For help on how to get the results you want, see our search tips.
61 results
Categories
Human Remove Human filter
Medicine
Orphan designations Remove Orphan designations filter
Periodic safety update report single assessments Remove Periodic safety update report single assessments filter
Orphan designation status
Expired Remove Expired filter
-
List item
Orphan designation: Cladribine for: Treatment of indolent non-Hodgkin's lymphoma
Date of designation: 18/09/2001, Expired, Last updated: 18/06/2014 -
List item
Orphan designation: 1,5-(Butylimino)-1,5-dideoxy, D-glucitol for: Treatment of Gaucher disease
Date of designation: 18/10/2000, Expired, Last updated: 27/02/2013 -
List item
Orphan designation: Bosentan for: Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension
Date of designation: 15/02/2001, Expired, Last updated: 27/02/2013 -
List item
Orphan designation: Anagrelide hydrochloride for: Treatment of essential thrombocythaemia
Date of designation: 30/12/2000, Expired, Last updated: 08/02/2017 -
List item
Orphan designation: Zinc acetate dihydrate for: Treatment of Wilson's disease
Date of designation: 31/07/2001, Expired, Last updated: 06/03/2015 -
List item
Orphan designation: Pirfenidone for: Treatment of idiopathic pulmonary fibrosis
Date of designation: 16/11/2004, Expired, Last updated: 22/03/2021 -
List item
Orphan designation: 4-(3,5-Bis(hydroxy-phenyl)-1,2,4) triazol-1-yl) benzoic acid for: Treatment of chronic iron overload requiring chelation therapy
Date of designation: 13/03/2002, Expired, Last updated: 30/09/2016 -
List item
Orphan designation: Dexrazoxane for: Treatment of anthracycline extravasations
Date of designation: 19/09/2001, Expired, Last updated: 30/09/2016 -
List item
Orphan designation: nitisinone for: Treatment of tyrosinaemia type 1
Date of designation: 30/12/2000, Expired, Last updated: 08/05/2015 -
List item
Orphan designation: Alpha-galactosidase A for: Treatment of Fabry disease
Date of designation: 08/08/2000, Expired, Last updated: 18/11/2011 -
List item
Orphan designation: imatinib mesilate for: Treatment of chronic myeloid leukaemia
Date of designation: 15/02/2001, Expired, Last updated: 10/02/2015 -
List item
Orphan designation: Recombinant human acid alpha-glucosidase for: Treatment of glycogen storage disease type II (Pompe's disease)
Date of designation: 15/02/2001, Expired, Last updated: 10/08/2016 -
List item
Orphan designation: imatinib mesilate for: Treatment of dermatofibrosarcoma protuberans
Date of designation: 26/08/2005, Expired, Last updated: 09/02/2015 -
List item
Orphan designation: Hydroxyurea for: Treatment of sickle cell syndrome
Date of designation: 09/07/2003, Expired, Last updated: 06/08/2017 -
List item
Orphan designation: Alpha-galactosidase A for: Treatment of Fabry disease
Date of designation: 08/08/2000, Expired, Last updated: 18/11/2011 -
List item
Orphan designation: Pegvisomant for: Treatment of acromegaly
Date of designation: 15/02/2001, Expired, Last updated: 27/02/2013 -
List item
Orphan designation: Sorafenib tosilate for: Treatment of hepatocellular carcinoma
Date of designation: 11/04/2006, Expired, Last updated: 31/01/2018 -
List item
Orphan designation: 2-Chloro-9-[2-deoxy-2-fluoro-ß-D-arabinofuranosyl]adenine for: Treatment of acute lymphoblastic leukaemia
Date of designation: 05/02/2002, Expired, Last updated: 10/08/2016 -
List item
Orphan designation: Caffeine citrate for: Treatment of primary apnoea of premature newborns
Date of designation: 18/02/2003, Expired, Last updated: 11/07/2019 -
List item
Orphan designation: stiripentol for: Treatment of severe myoclonic epilepsy in infancy
Date of designation: 06/12/2001, Expired, Last updated: 08/02/2017 -
List item
Orphan designation: trabectedin for: Treatment of ovarian cancer
Date of designation: 17/10/2003, Expired, Last updated: 27/11/2019 -
List item
Orphan designation: carglumic acid for: Treatment of propionic acidaemia
Date of designation: 07/11/2008, Expired, Last updated: 09/06/2021 -
List item
Orphan designation: icatibant acetate for: Treatment of angioedema
Date of designation: 18/02/2003, Expired, Last updated: 12/08/2020 -
List item
Orphan designation: carglumic acid for: Treatment of isovaleric acidaemia
Date of designation: 07/11/2008, Expired, Last updated: 09/06/2021 -
List item
Orphan designation: Eculizumab for: Treatment of paroxysmal nocturnal haemoglobinuria
Date of designation: 17/10/2003, Expired, Last updated: 20/11/2019