Kanuma

Country: Եվրոպական Միություն

language: անգլերեն

source: EMA (European Medicines Agency)

active_ingredient:

sebelipase alfa

MAH:

Alexion Europe SAS

ATC_code:

A16

INN:

sebelipase alfa

therapeutic_group:

Other alimentary tract and metabolism products,

therapeutic_area:

Lipid Metabolism, Inborn Errors

therapeutic_indication:

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

leaflet_short:

Revision: 10

authorization_status:

Authorised

authorization_date:

2015-08-28

PIL

27
B. PACKAGE LEAFLET
28
PACKAGE LEAFLET: INFORMATION FOR THE USER
KANUMA 2 MG/ML CONCENTRATE FOR SOLUTION FOR INFUSION
sebelipase alfa
This medicine is subject to additional monitoring. This will allow
quick identification of new
safety information. You can help by reporting any side effects you or
your child may get. See the end
of section 4 for how to report side effects.
READ ALL OF THIS LEAFLET CAREFULLY BEFORE YOU ARE GIVEN THIS MEDICINE
BECAUSE IT CONTAINS IMPORTANT
INFORMATION FOR YOU.
-
Keep this leaflet. You may need to read it again.
-
If you have any further questions, ask your doctor.
-
If you get any side effects, talk to your doctor. This includes any
possible side effects not listed
in this leaflet. See section 4.
WHAT IS IN THIS LEAFLET
1.
What KANUMA is and what it is used for
2.
What you need to know before you are given KANUMA
3.
How KANUMA is given
4.
Possible side effects
5.
How to store KANUMA
6.
Contents of the pack and other information
1.
WHAT KANUMA IS AND WHAT IT IS USED FOR
KANUMA contains the active substance sebelipase alfa. Sebelipase alfa
is similar to the naturally
occurring enzyme lysosomal acid lipase (LAL), which the body uses to
breakdown fats. It is used to
treat patients of all ages with lysosomal acid lipase deficiency (LAL
deficiency).
LAL deficiency is a genetic disease that leads to liver damage, high
blood cholesterol, and other
complications due to a build-up of certain types of fats (cholesteryl
esters and triglycerides).
HOW KANUMA WORKS
This medicine is an enzyme replacement therapy. This means that it
replaces the missing or defective
LAL enzyme in patients with LAL deficiency. This medicine works by
lowering the build-up of fat
that causes medical complications, including impaired growth, liver
damage and heart complications.
It also improves blood levels of fats, including elevated LDL (bad
cholesterol) and triglycerides.
2.
WHAT YOU NEED TO KNOW BEFORE KANUMA IS GIVEN
YOU MUST NOT BE GIVEN KANUMA
-
If you or your child has experienced life-threatening allergic

SPC

1
_ _
ANNEX I
SUMMARY OF PRODUCT CHARACTERISTICS
2
This medicinal product is subject to additional monitoring. This will
allow quick identification of
new safety information. Healthcare professionals are asked to report
any suspected adverse reactions.
See section 4.8 for how to report adverse reactions.
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.
* 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).
3
_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 either 1 mg/kg or 3 mg/kg administered as an
intravenous