TRANSLARNA 125 mg ORAL SUSPENSION GRANULES

Introduction

Package Leaflet: Information for the Patient

Translarna 125 mg granules for oral suspension

Translarna 250 mg granules for oral suspension

Translarna 1,000 mg granules for oral suspension

ataluren

This medicinal product is subject to additional monitoring, which will allow for quick identification of new safety information. You can help by reporting any side effects you may get. The last section of the leaflet includes information on 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 symptoms 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.

Contents of the pack

1. What is Translarna and what is it used for
2. What you need to know before you take Translarna
3. How to take Translarna
4. Possible side effects
5. Storage of Translarna
6. Contents of the pack and other information

1. What is Translarna and what is it used for

Translarna is a medicinal product that contains the active substance ataluren.

Translarna is used to treat Duchenne muscular dystrophy caused by a genetic defect that affects the normal function of muscles.

Translarna is used to treat patients aged 2 years and older who can walk.

Your doctor should have performed tests on you or your child before starting treatment with Translarna to confirm that your disease is suitable for this medicinal product.

How does Translarna work?

Duchenne muscular dystrophy is caused by genetic changes that produce an anomaly of a muscle protein called dystrophin, which is necessary for muscles to function properly. Translarna makes it possible to produce functional dystrophin and helps muscles to function properly.

2. What you need to know before you take Translarna

Do not take Translarna

• if you are allergic to ataluren or any of the other ingredients of this medicinal product (listed in section 6).

• if you are receiving intravenous antibiotics such as gentamicin, tobramycin or streptomycin.

Warnings and precautions

Your doctor should have performed a blood test to confirm that your disease is suitable for treatment with Translarna. If you have kidney problems, your doctor will perform regular checks on your kidneys.

If you have severe kidney impairment (eGFR <30 ml/min) or if you are receiving dialysis because your kidneys are not working (end-stage renal disease), your doctor will decide if treatment with Translarna is suitable for you.

Your doctor will check your lipid levels (fats such as cholesterol and triglycerides) in your blood and your kidney function every 6 or 12 months. If you are taking corticosteroid medicines, your doctor will check your blood pressure every 6 months.

Children and adolescents

Do not give this medicinal product to children under 2 years of age or weighing less than 12 kg, as it has not been tested in this group of patients.

Other medicines and Translarna

Tell your doctor if you are using, have recently used or might use any other medicines. In particular, do not take Translarna with intravenous antibiotics gentamicin, tobramycin or streptomycin. They may affect your kidney function.

You should tell your doctor if you are taking any of the following medicines:

Some of these medicines have not been tested in combination with Translarna, and your doctor may decide to monitor you closely.

Pregnancy and breastfeeding

If you are pregnant or breastfeeding, think you may be pregnant or are planning to have a baby, ask your doctor for advice before taking this medicine. If you become pregnant while taking Translarna, tell your doctor immediately, as it is not recommended to take Translarna during pregnancy or breastfeeding.

Driving and using machines

If you feel dizzy, do not drive or use machines.

3. How to take Translarna

Follow exactly the administration instructions of this medicinal product given by your doctor or pharmacist. If you are unsure, ask your doctor or pharmacist again.

Translarna is available in sachets of the following concentrations: 125 mg, 250 mg and 1,000 mg of ataluren per sachet. Your doctor or pharmacist will tell you the exact number of sachets and which concentration you should take at any given time.

Your dose of Translarna depends on your body weight. 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 (resulting in a total daily dose of 40 mg/kg body weight).

This medicinal product is taken orally mixed with liquid or with a semi-solid food.

Do not open the sachet until the moment you are going to take it and use the entire contents of the sachet. All the contents of the sachet must be mixed with at least 30 ml of liquid (water, milk, fruit juice) or 3 tablespoons of semi-solid food (yogurt or apple sauce). Mix the prepared dose well before taking it. You can increase the amount of liquid or semi-solid food if you prefer.

Dosing table

Take Translarna orally 3 times a day, in the morning, at midday and in the evening. There should be an interval of 6 hours between the morning dose and the midday dose, 6 hours between the midday dose and the evening dose, and 12 hours between the evening dose and the first dose of the next day. For example, you can take Translarna in the morning at 7:00 with breakfast, at midday at 13:00 with lunch and again in the evening at 19:00 with dinner.

