Table 6 All ODs with MA for the paediatric population
Medicine name | Active substance | Indication | Age range | Authorisation date | Status |
|---|---|---|---|---|---|
Fabrazyme | Agalsidase beta | Fabry disease (galactosidase-A deficiency) | 8 years and older | 03/08/2001 | End of marketing exclusivity |
Replagal | Agalsidase alpha | Fabry disease (galactosidase-A deficiency) | 7 years and older | 03/08/2001 | Authorised under exceptional circumstance, end of marketing exclusivity |
Glivec | Imatinib | Chronic myeloid leukaemia | > 1 and >2 years | 07/11/2001 | Withdrawn OD status |
Tracleer | Bosentan monohydrate | Pulmonary arterial hypertension (PAH) | 2 years and older | 15/05/2002 | Authorised |
Zavesca | Miglustat | Niemann-Pick type-C disease | Children* and adults | 20/11/2002 | Authorised |
Carbaglu | Carglumic acid | Hyperammonaemia due to - N-acetylglutamate-synthase (NAGS) primary deficiency | As early as the first day of life | 24/01/2003 | Authorised, end of marketing exclusivity for NAGS |
- isovaleric acidaemia | |||||
- methymalonic acidaemia | |||||
- propionic acidaemia | |||||
Aldurazyme | Laronidase | Mucopolysaccharidosis I (alpha-L-iduronidase deficiency) | Children* and adults | 10/06/2003 | Authorised under exceptional circumstance, end of marketing exclusivity |
Busilvex | Busulfan | Conditioning treatment prior to conventional haematopoietic progenitor cell transplantation (HPCT) | Newborn and older | 09/07/2003 | End of marketing exclusivity |
Lysodren | Mitotane | Advanced adrenal cortical carcinoma | Children** and adults | 28/04/2004 | Authorised |
Pedea | Ibuprofen | Patent ductus arteriosus | Premature newborns | 29/07/2004 | Authorised |
Wilzin | Zinc | Wilson’s disease | One year and older | 13/10/2004 | Authorised |
Xagrid | Anagrelide | Essential thrombocythaemia | Children** and adults | 16/11/2004 | Authorised under exceptional circumstances |
Orfadin | Nitisinone | Hereditary tyrosinaemia type 1 (HT-1) | Children* and adults | 21/02/2005 | Authorised |
Revatio | Sildenafil | Pulmonary arterial hypertension | one year and older | 28/10/2005 | Authorised |
Naglazyme | Galsulfase | Mucopolysaccharidosis VI (N-acetylgalactosamine-4-sulfatase deficiency; Maroteaux-Lamy syndrome) | Children* and adults | 24/01/2006 | Authorised under exceptional circumstances |
Myozyme | Alglucosidase alpha | Pompe disease (acid-α-glucosidase deficiency) | Children of all ages and adults | 29/03/2006 | Authorised |
Evoltra | Clofarabine | Acute lymphoblastic leukaemia | 1-21 years | 29/05/2006 | Authorised under exceptional circumstances |
Exjade | Deferasirox | Beta thalassaemia major with iron overload | 2 years and older | 28/08/2006 | Authorised |
Diacomit | Stiripentol | Severe myoclonic epilepsy in infancy (SMEI, Dravet's syndrome) | 3 years and older | 04/01/2007 | Conditional approval |
Elaprase | Idursulfase | Hunter syndrome (mucopolysaccharidosis II) | 5 years and older | 08/01/2007 | Authorised under exceptional circumstances |
Inovelon | Rufinamide | Lennox-Gastaut syndrome | 4 years and older | 16/01/2007 | Authorised |
Cystadane | Betaine anhydrous | Homocystinuria | Children* and adults | 15/02/2007 | Authorised |
Soliris | Eculizumab | Paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uremic syndrome (aHUS) | Children* and adults | 20/06/2007 | Authorised |
Siklos | Hydroxycarbamide | Sickle-cell syndrome | 2 years and older | 29/06/2007 | Authorised |
Increlex | Mecasermin | Primary insulin-like-growth-factor-1 deficiency (primary IGFD) | 2 to 18 years | 03/08/2007 | Authorised under exceptional circumstances |
Atriance | Nelarabine | Acute lymphoblastic leukaemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL) | Children* and adults | 22/08/2007 | Authorised under exceptional circumstances |
Kuvan | Sapropterin dihydrochloride | Phenylketonuria (PKU) and tetrahydrobiopterin (BH4) deficiency | 4 years and older | 02/12/2008 | Authorised |
Mepact | Mifamurtide | Osteosarcoma | 2 to 30 years | 06/03/2009 | Authorised |
Peyona | Caffeine citrate | Primary apnoea | Premature newborns | 02/07/2009 | Authorised |
Mozobil | Plerixafor | Lymphoma and multiple myeloma | Children** and adults | 31/07/2009 | Authorised |
Cayston | Aztreonam lysine | Cystic fibrosis (CF) | 6 years and older | 21/09/2009 | Authorised |
Ilaris | Canakinumab | Cryopyrin-Associated Periodic Syndromes (CAPS), and Systemic Juvenile Idiopathic Arthritis (SJIA) | 2 years and older | 23/10/2009 | Authorised under exceptional circumstances, withdrawn OD status |
Tepadina | Thiotepa | Allogeneic or autologous haematopoietic progenitor cell transplantation (HPCT) | Children* and adults | 15/03/2010 | Authorised |
Vpriv | Velaglucerase alpha | Type-1 Gaucher disease | >2 years | 26/08/2010 | Authorised |
Tobi Podhaler | Tobramycin | Cystic fibrosis | 6 years and older | 20/07/2011 | Authorised |
Votubia | Everolimus | Subependymal giant-cell astrocytoma (SEGA) associated with tuberous-sclerosis complex (TSC) | 3 years and older | 02/09/2011 | Conditional approval |
Xaluprine | 6-Mercaptopurine monohydrate | Acute lymphoblastic leukaemia (ALL) | Children* and adults | 09/03/2012 | Authorised |
Kalydeco | Ivacaftor | Cystic fibrosis (CF) with G551D mutation in the CFTR gene | 6 years and older | 23/07/2012 | Authorised |
Novothirteen | Catridecacog | Congenital factor-XIII-A-subunit deficiency | 6 years and above | 03/09/2012 | Withdrawn OD status |
Procysbi | Mercaptamine bitartrate | Nephropathic cystinosis | Children* and adults | 06/09/2013 | Authorised |
Orphacol | Cholic acid | Inborn errors in primary bile-acid synthesis due to 3-hydroxy-5-C27-steroid oxidoreductase deficiency or 4-3-oxosteroid-5-reductase deficiency | One month and older | 12/09/2013 | Authorised under exceptional circumstances |
Defitelio | Defibrotide | Severe hepatic veno-occlusive disease (VOD) in haematopoietic stem-cell transplantation (HSCT) therapy | One month and older | 18/10/2013 | Authorised under exceptional circumstances |