Země: Evropská unie
Jazyk: angličtina
Zdroj: EMA (European Medicines Agency)
cerliponase alfa
BioMarin International Limited
A16AB
cerliponase alfa
Other alimentary tract and metabolism products,
Neuronal Ceroid-Lipofuscinoses
Brineura is indicated for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency,
Revision: 6
Authorised
2017-05-30
27 B. PACKAGE LEAFLET 28 PACKAGE LEAFLET: INFORMATION FOR THE PATIENT BRINEURA 150 MG SOLUTION FOR INFUSION cerliponase alfa 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 OR YOUR CHILD IS GIVEN 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. - If you or your child get any side effects, talk to your doctor. This includes any possible side effects not listed in this leaflet. See section 4. WHAT IS IN THIS LEAFLET 1. What Brineura is and what it is used for 2. What you need to know before you or your child is given Brineura 3. How Brineura is given 4. Possible side effects 5. How to store Brineura 6. Contents of the pack and other information 1. WHAT BRINEURA IS AND WHAT IT IS USED FOR Brineura contains the active substance cerliponase alfa, which belongs to a group of medicines known as enzyme replacement therapies. It is used to treat patients with neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase-1 (TPP1) deficiency. People with CLN2 disease do not have any enzyme called TPP1 or they have too little of it and this causes a build-up of substances called lysosomal storage materials. In people with CLN2 disease, these materials build-up in certain parts of the body, mainly the brain. HOW BRINEURA WORKS This medicine replaces the missing enzyme, TPP1, which minimises the build-up of the lysosomal storage materials. This medicine works to slow the progression of the disease. 2. WHAT YOU NEED TO KNOW BEFORE YOU OR YOUR CHILD IS GIVEN BRINEURA YOU MUST NOT RECEIVE BRINEURA - If you or your child has had life-threatening allergic reactions to cerliponase alfa or any of the other ingredients of this medicine (listed in section 6), and the Přečtěte si celý dokument
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 Brineura 150 mg solution for infusion 2. QUALITATIVE AND QUANTITATIVE COMPOSITION Each vial of Brineura contains 150 mg of cerliponase alfa* in 5 ml of solution. Each ml of solution for infusion contains 30 mg of cerliponase alfa. *Produced in mammalian Chinese Hamster Ovary cells. Excipient with known effect: Each vial contains 17.4 mg of sodium in 5 ml of solution. For the full list of excipients, see section 6.1. 3. PHARMACEUTICAL FORM Solution for infusion. Clear to slightly opalescent and colourless to pale yellow solution, that may occasionally contain thin translucent fibres or opaque particles. 4. CLINICAL PARTICULARS 4.1 THERAPEUTIC INDICATIONS Brineura is indicated for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency. 4.2 POSOLOGY AND METHOD OF ADMINISTRATION Brineura must only be administered by a trained healthcare professional knowledgeable in intracerebroventricular administration in a healthcare setting. Posology The recommended dose is 300 mg cerliponase alfa administered once every other week by intracerebroventricular infusion. In patients less than 2 years of age, lower doses are recommended, see paediatric population section. Pre-treatment of patients with antihistamines with or without antipyretics is recommended 30 to 60 minutes prior to the start of infusion. Continuation of long-term treatment should be subject to regular clinical evaluation whether the benefits are considered to outweigh the potential risks to individual patients. 3 _ _ _Dose adjustments _ Consideration of dose adjustments may be necessary for patients who may not tolerate the infusion. The dose may be reduced by 50% Přečtěte si celý dokument