Drink water or other liquids regularly to avoid dehydration while taking Translarna.

If you take more Translarna than you should

If you have taken more Translarna than the recommended dose, contact your doctor.

You may experience headache, nausea, vomiting or diarrhea.

If you forget to take Translarna

If you have forgotten to take Translarna and it has been less than 3 hours since the usual morning or midday dose, or less than 6 hours since the evening dose, you can still take the dose. Remember to take the next dose at the usual time.

If you have forgotten the dose and it has been more than 3 hours since the usual morning or midday dose, or more than 6 hours since the evening dose, do not take the dose. But take the next doses at the usual time.

Do not take a double dose to make up for forgotten doses. It is important that you take the correct dose. Translarna may not be as effective in treating your symptoms if you take more than the recommended dose.

If you stop taking Translarna

Do not stop taking Translarna without talking to your doctor.

If you have any further questions on the use of this medicinal product, ask your doctor.

4. Possible side effects

Like all medicines, this medicine can cause side effects, although not everybody gets them. After taking Translarna you may experience one or more of the following side effects:

Very common side effects (may affect more than 1 in 10 people):

• Vomiting

Common side effects (may affect up to 1 in 10 people):

• Lack of appetite
• High levels of triglycerides in the blood
• Headache
• Nausea
• Weight loss
• High blood pressure
• Cough
• Nosebleeds
• Constipation
• Wind
• Stomach upset
• Stomach pain
• Rash
• Pain in arms or legs
• Chest pain
• Bedwetting
• Blood in the urine
• Fever

Frequency not known (cannot be estimated from the available data):

• Increased blood lipids
• Increased renal function tests

Reporting of side effects

If you experience any side effects, talk to your doctor or pharmacist. This includes any possible side effects not listed in this leaflet. You can also report side effects directly via the national reporting system listed in Appendix V. By reporting side effects, you can help provide more information on the safety of this medicinal product.

5. Storage of Translarna

Keep this medicinal product out of the sight and reach of children.

Do not use this medicinal product after the expiry date which is stated on the packaging and sachet after ‘EXP’. The expiry date refers to the last day of the month shown.

No special storage conditions are required.

Take each prepared dose immediately after preparation. If the prepared dose is refrigerated (2 - 8 °C) it should be discarded if not used within 24 hours of preparation or within 3 hours if stored at room temperature (15 - 30 °C).

Medicines should not be disposed of via wastewater or household waste. Ask your pharmacist how to dispose of medicines no longer required. This will help protect the environment.

6. Contents of the pack and other information

Composition of Translarna

Translarna is available in 3 concentrations, which contain respectively 125 mg, 250 mg and 1,000 mg of the active substance ataluren. The other ingredients (excipients) are: polydextrose (E1200), macrogol, poloxamer, mannitol (E421), crospovidone, hydroxyethyl cellulose, artificial vanilla flavor, (maltodextrin, artificial flavorings and propylene glycol), silica, colloidal (E551) and magnesium stearate.

Appearance and pack contents

Translarna is a white to off-white granule for oral suspension in sachets.

Translarna is available in packs containing 30 sachets.

Marketing authorisation holder and manufacturer

PTC Therapeutics International Limited

5th Floor

3 Grand Canal Plaza

Grand Canal Street Upper

Dublin 4

D04 EE70

Ireland

You can request more information about this medicinal product from the local representative of the marketing authorisation holder:

Manufacturer

Almac Pharma Services

22 Seagoe Industrial Estate

Craigavon BT63 5QD United Kingdom

PTC Therapeutics International Limited

5th Floor

3 Grand Canal Plaza

Grand Canal Street Upper

Dublin 4

D04 EE70

Ireland

Almac Pharma Services (Ireland) Limited

Finnabair Industrial Estate

Dundalk, Co. Louth, A91 P9KD

Ireland

Date of last revision of this leaflet:

This medicinal product has been authorised with a ‘conditional approval’. This type of approval means that more information on this medicinal product is expected.

The European Medicines Agency will review the new information on this medicinal product at least once a year and this leaflet will be updated as necessary.

Detailed information on this medicinal product is available on the European Medicines Agency web site: http://www.ema.europa.eu. There are also links to other web sites about rare diseases and orphan medicines.