Document Details

Generated Narrative: Bundle TEST PURPOSES ONLY - translarna

Document Subject

Generated Narrative: MedicinalProductDefinition mp9c1ad55578ad6de333b716cfcc4a7d73

identifier: http://ema.europa.eu/identifier/EU/1/13/902/001

type: Medicinal Product

domain: Human use

status: active

legalStatusOfSupply: Medicinal product subject to medical prescription

name

productName: Translarna 125 mg granules for oral suspension

type: Full name

part

part: nan

type: Invented name part

part

part: nan

type: Scientific name part

part

part: nan

type: Strength part

part

part: nan

type: Pharmaceutical dose form part

Usages

-	Country	Jurisdiction	Language
*	EU	EU	en

Document Content

Generated Narrative: Composition composition-en-9c1ad55578ad6de333b716cfcc4a7d73

identifier: http://ema.europa.eu/identifier/EU/1/13/902/001

status: Final

type: Package Leaflet

category: Raw

date: 2022-02-16 13:28:17+0000

author: Organization ACME industry

title: TEST PURPOSES ONLY - translarna

Attesters

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

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.

Do not take Translarna

• If you are allergic to ataluren or any of the other ingredients of this medicine (listed in section 6).
• If you are receiving treatment with certain antibiotics, such as gentamicin, tobramycin, or streptomycin by injection into a vein.

Warnings and precautions
Your doctor must have done a blood test to confirm that your disease is suitable for treatment with Translarna. If you have any kidney problem, your doctor should check your kidney function regularly.

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

Your doctor will test the levels of lipids (fats such as cholesterol and triglycerides) in your blood and your kidney function every 6 to 12 months. Your doctor will monitor your blood pressure every 6 months, if you are taking a corticosteroid medicine.

Children and adolescents Do not give this medicine to children under the age of 2 years 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 taking, have recently taken, or might take any other medicines. In particular do not take Translarna with the antibiotics gentamicin, tobramycin, or streptomycin given by injection. These may affect your kidney function.

Tell your doctor if you are taking any of the following medicines:

Medicine Usually prescribed for acyclovir treatment of chickenpox [varicella] adefovir treatment of chronic hepatitis B and/or HIV atorvastatin lipid-lowering benzylpenicillin severe infections bumetanide treatment or prevention of congestive heart failure captopril treatment or prevention of congestive heart failure ciprofloxacin treatment of infections famotidine treatment of active duodenal ulcer, gastroesophageal reflux disease furosemide treatment or prevention of congestive heart failure methotrexate rheumatoid arthritis, psoriasis olmesartan essential hypertension in adults oseltamivir prevention of influenza phenobarbital sleep-inducing, prevention of seizures pitavastatin lipid-lowering pravastatin lipid-lowering rifampicin treatment for tuberculosis rosuvastatin lipid-lowering sitagliptin type 2 diabetes valsartan treatment or prevention of congestive heart failure

Some of these medicines were not tested together with Translarna and your doctor may decide to monitor you closely.

Pregnancy and breast-feeding If you are pregnant or breast-feeding, 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, consult your doctor immediately as it is recommended not to take Translarna while you are pregnant or breast-feeding.

Driving and using machines If you feel dizzy, do not drive, cycle or use machines.

Always take this medicine exactly as your doctor or pharmacist has told you. Check with them if you are not sure.

Translarna is available in the following sachet strengths: 125 mg, 250 mg and 1000 mg of ataluren per sachet. Your doctor or pharmacist will tell you the exact number of sachets and what strength to take at each 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 (adding up to a total daily dose of 40 mg/kg body weight).

The medicine is taken by mouth mixed in liquid or semi-solid food.

Open the sachet only at the time you are taking the medicine and use the entire amount from the sachet. The full contents of each sachet should be mixed with at least 30 ml of liquid (water, milk, fruit juice) or 3 tablespoons of semi-solid food (yoghurt or apple sauce). Mix the prepared dose well before taking it. The amount of the liquid or semi-solid food can be increased based on your preference.

