Translarna

Country: European Union

Language: English

Source: EMA (European Medicines Agency)

Active ingredient:

Ataluren

Available from:

PTC Therapeutics International Limited

ATC code:

M09AX03

INN (International Name):

ataluren

Therapeutic group:

Other drugs for disorders of the musculo-skeletal system

Therapeutic area:

Muscular Dystrophy, Duchenne

Therapeutic indications:

Translarna is indicated for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older. Efficacy has not been demonstrated in non-ambulatory patients.The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing.

Product summary:

Revision: 22

Authorization status:

Authorised

Authorization date:

2014-07-31

Patient Information leaflet

34
B. PACKAGE LEAFLET
35
PACKAGE LEAFLET: INFORMATION FOR THE PATIENT
TRANSLARNA 125 MG GRANULES FOR ORAL SUSPENSION
TRANSLARNA 250 MG GRANULES FOR ORAL SUSPENSION
TRANSLARNA 1000 MG GRANULES FOR ORAL SUSPENSION
ataluren
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 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 Translarna is and what it is used for
2.
What you need to know before you take Translarna
3.
How to take Translarna
4.
Possible side effects
5.
How to store Translarna
6.
Contents of the pack and other information
1.
WHAT TRANSLARNA IS AND WHAT IT IS USED FOR
Translarna is a medicine that contains the active substance ataluren.
Translarna is used to treat Duchenne muscular dystrophy resulting from
a specific genetic defect that
affects normal muscle function.
Translarna is used to treat patients aged 2 years and older, who are
able to walk.
You or your child will have been tested by your doctor before starting
treatment with Translarna, in
order to confirm that your disease is suitable for treatment with this
medicine.
HOW DOES TRANSLARNA WORK?
Duchenne muscular dystrophy is caused by genetic changes that result
in an abnormality in a muscle
protein called dystrophin which is needed for muscles to work
properly. Translarna enables the
production of working dystrophin and helps muscles work properly.
2.
WHAT

Summary of Product characteristics

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
Translarna 125 mg granules for oral suspension
Translarna 250 mg granules for oral suspension
Translarna 1000 mg granules for oral suspension
2.
QUALITATIVE AND QUANTITATIVE COMPOSITION
Translarna 125 mg granules for oral suspension
Each sachet contains 125 mg ataluren.
Translarna 250 mg granules for oral suspension
Each sachet contains 250 mg ataluren.
Translarna 1000 mg granules for oral suspension
Each sachet contains 1000 mg ataluren.
For the full list of excipients, see section 6.1.
3.
PHARMACEUTICAL FORM
Granules for oral suspension.
White to off-white granules.
4.
CLINICAL PARTICULARS
4.1
THERAPEUTIC INDICATIONS
Translarna is indicated for the treatment of Duchenne muscular
dystrophy resulting from a nonsense
mutation in the dystrophin gene, in ambulatory patients aged 2 years
and older (see section 5.1).
The presence of a nonsense mutation in the dystrophin gene should be
determined by genetic testing
(see section 4.4).
4.2
POSOLOGY AND METHOD OF ADMINISTRATION
_ _
Treatment with Translarna should only be initiated by specialist
physicians with experience in the
management of Duchenne/Becker muscular dystrophy.
Posology
Ataluren should be administered orally every day in 3 doses.
The first dose should be taken in the morning, the second at midday,
and the third in the evening.
Recommended dosing intervals are 6 hours between morning and midday
doses, 6 hours between
midday and evening doses, and 12 hours between the evening dose and
the first dose on the next day.
The recommended dose is 10 mg/kg body weight in the morning, 10 mg/kg
body weight at midday,
and 20 mg/kg body weight in the evening (for a total daily dose of 40
mg/kg body weight).
3
Translarna is available in sach