125 mg, 250 mg, 1000 mg granules for oral suspension
ataluren
What Translarna is and what it is used for
Translarna is a medicine that contains the active substance ataluren.
Translaren is used to treat Duchenne muscular dystrophy. This disease is caused by a specific error in the genes that affect normal muscle function.
The translars are used to treat patients who are 2 years of age and older, and who can walk.
You or your child will have been tested by your doctor before starting treatment with Translarna. This is done to confirm that your/your child’s illness is suitable for treatment with this medicine.
How do the Translars work?
Duchenne muscular dystrophy is caused by genetic changes. These changes result in an abnormality in a muscle protein called dystrophin. Dystrophin is needed for muscles to function properly. The translans enable the production of normal dystrophin and help the muscles to function properly.
What you need to know before taking Translarna
Do not take the Translarna
- if you are allergic to ataluren or any of the other ingredients of this medicine (listed in section 6).
- if you receive treatment with certain antibiotics, such as gentamicin, tobramycin, or streptomycin by injection into a vein.
Warnings and precautions
The doctor must have taken a blood test on you to confirm that your disease is suitable for treatment with Translarna. If you have any kidney problems, your doctor should check your kidney function regularly.
If you have serious problems with your kidneys (eGFR <30 ml/min) or if you are being treated with dialysis because your kidneys are not working ( chronic renal failure ), the doctor will decide whether treatment with Translarna is suitable for you.
The doctor will test the levels of lipids (fats such as cholesterol and triglycerides ) in your blood and your kidney function every six to twelve months. The doctor will monitor your blood pressure every 6 months if you are taking a corticosteroid medicine.
Children and young people
Do not give this medicine to children under 2 years of age or with a body weight of less than 12 kg, as it has not been tested in this patient group.
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 together with the antibiotics gentamicin, tobramycin, or streptomycin, which are given by injection. These can affect your kidney function.
Tell your doctor if you are taking any of the following medicines:
| Drug | Commonly prescribed for |
|---|---|
| aciclovir | treatment of chicken pox [varicella] |
| adefovir | treatment of chronic hepatitis B and/or HIV |
| atorvastatin | lipid-lowering |
| benzylpenicillin | serious infection _ |
| bumetanide | treatment or prevention of chronic heart failure |
| captopril | treatment or prevention of chronic heart failure |
| ciprofloxacin | treatment of infection er |
| famotidine | treatment of stomach ulcers, gastroesophageal reflux disease |
| furosemide | treatment or prevention of chronic heart failure |
| methotrexate | rheumatoid arthritis, psoriasis |
| olmesartan | essential hypertension in adults |
| oseltamivir | prevention of influenza |
| phenobarbital | hypnotic, seizure prevention |
| pitavastatin | lipid-lowering |
| pravastatin | lipid-lowering |
| rifampicin | treatment against tuberculosis |
| rosuvastatin | lipid-lowering |
| sitagliptin | type 2- diabetes |
| valsartan | treatment or prevention of chronic heart failure |
Some of these medicines have not been tested with Translarna and doctors 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, consult your doctor before using this medicine. If you become pregnant while taking Translarna, talk to your doctor immediately as it is recommended not to take Translarna while you are pregnant or breastfeeding.
Driving ability and use of machinery
If you feel dizzy, do not drive, cycle or use machinery.
How to take Translarna
Always use this medicine as directed by your doctor or pharmacist. Ask your doctor or pharmacist if you are unsure.
Translarna is available in the following sachet strengths: 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 the strength to take each time.
The transparent dose depends on your body weight. The recommended dose is 10 mg/kg body weight in the morning, 10 mg/kg body weight in the middle of the day, and 20 mg/kg body weight in the evening (for a combined total daily dose of 40 mg/kg body weight).
The medicine is taken orally and mixed with liquid or semi-solid food.
Open the sachet only at the time you take the medicine and use the entire amount in the sachet. The entire 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 (yogurt or applesauce). Mix the prepared dose well before taking it. The amount of liquid or semi-solid food can be increased if you wish.