Posology table

Weight Range (kg) Number of Sachets Morning
Midday Evening mg sachets mg sachets 1mg sachets 125 mg sachets 250 mg sachets 1mg sachets 125 mg sachets 250 mg sachets 1mg sachets 1 0 0 1 0 0 0 1 0 1 0 0 1 0 0 1 1 0 0 1 0 0 1 0 0 1 0 0 1 0 0 1 0 1 1 0 0 1 0 0 1 0 0 2 0 0 1 0 0 1 0 1 2 0 1 1 0 1 1 0 1 2 0 1 1 0 1 1 0 0 3 0 1 1 0 1 1 0 1 3 0 0 2 0 0 2 0 1 3 0 0 2 0 0 2 0 0 0 1 0 2 0 0 2 0 0 1 1 0 3 0 0 3 0 0 1 1 0 3 0 0 3 0 0 2 1 0 3 0 0 3 0 0 3 1 0 0 1 0 0 1 0 3 1 0 0 1 0 0 1 0 0 2 0 0 1 0 0 1 0 1 2 0 1 1 0 1 1 0 1 2 0 1 1 0 1 1 0 2 2

Take Translarna by mouth 3 times per day; in the morning, midday and evening. There should be 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. For example, you might take Translarna at 7:00 AM in the morning with breakfast, at 1:00 PM in the afternoon with lunch, and again at around 7:00 PM in the evening with dinner.

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

If you take more Translarna than you should Contact your doctor if you take more than the recommended dose of Translarna.
You may experience mild headache, nausea, vomiting or diarrhoea.

If you forget to take Translarna If you are late in taking Translarna by less than 3 hours after the morning or midday doses, or by less than 6 hours after the evening dose, take the dose. Remember to take the next dose on time.
If you are late by more than 3 hours after the morning or midday doses, or by more than 6 hours after the evening dose, do not take the dose. But, take the next doses on time.

Do not take a double dose to make up for a forgotten dose. It is important to 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 medicine, ask your doctor.

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

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

• Vomiting

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

• Decreased appetite
• High blood triglyceride levels
• Headache
• Feeling sick
• Weight loss
• High blood pressure
• Cough
• Nosebleed
• Constipation
• Wind
• Stomach discomfort
• Stomach pain
• Rash
• Arm or leg pain
• Chest pain
• Involuntary urination
• Blood in urine
• Fever

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

• Increases in blood lipids
• Increases in test for kidney function

Reporting of side effects If you get 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 medicine.

Keep this medicine out of the sight and reach of children.

Do not use this medicine after the expiry date, which is stated on the carton and sachet after EXP . The expiry date refers to the last day of that month.

This medicine does not require any special storage conditions.

Take each prepared dose immediately after preparation. Discard the prepared dose if not taken within 24 hours of preparation if kept refrigerated (2 8 C), or within 3 hours at room temperature (15 - 30 C).

Do not throw away any medicines via wastewater or household waste. Ask your pharmacist how to throw away medicines you no longer use. These measures will help protect the environment.

What Translarna contains Translarna is available in 3 strengths, each containing 125 mg, 250 mg and 1000 mg of the active substance, called ataluren. The other ingredients are: polydextrose (E1200), macrogol, poloxamer, mannitol (E421), crospovidone, hydroxyethyl cellulose, artificial vanilla flavour (maltodextrin, artificial flavours and propylene glycol), silica, colloidal anhydrous (E551), magnesium stearate.

What Translarna looks like and contents of the pack Translarna is white to off-white granules for oral suspension in sachets. Translarna is available in packs containing 30 sachets.

Marketing Authorisation Holder PTC Therapeutics International Limited 5th Floor 3 Grand Canal Plaza Grand Canal Street Upper Dublin 4 D04 EEIreland

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 EEIreland

Almac Pharma Services (Ireland) Limited Finnabair Industrial Estate Dundalk, Co. Louth, A91P9KD Ireland

For any information about this medicine, please contact the local representative of the Marketing Authorisation Holder:

AT, BE, BG, CY, CZ, DK, DE, EE, EL, ES, HR, HU, IE, IS, IT, LT, LU, LV, MT, NL, NO, PL, PT, RO, SI, SK, FI, SE, UK (NI) PTC Therapeutics International Ltd. (Ireland) +353 (0)1 447 5medinfo@ptcbio.com FR PTC Therapeutics France Tel: +33(0)1 76 70 10 medinfo@ptcbio.com

This leaflet was last revised in

This medicine has been given conditional approval . This means that there is more evidence to come about this medicine. The European Medicines Agency will review new information on this medicine at least every year and this leaflet will be updated as necessary.

Detailed information on this medicine is available on the European Medicines Agency web site: