Hemgenix

Country: Եվրոպական Միություն

language: անգլերեն

source: EMA (European Medicines Agency)

active_ingredient:

Etranacogene dezaparvovec

MAH:

CSL Behring GmbH

ATC_code:

B06

INN:

etranacogene dezaparvovec

therapeutic_group:

Other hematological agents

therapeutic_area:

Hemophilia B

therapeutic_indication:

Treatment of severe and moderately severe Haemophilia B (congenital Factor IX deficiency) in adult patients without a history of Factor IX inhibitors.

leaflet_short:

Revision: 2

authorization_status:

Authorised

authorization_date:

2023-02-20

PIL

33
B. PACKAGE LEAFLET
34
PACKAGE LEAFLET: INFORMATION FOR THE USER
HEMGENIX 1 X 10
13
GENOME COPIES/ML CONCENTRATE FOR SOLUTION FOR INFUSION
etranacogene dezaparvovec
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 may get.
See the end of section 4 for how
to report side effects.
READ ALL OF THIS LEAFLET CAREFULLY BEFORE YOU START USING 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 or nurse.
-
If you get any side effects, talk to your doctor or nurse. This
includes any possible side effects
not listed in this leaflet. See section 4.
-
Your doctor will give you a Patient Card. Read it carefully and follow
the instructions on it.
WHAT IS IN THIS LEAFLET
1.
What Hemgenix is and what it is used for
2.
What you need to know before you are given Hemgenix
3.
How Hemgenix is given
4.
Possible side effects
5.
How to store Hemgenix
6.
Contents of the pack and other information
1.
WHAT HEMGENIX IS AND WHAT IT IS USED FOR
WHAT HEMGENIX IS AND WHAT IT IS USED FOR
Hemgenix is a gene therapy product that contains the active substance
etranacogene dezaparvovec. A
gene therapy product works by delivering a gene into the body to
correct a genetic defect.
Hemgenix is used for the treatment of severe and moderately severe
Haemophilia B (congenital
Factor IX deficiency) in adults who do not have current or past
inhibitors (neutralising antibodies)
against the Factor IX protein.
People with Haemophilia B are born with an altered form of a gene
needed to make Factor IX, an
essential protein required for blood to clot and stop any bleeding.
People with Haemophilia B have
insufficient levels of Factor IX and are prone to internal or external
bleeding episodes.
HOW HEMGENIX WORKS
The active substance in Hemgenix is based on a virus that does not
cause disease in humans. This virus
has been modified so tha

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
Hemgenix 1 x 10
13
genome copies/mL concentrate for solution for infusion
2.
QUALITATIVE AND QUANTITATIVE COMPOSITION
2.1
GENERAL DESCRIPTION
Etranacogene dezaparvovec is a gene therapy medicinal product that
expresses the human coagulation
Factor IX. It is a non-replicating, recombinant adeno-associated virus
serotype 5 (AAV5) based vector
containing a codon-optimised cDNA of the human coagulation Factor IX
variant R338L (FIX-Padua)
gene under the control of a liver-specific promoter (LP1).
Etranacogene dezaparvovec is produced in
insect cells by recombinant DNA technology.
2.2
QUALITATIVE AND QUANTITATIVE COMPOSITION
Each mL of etranacogene dezaparvovec contains 1 x 10
13
genome copies (gc).
Each vial contains an extractable volume of 10 mL of concentrate for
solution for infusion, containing a
total of 1 x 10
14
genome copies.
The total number of vials in each pack corresponds to the dosing
requirement for the individual patient,
depending on the patient`s body weight (see sections 4.2 and 6.5).
Excipient with known effect
This medicinal product contains 35.2 mg sodium per vial (3.52 mg/mL).
For the full list of excipients, see section 6.1.
3.
PHARMACEUTICAL FORM
Concentrate for solution for infusion (sterile concentrate)
Clear, colourless solution.
4.
CLINICAL PARTICULARS
4.1
THERAPEUTIC INDICATIONS
Hemgenix is indicated for the treatment of severe and moderately
severe Haemophilia B (congenital
Factor IX deficiency) in adult patients without a history of Factor IX
inhibitors.
4.2
POSOLOGY AND METHOD OF ADMINISTRATION
Treatment should be initiated under the supervision of a physician
experienced in the treatment of
Haemophilia and/or bleeding disorders. This medicinal product shou