerance, total lactase deficiency or glucose-galactose malabsorption should not take this medicine.

1. 5). 2).