Ορφανά φάρμακα (ΕΜΕΑ) που εισάγονται μέσω ΙΦΕΤ
Ταξινόμηση κατά δραστική ουσία
Επιλέξτε τη δραστική ουσία για να δείτε την Περίληψη των Χαρακτηριστικών χρήσης του προϊόντος (Spc)
EMΠOPIKΗ ΟΝΟΜΑΣΙΑ
ΔPAΣTIKH ΟΥΣΙΑ
Designated Orphan Indication
SPONSOR
ΔΙΑΘΕΣΗ
Endocrinology / metabolism
CYSTADANE Betaine anhydrous Treatment of homocystinuria Orphan Europe S.a.r.l. ΕΙΔΙΚΗ ΠΑΡΑΓΓΕΛΙΑ
CARBAGLU Carglumic acid Treatment of N-acetylglutamate synthetase (NAGS) deficiency Orphan Europe S.a.r.l. ΕΙΔΙΚΗ ΠΑΡΑΓΓΕΛΙΑ
KUVAN sapropterin dihydrochloride Phenylketonurias Merck Serono Europe Ltd. ΕΙΔΙΚΗ ΠΑΡΑΓΓΕΛΙΑ
NAGLAZYME Galsulfase Treatment of Mucopolysaccharidosis, type VI (Maroteaux-Lamy Syndrome) BioMarin Europe Ltd ΕΙΔΙΚΗ ΠΑΡΑΓΓΕΛΙΑ
ORFADIN Nitisinone Treatment of tyrosinaemia type I Swedish Orphan International AB ΕΙΔΙΚΗ ΠΑΡΑΓΓΕΛΙΑ
WILZIN Zinc acetate dihydrate Treatment of Wilson/s disease Orphan Europe S.a.r.l. STOCK
Haematology
SOLIRIS Eculizumab Treatment of atypical haemolytic uraemic syndrome (aHUS) Alexion Europe SAS ΕΙΔΙΚΗ ΠΑΡΑΓΓΕΛΙΑ
SOLIRIS Eculizumab Treatment of paroxysmal nocturnal haemoglobinuria Alexion Europe SAS ΕΙΔΙΚΗ ΠΑΡΑΓΓΕΛΙΑ
Cardiovascular & respiratory
ESBRIET Pirfenidone Treatment of idiopathic pulmonary fibrosis Intermune ΕΙΔΙΚΗ ΠΑΡΑΓΓΕΛΙΑ
KALYDECO ivacaftor Kalydeco is indicated for the treatment of cystic fibrosis (CF) in patients age six years and older who have a G551D mutation in the CFTR gene (see sections 4.4 and 5.1). Vertex Pharmaceuticals (U.K.) Ltd. ΕΙΔΙΚΗ ΠΑΡΑΓΓΕΛΙΑ
Para-aminosalicylic acid Lucane Para-aminosalicylic acid Treatment of tuberculosis Lucane Pharma STOCK
PEDEA Ibuprofen Treatment of patent ductus arteriosus Orphan Europe S.a.r.l. ΕΙΔΙΚΗ ΠΑΡΑΓΓΕΛΙΑ
Rare nervous system disease
DIACOMIT Stiripentol Treatment of severe myoclonic epilepsy in infancy Biocodex ΕΙΔΙΚΗ ΠΑΡΑΓΓΕΛΙΑ
FIRDAPSE 3,4 diaminopyridine phosphate Treatment of Lambert-Eaton myasthenic syndrome BioMarin Europe Ltd ΕΙΔΙΚΗ ΠΑΡΑΓΓΕΛΙΑ
Rare oncologic disease
COMETRIQ Cyclopropane-1,1-dicarboxylic acid [4-(6,7-dimethoxy-quinolin-4-yloxy)-phenyl]-amide (4-fluoro-phenyl)-amide, (L)-malate salt Treatment of medullary thyroid carcinoma Swedish Orphan Biovitrum S.r.l. ΕΙΔΙΚΗ ΠΑΡΑΓΓΕΛΙΑ
ICLUSIG Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] Treatment of acute lymphoblastic leukaemia Ariad Pharmaceuticals ΕΙΔΙΚΗ ΠΑΡΑΓΓΕΛΙΑ
ICLUSIG Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] Treatment of chronic myeloid leukaemia Ariad Pharmaceuticals ΕΙΔΙΚΗ ΠΑΡΑΓΓΕΛΙΑ
LITAK Cladribine Treatment of indolent non-Hodgkin’s lymphoma Lipomed ΕΙΔΙΚΗ ΠΑΡΑΓΓΕΛΙΑ
TEPADINA thiotepa In combination with other chemotherapy medicinal products:
1) with or without total body irradiation (TBI), as conditioning treatment prior to allogeneic or autologous haematopoietic progenitor cell transplantation (HPCT) in haematological diseases in adu
Adienne S.r.l. ΕΙΔΙΚΗ ΠΑΡΑΓΓΕΛΙΑ