Kalydeco

Country: European Union

Language: English

Source: EMA (European Medicines Agency)

Active ingredient:

ivacaftor

Available from:

Vertex Pharmaceuticals (Ireland) Limited

ATC code:

R07AX02

INN (International Name):

ivacaftor

Therapeutic group:

Other respiratory system products

Therapeutic area:

Cystic Fibrosis

Therapeutic indications:

Kalydeco tablets are indicated:As monotherapy for the treatment of adults, adolescents, and children aged 6 years and older and weighing 25 kg or more with cystic fibrosis (CF) who have an R117H CFTR mutation or one of the following gating (class III) mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R (see sections 4.4 and 5.1).In a combination regimen with tezacaftor/ivacaftor tablets for the treatment of adults, adolescents, and children aged 6 years and older with cystic fibrosis (CF) who are homozygous for the F508del mutation or who are heterozygous for the F508del mutation and have one of the following mutations in the CFTR gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272 26A→G, and 3849+10kbC→T.In a combination regimen with ivacaftor/tezacaftor/elexacaftor tablets for the treatment of adults, adolescents, and children aged 6 years and older with cystic fibrosis (CF) who have at least one F508del mutation in the CFTR gene (see section 5.1).Kalydeco granules are indicated for the treatment of infants aged at least 4 months, toddlers and children weighing 5 kg to less than 25 kg with cystic fibrosis (CF) who have an R117H CFTR mutation or one of the following gating (class III) mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R (see sections 4.4 and 5.1).In a combination regimen with ivacaftor/tezacaftor/elexacaftor for the treatment of cystic fibrosis (CF) in paediatric patients aged 2 to less than 6 years who have at least one F508del mutation in the CFTR gene.

Product summary:

Revision: 38

Authorization status:

Authorised

Authorization date:

2012-07-23

Patient Information leaflet

103
B. PACKAGE LEAFLET
104
PACKAGE LEAFLET: INFORMATION FOR THE PATIENT
KALYDECO 75 MG FILM-COATED TABLETS
KALYDECO 150 MG FILM-COATED TABLETS
ivacaftor
READ ALL OF THIS LEAFLET CAREFULLY BEFORE YOU START TAKING 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 pharmacist.
•
This medicine has been prescribed for you only. Do not pass it on to
others. It may harm them,
even if their signs of illness are the same as yours.
•
If you get any side effects, talk to your doctor or pharmacist. This
includes any possible side
effects not listed in this leaflet. See section 4.
WHAT IS IN THIS LEAFLET
1.
What Kalydeco is and what it is used for
2.
What you need to know before you take Kalydeco
3.
How to take Kalydeco
4.
Possible side effects
5.
How to store Kalydeco
6.
Contents of the pack and other information
1.
WHAT KALYDECO IS AND WHAT IT IS USED FOR
Kalydeco contains the active substance ivacaftor. Ivacaftor acts at
the level of the cystic fibrosis
transmembrane conductance regulator (CFTR), a protein that forms a
channel at the cell surface that
allows the movement of particles such as chloride in and out of the
cell. Due to mutations in the
_CFTR_
gene (see below), chloride movement is reduced in those with cystic
fibrosis (CF). Ivacaftor helps
certain abnormal CFTR proteins open more often to improve chloride
movement in and out of the cell.
Kalydeco tablets are indicated:
•
As monotherapy for patients aged 6 years and older and weighing 25 kg
or more with cystic
fibrosis (CF) who have an
_R117H CFTR_
mutation or one of the following gating mutations in
the
_CFTR_
gene:
_G551D_
,
_G1244E_
,
_G1349D_
,
_G178R_
,
_G551S_
,
_S1251N_
,
_S1255P_
,
_S549N_
or
_S549R_
.
•
In combination with tezacaftor/ivacaftor tablets for patients aged 6
years and older with CF who
have two
_F508del _
mutations in the
_CFTR _
gene (homozygous for the
_F508del _
mutation) or who
have an
_F508del _
mutation

Summary of Product characteristics

1
ANNEX I
SUMMARY OF PRODUCT CHARACTERISTICS
2
1.
NAME OF THE MEDICINAL PRODUCT
Kalydeco 75 mg film-coated tablets
Kalydeco 150 mg film-coated tablets
2.
QUALITATIVE AND QUANTITATIVE COMPOSITION
Kalydeco 75 mg film-coated tablets
Each film-coated tablet contains 75 mg of ivacaftor.
_Excipient with known effect _
_ _
Each film-coated tablet contains 83.6 mg of lactose monohydrate.
Kalydeco 150 mg film-coated tablets
Each film-coated tablet contains 150 mg of ivacaftor.
_Excipient with known effect _
Each film-coated tablet contains 167.2 mg of lactose monohydrate.
For the full list of excipients, see section 6.1.
3.
PHARMACEUTICAL FORM
Film-coated tablet (tablet)
Kalydeco 75 mg film-coated tablets
Light blue, capsule-shaped film-coated tablets, printed with “V
75” in black ink on one side and plain
on the other (12.7 mm × 6.8 mm in modified tablet shape).
Kalydeco 150 mg film-coated tablets
Light blue, capsule-shaped film-coated tablets, printed with “V
150” in black ink on one side and plain
on the other (16.5 mm × 8.4 mm in modified tablet shape).
4.
CLINICAL PARTICULARS
4.1
THERAPEUTIC INDICATIONS
Kalydeco tablets are indicated:
•
As monotherapy for the treatment of adults, adolescents, and children
aged 6 years and older
and weighing 25 kg or more with cystic fibrosis (CF) who have an
_R117H CFTR_
mutation or
one of the following gating (class III) mutations in the cystic
fibrosis transmembrane
conductance regulator (
_CFTR_
) gene:
_G551D_
,
_G1244E_
,
_G1349D_
,
_G178R_
,
_G551S_
,
_S1251N_
,
_S1255P_
,
_S549N_
or
_S549R_
(see sections 4.4 and 5.1).
•
In a combination regimen with tezacaftor/ivacaftor tablets for the
treatment of adults,
adolescents, and children aged 6 years and older with cystic fibrosis
(CF) who are homozygous
for the
_F508del _
mutation or who are heterozygous for the
_F508del_
mutation and have one of the
following mutations in the
_CFTR_
gene:
_P67L, R117C, L206W, R352Q, A455E, D579G, _
3
_711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G,
_
and
_ _
_384