Страна: Европейски съюз
Език: английски
Източник: EMA (European Medicines Agency)
Ataluren
PTC Therapeutics International Limited
M09AX03
ataluren
Other drugs for disorders of the musculo-skeletal system
Muscular Dystrophy, Duchenne
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.
Revision: 22
Authorised
2014-07-31
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 Прочетете целия документ
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 Прочетете целия документ