ELELYSO POWDER FOR CONCENTRATE FOR SOLUTION FOR INFUSION 200 UNITS/ VIAL

4.1. Therapeutic indications

Taliglucerase alfa for infusion is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term enzyme replacement therapy for adult and pediatric patients with a confirmed diagnosis of Type 1 Gaucher disease associated with at least one of the following: splenomegaly, hepatomegaly, anemia, thrombocytopenia.

4.3. Contraindications

Severe allergic reactions to taliglucerase alfa or any of the excipients listed in section 6.1. List of excipients – please refer to the Product Insert/Patient Information Leaflet published on HSA for the full drug information.

4.2. Posology and method of administration

Treatment with taliglucerase alfa should be supervised by a physician experienced in the management of patients with Gaucher disease. Home administration under the supervision of a healthcare professional may be considered only for those patients who have been tolerating their infusions (see section 4.8. Undesirable effects – please refer to the Product Insert/Patient Information Leaflet published on HSA for the full drug information).

Posology

Due to the heterogeneity and the multi-systemic nature of Gaucher disease, dosage adjustments should be made on an individual basis. Dose requirements may increase or decrease, based on achievement of therapeutic goals, as assessed by regular comprehensive evaluations of the patient’s clinical manifestations.

Adult dosing

Initial doses of taliglucerase alfa in adult subjects range from 30 units/kg to 60 units/kg of body weight once every 2 weeks, depending upon the clinical assessment of the treating physician. Clinical studies have evaluated median dose ranges from 9 units/kg to 67 units/kg every other week.

Adult patients currently being treated with imiglucerase for Gaucher disease can be switched to taliglucerase alfa. It is recommended that patients previously treated on a stable dose of imiglucerase begin treatment with taliglucerase alfa at the same dose of imiglucerase when they switch from imiglucerase to taliglucerase alfa.

Pediatric dosing

Initial doses of taliglucerase alfa in pediatric subjects range from 30 units/kg to 60 units/kg of body weight once every 2 weeks, depending upon the clinical assessment of the treating physician. Clinical studies have evaluated dose ranges from 26 units/kg to 78 units/kg every other week (see section 4.9. Overdose – please refer to the Product Insert/Patient Information Leaflet published on HSA for the full drug information).

Pediatric patients currently being treated with imiglucerase for Gaucher disease can be switched to taliglucerase alfa. It is recommended that patients previously treated on a stable dose of imiglucerase begin treatment with taliglucerase alfa at the same dose of imiglucerase when they switch from imiglucerase to taliglucerase alfa.

The safety and efficacy of taliglucerase alfa in children less than 2 years of age have not yet been established. No data are available.

Method of administration

After reconstitution and dilution, the total volume of prepared solution is administered intravenously by infusion over a period of 60 to 120 minutes (see section 4.4. Special warnings and precautions for use – please refer to the Product Insert/Patient Information Leaflet published on HSA for the full drug information). For pediatric patients with weights less than 30 kg, an infusion rate of no greater than 1 mL/minute should be used. For pediatric patients with weights more than 30 kg, after an initial infusion rate of 1 mL/minute and after tolerability to taliglucerase alfa is established, the infusion rate may be increased to 2 mL/minute. For adult patients, after an initial infusion rate of 1.2 mL/minute and after tolerability to taliglucerase alfa is established, the infusion rate may be increased to a maximum of 2.2 mL/minute. The duration of infusion may be adjusted as tolerated by the patient. The diluted solution should be filtered through an in-line low protein-binding 0.2 μm filter during administration.

The number of taliglucerase alfa vials necessary for the patient at the recommended dose is reconstituted in Sterile Water for Injection, as instructed. The reconstituted medicinal product is pooled, and the volume of infusion is adjusted with sodium chloride 9 mg/mL (0.9%) solution for injection to a total volume of 100 to 200 mL (see section 6.6. Special precautions for disposal and other handling – please refer to the Product Insert/Patient Information Leaflet published on HSA for the full drug information).

Each vial of taliglucerase alfa is for single use in one patient only.

For instructions on reconstitution and dilution of taliglucerase alfa, see section 6.6. Special precautions for disposal and other handling – please refer to the Product Insert/Patient Information Leaflet published on HSA for the full drug information.

Renal or hepatic impairment

Studies of taliglucerase alfa in patients with Gaucher disease with renal or hepatic impairment have not been conducted.

Elderly (≥65 years old)

During clinical studies, 8 patients aged 65 years or older were treated with taliglucerase alfa. This limited data set does not indicate the need for dose adjustment in this age group.