Dosage table
| Weight range(kg) | Number of sachets | |||||||||
| Morning | Lunch | Evening | ||||||||
| 125 mg sachets | 250 mg sachets | 1,000 mg sachets | 125 mg sachets | 250 mg sachets | 1,000 mg sachets | 125 mg sachets | 250 mg sachets | 1,000 mg sachets | ||
| 12 | 14 | 1 | 0 | 0 | 1 | 0 | 0 | 0 | 1 | 0 |
| 15 | 16 | 1 | 0 | 0 | 1 | 0 | 0 | 1 | 1 | 0 |
| 17 | 20 | 0 | 1 | 0 | 0 | 1 | 0 | 0 | 1 | 0 |
| 21 | 23 | 0 | 1 | 0 | 0 | 1 | 0 | 1 | 1 | 0 |
| 24 | 26 | 0 | 1 | 0 | 0 | 1 | 0 | 0 | 2 | 0 |
| 27 | 31 | 0 | 1 | 0 | 0 | 1 | 0 | 1 | 2 | 0 |
| 32 | 35 | 1 | 1 | 0 | 1 | 1 | 0 | 1 | 2 | 0 |
| 36 | 39 | 1 | 1 | 0 | 1 | 1 | 0 | 0 | 3 | 0 |
| 40 | 44 | 1 | 1 | 0 | 1 | 1 | 0 | 1 | 3 | 0 |
| 45 | 46 | 0 | 2 | 0 | 0 | 2 | 0 | 1 | 3 | 0 |
| 47 | 55 | 0 | 2 | 0 | 0 | 2 | 0 | 0 | 0 | 1 |
| 56 | 62 | 0 | 2 | 0 | 0 | 2 | 0 | 0 | 1 | 1 |
| 63 | 69 | 0 | 3 | 0 | 0 | 3 | 0 | 0 | 1 | 1 |
| 70 | 78 | 0 | 3 | 0 | 0 | 3 | 0 | 0 | 2 | 1 |
| 79 | 86 | 0 | 3 | 0 | 0 | 3 | 0 | 0 | 3 | 1 |
| 87 | 93 | 0 | 0 | 1 | 0 | 0 | 1 | 0 | 3 | 1 |
| 94 | 105 | 0 | 0 | 1 | 0 | 0 | 1 | 0 | 0 | 2 |
| 106 | 111 | 0 | 0 | 1 | 0 | 0 | 1 | 0 | 1 | 2 |
| 112 | 118 | 0 | 1 | 1 | 0 | 1 | 1 | 0 | 1 | 2 |
| 119 | 125 | 0 | 1 | 1 | 0 | 1 | 1 | 0 | 2 | 2 |
Take Translarna by mouth three times a day: in the morning, in the middle of the day, and the evening. There should be 6 hours between the morning and mid-day doses, 6 hours between the mid-day and evening doses, and 12 hours between the evening dose and the first dose the next day. For example, if you take the Translarna at 7.00 in the morning with breakfast, at 13.00 in the afternoon with lunch, and again at approx. 19.00 in the evening with dinner.
Drink water or other fluids regularly to avoid dehydration while taking Translarna.
If you have taken too much Translarna
Contact a doctor if you have taken more than the recommended dose of Translarna.
You may experience mild headache, nausea, vomiting, or diarrhea.
If you have forgotten to take Translarna
If you are late taking Translarna, less than 3 hours after dose one in the morning or mid-day, or less than 6 hours after the evening dose, you should take dose one. Remember to take the next dose on time. If you are late by more than 3 hours after dose one in the morning or the middle of the day, or by more than 6 hours after the evening dose, do not take dose one. However, take the next dose on time.
Do not take a double dose to make up for a missed dose. It is important to take the right 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 first talking to your doctor.
If you have any further questions about this medicine, ask your doctor.
Possible side effects
Like all medicines, this medicine can cause side effects, although not everybody gets them. You may experience 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 triglyceride levels in the blood
- Headache
- Nausea
- Weight loss
- High blood pressure
- Cough
- Nosebleed
- Constipation
- Gases
- Stomach upset
- Pain in the stomach
- Rash
- Arm or leg pain
- Chest pain
- Involuntary voiding of urine
- Blood in the urine
- Fever
Frequency not known (frequency cannot be estimated from available data):
- Increases in blood lipids
- Increases in kidney function tests
How the Translator should be stored
Keep this medicine out of the sight and reach of children.
Use before the expiry date stated on the carton and sachet after EXP. The expiration date is the last day of the specified month.
No special storage instructions.
Take each prepared dose immediately after preparation. Discard the prepared dose if not taken within 24 hours after preparation if stored in a refrigerator (2-8°C), or within 3 hours if stored at room temperature (15-30°C).
The medicine must not be thrown into the drain or among the household waste. Ask the pharmacist how to dispose of medicines that are no longer used. These measures will help to protect the environment.
Contents of the packaging and other information
Contents declaration
Translarna is available in 3 strengths, each containing 125 mg, 250 mg, and 1,000 mg of the active substance, called ataluren. Other ingredients are polydextrose (E1200), macrogol, poloxamer, mannitol (E421), crospovidone, hydroxyethyl cellulose, artificial vanilla flavor (maltodextrin, artificial flavors, and propylene glycol), colloidal anhydrous silicon dioxide (E551), magnesium stearate.
Appearance and package sizes of the medicine
Translarna is a white to off-white granule 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 EE70
Ireland
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
Contact the representative of the Marketing Authorization Holder if you would like to know more about this medicine:
| 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 5165 medinfo@ptcbio.com | FRPTC Therapeutics France Tel: +33(0)1 76 |