For help on how to get the results you want, see our search tips.
249 results
Medicine
European public assessment reports (EPAR) Remove European public assessment reports (EPAR) filter
Direct healthcare professional communication Remove Direct healthcare professional communication filter
Summaries of opinion Remove Summaries of opinion filter
Orphan designations Remove Orphan designations filter
Medicine type
Exceptional circumstances Remove Exceptional circumstances filter
Orphan medicine Remove Orphan medicine filter
Orphan designation status
Positive Remove Positive filter
-
List item
Orphan designation: Masitinib mesilate for: Treatment of pancreatic cancer
Date of designation: 28/10/2009, Positive, -
List item
Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Treatment of hyperargininaemia
Date of designation: 10/06/2010, Positive, Last updated: 18/11/2022 -
List item
Orphan designation: Gallium (68Ga)-edotreotide for: Diagnosis of gastro-entero-pancreatic neuroendocrine tumours
Date of designation: 19/03/2015, Positive, Last updated: 03/05/2023 -
List item
Orphan designation: Treprostinil diethanolamine for: Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension
Date of designation: 26/08/2005, Positive, Last updated: 13/01/2022 -
List item
Orphan designation: Caffeine citrate for: Prevention of bronchopulmonary dysplasia
Date of designation: 11/04/2014, Positive, -
List item
Orphan designation: 1,3-Propanedisulfonic acid, disodium salt for: Treatment of systemic secondary amyloidosis
Date of designation: 31/07/2001, Positive, -
List item
Orphan designation: Fluocinolone acetonide for: Treatment of non-infectious uveitis affecting the posterior segment of the eye
Date of designation: 07/03/2005, Positive, Last updated: 30/05/2007 -
List item
Orphan designation: Recombinant human alpha-Mannosidase (velmanase alfa) for: Treatment of alpha-mannosidosis
Date of designation: 26/01/2005, Positive, -
List item
Orphan designation: Taliglucerase alfa for: Treatment of Gaucher disease
Date of designation: 23/03/2010, Positive, Last updated: 10/03/2011 -
List item
Orphan designation: Treprostinil sodium for: Treatment of chronic thromboembolic pulmonary hypertension
Date of designation: 08/02/2013, Positive, Last updated: 08/04/2020 -
List item
Orphan designation: Human heterologous liver cells for: Treatment of argininosuccinic aciduria
Date of designation: 17/12/2010, Positive, Last updated: 16/08/2016 -
List item
Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Treatment of ornithine carbamoyltransferase deficiency
Date of designation: 10/06/2010, Positive, Last updated: 18/11/2022 -
List item
Orphan designation: Isavuconazonium sulfate for: Treatment of invasive aspergillosis
Date of designation: 04/07/2014, Positive, Last updated: 21/05/2019 -
List item
Orphan designation: Levamisole (hydrochloride) for: Treatment of nephrotic syndrome
Date of designation: 28/10/2005, Positive, Last updated: 08/08/2006 -
List item
Orphan designation: Nanoliposomal irinotecan for: Treatment of pancreatic cancer
Date of designation: 09/12/2011, Positive, Last updated: 17/12/2018 -
List item
Orphan designation: cytarabine, daunorubicin for: Treatment of acute myeloid leukaemia
Date of designation: 11/01/2012, Positive, Last updated: 08/10/2014 -
List item
Orphan designation: Adenovirus-mediated herpes-simplex-virus thymidine-kinase (HKSV-tk) gene for: Treatment of high-grade glioma with subsequent use of ganciclovir sodium
Date of designation: 06/02/2002, Positive, Last updated: 16/09/2021 -
List item
Orphan designation: Human heterologous liver cells for: Treatment of carbamoyl-phosphate synthase-1 deficiency
Date of designation: 17/12/2010, Positive, Last updated: 16/08/2016 -
List item
Orphan designation: Para-aminosalicylic acid for: Treatment of tuberculosis
Date of designation: 17/12/2010, Positive, Last updated: 08/06/2018 -
List item
Orphan designation: Bardoxolone methyl for: Treatment of Alport syndrome
Date of designation: 25/05/2018, Positive, Last updated: 30/01/2023 -
List item
Orphan designation: Aplidine for: Treatment of multiple myeloma
Date of designation: 16/11/2004, Positive, Last updated: 11/10/2005 -
List item
Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Treatment of citrullinaemia type 1
Date of designation: 10/06/2010, Positive, Last updated: 18/11/2022 -
List item
Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Treatment of argininosuccinic aciduria
Date of designation: 10/06/2010, Positive, Last updated: 18/11/2022 -
List item
Orphan designation: Autologous Tumor-Derived gp96 Heat Shock Protein-Peptide Complex for: Treatment of renal-cell carcinoma
Date of designation: 11/04/2005, Positive, Last updated: 04/01/2006 -
List item
Orphan designation: 1-{3-[3-(4-Chlorophenyl)propoxy]propyl}piperidine, hydrochloride for: Treatment of narcolepsy
Date of designation: 10/07/2007, Positive, Last updated: 20/04/2016