KANUMA CONCENTRATE FOR SOLUTION FOR INFUSION 2 MG/ML [SIN17346P]

Product Info

KANUMA CONCENTRATE FOR SOLUTION FOR INFUSION 2 MG/ML

[SIN17346P]

Indication

4.1 Therapeutic indications

KANUMA is indicated for long-term enzyme replacement therapy (ERT) in patients of all ages with lysosomal acid lipase (LAL) deficiency.

Dosing

4.2 Posology and method of administration

Posology

It is important to initiate treatment as early as possible after diagnosis of LAL Deficiency.

For instructions on the preventive measures and monitoring of hypersensitivity reactions, see section 4.4 – please refer to the Product Insert/Patient Information Leaflet published on HSA for the full drug information. Following the occurrence of a hypersensitivity reaction, appropriate pre-treatment should be considered according to the standard of care (see section 4.4 – please refer to the Product Insert/Patient Information Leaflet published on HSA for the full drug information).

Patients with rapidly progressive LAL Deficiency presenting within the first 6 months of life

The recommended starting dose in infants (< 6 months of age) presenting with rapidly progressive LAL Deficiency is 1 mg/kg or 3 mg/kg administered as an intravenous (IV) infusion once weekly (qw), depending on the clinical status of the patient. Dose escalations should be considered based on suboptimal response to clinical and biochemical criteria, including, e.g., poor growth (especially mid-upper arm circumference, MUAC), deteriorating biochemical markers (e.g., liver transaminases, ferritin, C-reactive protein, and coagulation parameters), persistent or worsening organomegaly, increased frequency of intercurrent infections, and persistent worsening of other symptoms (e.g., gastrointestinal symptoms).

• a dose escalation to 3 mg/kg should be considered in case of suboptimal clinical response.

• a further dose escalation up to 5 mg/kg should be considered in case of persistent suboptimal clinical response.

Further dose adjustments, as a reduction of the dose or an extension of the dose interval, can be made on an individual basis based on achievement and maintenance of therapeutic goals. Clinical studies evaluated doses ranging from 1 to 5 mg/kg once weekly, with one patient receiving a higher dose of 7.5 mg/kg once weekly. Doses higher than 7.5 mg/kg have not been studied.

Pediatric and adult patients with LAL Deficiency

The recommended dose in children and adults presenting with LAL Deficiency is 1 mg/kg administered as an IV infusion once every other week (qow). Dose escalation to 3 mg/kg once every other week should be considered based on suboptimal response to clinical and biochemical criteria, including, e.g., poor growth persistent or deteriorating biochemical markers (e.g., parameters of liver injury (ALT, AST), parameters of lipid metabolism (TC, LDL-c, HDL-c, TG), persistent or worsening organomegaly, and persistent worsening of other symptoms (e.g., gastrointestinal symptoms).

Special populations

Elderly population (≥ 65 years old)

The safety and efficacy of KANUMA in patients older than 65 years have not been evaluated and no alternative dosage regimens can be recommended for these patients.

Renal or hepatic impairment

No dosing adjustment is recommended in patients with renal or hepatic impairment based on current knowledge of the pharmacokinetics and pharmacodynamics of sebelipase alfa. See Section 5.1 and Section 5.2 – please refer to the Product Insert/Patient Information Leaflet published on HSA for the full drug information.

Paediatric population

Eighty-eight of 125 patients (70%) who received KANUMA during clinical studies were in the paediatric and adolescent age range (1 month up to 18 years) at the time of first dose. Currently available data are described in Section 4.8 and Section 5.1 – please refer to the Product Insert/Patient Information Leaflet published on HSA for the full drug information.

Method of administration

KANUMA is for intravenous (IV) use only.

The total volume of the infusion should be administered over approximately 2 hours. A 1-hour infusion may be considered for those patients receiving the 1 mg/kg dose after patient tolerability is established. The infusion period may be extended in the event of dose escalation. KANUMA should be administered through a 0.2 micrometre filter.

For instructions on the preventive measures and monitoring of hypersensitivity reactions, see Section 4.4 – please refer to the Product Insert/Patient Information Leaflet published on HSA for the full drug information.

For instructions on dilution of the medicinal product before administration, see Section 6.5 – please refer to the Product Insert/Patient Information Leaflet published on HSA for the full drug information.

Contraindications

4.3 Contraindications

Life-threatening hypersensitivity (anaphylactic reaction) to the active substance when attempts to rechallenge are unsuccessful, or to egg or any of the excipients listed in section 6.1 – please refer to the Product Insert/Patient Information Leaflet published on HSA for the full drug information, (see section 4.4 – please refer to the Product Insert/Patient Information Leaflet published on HSA for the full drug information).