For help on how to get the results you want, see our search tips.
24 results
Medicine
European public assessment reports (EPAR) Remove European public assessment reports (EPAR) filter
Orphan designations Remove Orphan designations filter
Categories
Human Remove Human filter
Orphan designation status
Positive Remove Positive filter
-
List item
Orphan designation: for: Treatment of lysosomal acid lipase deficiency (updated)
Date of first decision: 17/12/2010, Positive, Last updated: 20/11/2019 -
List item
Orphan designation: for: Treatment myasthenia gravis (updated)
Date of first decision: 29/07/2014, Positive, Last updated: 20/11/2019 -
List item
Orphan designation: for: Treatment of neuromyelitis optica spectrum disorders (updated)
Date of first decision: 05/08/2013, Positive, Last updated: 20/11/2019 -
List item
Orphan designation: for: Treatment of atypical haemolytic uraemic syndrome (aHUS) (updated)
Date of first decision: 24/07/2009, Positive, Last updated: 20/11/2019 -
List item
Orphan designation: for: Treatment of beta thalassaemia intermedia and major
Date of first decision: 24/01/2013, Positive, Last updated: 03/06/2019 -
List item
Orphan designation: for: Treatment of ATTR amyloidosis
Date of first decision: 26/03/2014, Positive, Last updated: 17/05/2019 -
List item
Orphan designation: for: Treatment of hereditary factor X deficiency
Date of first decision: 14/09/2007, Positive, Last updated: 26/03/2019 -
List item
Orphan designation: for: Treatment of cystic fibrosis
Date of first decision: 08/07/2008, Positive, Last updated: 08/11/2018 -
List item
Orphan designation: for: Treatment of 5q spinal muscular atrophy
Date of first decision: 02/04/2012, Positive, Last updated: 16/10/2018 -
List item
Orphan designation:
Date of first decision: 17/07/2013, Positive, Last updated: 02/10/2018 -
List item
Orphan designation: for: Treatment of vernal keratoconjunctivitis
Date of first decision: 06/04/2006, Positive, Last updated: 12/07/2018 -
List item
Orphan designation: for: Treatment of transthyretin-mediated amyloidosis
Date of first decision: 06/04/2017, Positive, Last updated: 30/05/2018 -
List item
Orphan designation: for: Treatment of eosinophilic oesophagitis
Date of first decision: 05/08/2013, Positive, Last updated: 18/01/2018 -
List item
Orphan designation: for: Treatment of hereditary angioedema
Date of first decision: 09/10/2015, Positive, Last updated: 28/09/2017 -
List item
Orphan designation: for: Treatment of soft tissue sarcoma
Date of first decision: 12/02/2015, Positive, -
List item
Orphan designation: for: Treatment of neuronal ceroid lipofuscinosis type 2
Date of first decision: 12/03/2013, Positive, Last updated: 09/02/2016 -
List item
Orphan designation: for: Treatment of neurotrophic keratitis
Date of first decision: 14/12/2015, Positive, Last updated: 02/02/2016 -
List item
Orphan designation: for: Treatment of multiple myeloma
Date of first decision: 03/06/2008, Positive, Last updated: 02/12/2015 -
List item
Orphan designation: for: Treatment of chronic myeloid leukaemia
Date of first decision: 02/02/2010, Positive, Last updated: 23/03/2015 -
List item
Orphan designation: for: Treatment of acute lymphoblastic leukaemia
Date of first decision: 02/02/2010, Positive, Last updated: 23/03/2015 -
List item
Orphan designation: for: Treatment of Cushing's syndrome
Date of first decision: 23/04/2012, Positive, Last updated: 12/03/2015 -
List item
Orphan designation: for: Treatment of idiopathic pulmonary fibrosis
Date of first decision: 26/04/2013, Positive, Last updated: 12/03/2015 -
List item
Orphan designation: for: Treatment of Castleman's disease
Date of first decision: 30/11/2007, Positive, Last updated: 11/07/2014 -
List item
Orphan designation: for: Treatment of Gaucher disease
Date of first decision: 06/06/2010, Positive, Last updated: 23/06/2010