Roctavian

Country: Evrópusambandið

Tungumál: enska

Heimild: EMA (European Medicines Agency)

Virkt innihaldsefni:

valoctocogene roxaparvovec

Fáanlegur frá:

BioMarin International Limited

ATC númer:

B02BD15

INN (Alþjóðlegt nafn):

Valoctocogene roxaparvovec

Meðferðarhópur:

Antihemorrhagics

Ábendingar:

Treatment of severe haemophilia A (congenital factor VIII deficiency) in adult patients without a history of factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5).

Vörulýsing:

Revision: 3

Leyfisstaða:

Authorised

Leyfisdagur:

2022-08-24

Upplýsingar fylgiseðill

36
B. PACKAGE LEAFLET
37
PACKAGE LEAFLET: INFORMATION FOR THE PATIENT
ROCTAVIAN 2 × 10
13 VECTOR GENOMES/ML SOLUTION FOR INFUSION
valoctocogene roxaparvovec
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.
-
If you get any side effects, talk to your doctor. 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 ROCTAVIAN is and what it is used for
2.
What you need to know before you are given ROCTAVIAN
3.
How ROCTAVIAN is given
4.
Possible side effects
5.
How ROCTAVIAN is stored
6.
Contents of the pack and other information
1.
WHAT ROCTAVIAN IS AND WHAT IT IS USED FOR
WHAT ROCTAVIAN IS
ROCTAVIAN is a gene therapy product that contains the active substance
valoctocogene
roxaparvovec. A gene therapy product works by delivering a gene into
the body to correct a genetic
deficiency.
WHAT ROCTAVIAN IS USED FOR
This medicine is used for the treatment of severe haemophilia A in
adults who do not have current or
past inhibitors to factor VIII and who do not have antibodies to the
virus vector AAV5.
Haemophilia A is a condition where people inherit an altered form of a
gene needed to make
factor VIII, an essential protein required for blood to clot and stop
any bleeding. People with
haemophilia A cannot produce factor VIII and are prone to internal or
external bleeding episodes.
HOW DOES ROCTAVIAN WORK
The active substance in ROCTAVIAN is based on a virus which does not
cause disease in humans.
This virus has been modified so that it cannot spread in the body but
can

Vara einkenni

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
ROCTAVIAN 2 × 10
13
vector genomes/mL solution for infusion
2.
QUALITATIVE AND QUANTITATIVE COMPOSITION
2.1
GENERAL DESCRIPTION
Valoctocogene roxaparvovec is a gene therapy medicinal product that
expresses the B-domain deleted
SQ form of human coagulation factor VIII (hFVIII-SQ). It is a
non-replicating recombinant
adeno-associated virus serotype AAV5 based vector containing the cDNA
of the B-domain deleted
SQ form of human coagulation factor VIII gene under the control of a
liver-specific promoter.
Valoctocogene roxaparvovec is produced in a baculovirus expression
system that derived from cells of
_Spodoptera frugiperda_
(Sf9 cell line) by recombinant DNA technology.
2.2
QUALITATIVE AND QUANTITATIVE COMPOSITION
Each mL of valoctocogene roxaparvovec solution for infusion contains 2
× 10
13
vector genomes.
Each vial contains 16 × 10
13
vector genomes of valoctocogene roxaparvovec in 8 mL solution.
Excipient with known effect
This medicinal product contains 29 mg sodium per vial.
For the full list of excipients, see section 6.1.
3.
PHARMACEUTICAL FORM
Solution for infusion.
A clear, colourless to pale yellow solution with a pH of 6.9 - 7.8 and
an osmolarity of
364 - 445 mOsm/L.
4.
CLINICAL PARTICULARS
4.1
THERAPEUTIC INDICATIONS
ROCTAVIAN is indicated for the treatment of severe haemophilia A
(congenital factor VIII
deficiency) in adult patients without a history of factor VIII
inhibitors and without detectable
antibodies to adeno-associated virus serotype 5 (AAV5).
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 should
be administ