KANUMA

Country: Israel

Language: English

Source: Ministry of Health

Active ingredient:

SEBELIPASE ALFA

Available from:

ALEXION PHARMA ISRAEL LTD

ATC code:

A16AB14

Pharmaceutical form:

CONCENTRATE FOR SOLUTION FOR INFUSION

Composition:

SEBELIPASE ALFA 2 MG / 1 ML

Administration route:

I.V

Prescription type:

Required

Manufactured by:

ALEXION PHARMA GMBH, SWITZERLAND

Therapeutic area:

SEBELIPASE ALFA

Therapeutic indications:

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

Authorization date:

2022-01-31

Patient Information leaflet

1
2
.20
04
KANUMA® (34903)
Active ingredient and strength:
SEBELIPASE ALFA 2MG/1ML
המונק
ליעפ רמוח
קזוחו
: אפלא סאביליביס
2
/ ג"מ
1
ל"מ
CONCENTRATE FOR SOLUTION FOR INFUSION
,ה/דבכנ ת/חקור ,ה/אפור
מ"עב לארשי המראפ ןויסקלא
_ _
לע עידוהל החמש
ןוכדע
ןולע
ל
אפור
לש
רישכתה
Kanuma
.
•
ולע
ן
ה
רישכת
כדוע
ן
ב
לירפא
2021
,
םיראותמ וז העדוהב
תוחיטבה ייוניש
םיירקיעה
ןולעב
,
ןולעב
ק
םייוניש םימיי
םיפסונ
.
•
עדימ
שדח
מ
עקר לע עיפו
בוהצ
הצוח וקב ןמוסמ רסוהש טסקט ,
.
KANUMA is indicated for long-term enzyme replacement therapy (ERT) in
patients of all ages with lysosomal acid
lipase (LAL) deficiency.
םיירקיעה םינוכדעה
:םיאבה םיפיעסב ושענ
4.4. SPECIAL WARNINGS AND PRECAUTIONS FOR USE
Traceability
In order to improve the traceability of biological medicinal products,
the name and the batch number of the
administered product should be clearly recorded.
(…)
Immunogenicity
As with all therapeutic proteins, there is potential for
immunogenicity. In the sebelipase alfa clinical program, patients
were routinely tested for anti-sebelipase alfa anti-drug antibodies
(ADAs) to determine the immunogenicity potential
of sebelipase alfa. Patients who tested positive for ADAs were also
tested for inhibitory antibody activity. The
presence of inhibitory activity has been detected at some postbaseline
timepoints in clinical studies (see section 4.8).
Overall, no conclusion on the relationship between development of
ADAs/NAbs and associated hypersensitivity
reactions or suboptimal clinical response can be made.
In clinical studies, 3 patients homozygous for a deletion affecting
both alleles of genes Lipase A, lysosomal acid
[LIPA] and Cholesterol 25-Hydroxylase developed inhibitory antibody
activity associated with a suboptimal clinical
response. These patients underwent either im

Summary of Product characteristics

1
SUMMARY OF PRODUCT CHARACTERISTICS
1.
NAME OF THE MEDICINAL PRODUCT
KANUMA
2 mg/ml concentrate for solution for infusion
2.
QUALITATIVE AND QUANTITATIVE COMPOSITION
Each ml of concentrate contains 2 mg sebelipase alfa*. Each vial of 10
ml contains 20 mg sebelipase
alfa.
*Sebelipase alfa is produced in egg white of transgenic _Gallus _by
recombinant DNA (rDNA)
technology.
Excipient with known effect:
Each vial contains 33 mg sodium.
For the full list of excipients, see section 6.1.
3.
PHARMACEUTICAL FORM
Concentrate for solution for infusion (sterile concentrate).
Clear to slightly opalescent, colourless to slightly coloured
solution.
4.
CLINICAL PARTICULARS
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.
4.2
POSOLOGY AND METHOD OF ADMINISTRATION
KANUMA treatment should be supervised by a healthcare professional
experienced in the
management of patients with LAL deficiency, other metabolic disorders,
or chronic liver diseases.
KANUMA should be administered by a trained healthcare professional who
can manage medical
emergencies.
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. Following the occurrence of a hypersensitivity reaction,
appropriate pre-treatment should
be considered according to the standard of care (see section 4.4).
2
_Infants (< 6 months of age)_
The recommended starting dose in infants (< 6 months of age)
presenting with rapidly
progressive LAL deficiency is 1 mg/kg administered as an intravenous
infusion once weekly.
Dose escalation to 3 mg/kg once weekly should be considered based on
clinical response.
_Children and adults_
The recommended dose in children and adults who do not present with
rapidly progressive
LAL deficiency prior to 6 months of age is 1 mg/kg administered as an
intravenous infusion
once every other week.