For help on how to get the results you want, see our search tips.
49 results
Categories
Human Remove Human filter
-
List item
Orphan designation: tafamidis for: Treatment of senile systemic amyloidosis
Date of designation: 08/11/2012, Positive, Last updated: 25/02/2020 -
List item
Orphan designation: Eculizumab for: Treatment of neuromyelitis optica spectrum disorders
Date of designation: 05/08/2013, Positive, Last updated: 20/11/2019 -
List item
Orphan designation: Eculizumab for: Treatment myasthenia gravis
Date of designation: 29/07/2014, Positive, Last updated: 20/11/2019 -
List item
Orphan designation: Recombinant human tissue non-specific alkaline phosphatase - Fc - deca-aspartate fusion protein for: Treatment of hypophosphatasia
Date of designation: 04/12/2008, Positive, Last updated: 20/11/2019 -
List item
Orphan designation: Recombinant human lysosomal acid lipase for: Treatment of lysosomal acid lipase deficiency
Date of designation: 17/12/2010, Positive, Last updated: 20/11/2019 -
List item
Orphan designation: Eculizumab for: Treatment of atypical haemolytic uraemic syndrome (aHUS)
Date of designation: 24/07/2009, Positive, Last updated: 20/11/2019 -
List item
Orphan designation: Lonafarnib for: Treatment of Hutchinson-Gilford progeria
Date of designation: 14/12/2018, Positive, Last updated: 01/08/2019 -
List item
Orphan designation: Phosphorothioate oligonucleotide targeted to transthyretin (inotersen) for: Treatment of ATTR amyloidosis
Date of designation: 26/03/2014, Positive, Last updated: 17/05/2019 -
List item
Orphan designation: Human coagulation factor X for: Treatment of hereditary factor X deficiency
Date of designation: 14/09/2007, Positive, Last updated: 26/03/2019 -
List item
Orphan designation: Antisense oligonucleotide targeted to the SMN2 gene (nusinersen) for: Treatment of 5q spinal muscular atrophy
Date of designation: 02/04/2012, Positive, Last updated: 16/10/2018 -
List item
Orphan designation: Daratumumab for: Treatment of plasma-cell myeloma
Date of designation: 17/07/2013, Positive, Last updated: 02/10/2018 -
List item
Orphan designation: ciclosporin for: Treatment of vernal keratoconjunctivitis
Date of designation: 06/04/2006, Positive, Last updated: 12/07/2018 -
List item
Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA (also known as patisiran) for: Treatment of transthyretin-mediated amyloidosis
Date of designation: 06/04/2017, Positive, Last updated: 30/05/2018 -
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: Budesonide for: Treatment of eosinophilic oesophagitis
Date of designation: 05/08/2013, Positive, Last updated: 18/01/2018 -
List item
Orphan designation: Chenodeoxycholic acid for: Treatment of inborn errors in primary bile acid synthesis
Date of designation: 16/12/2014, Positive, Last updated: 29/06/2017 -
List item
Orphan designation: defibrotide for: Treatment of hepatic veno-occlusive disease
Date of designation: 29/07/2004, Positive, Last updated: 17/08/2016 -
List item
Orphan designation: idebenone for: Treatment of Leber's hereditary optic neuropathy
Date of designation: 16/02/2007, Positive, Last updated: 11/04/2016 -
List item
Orphan designation: [Nle4, D-Phe7]-alfa-melanocyte stimulating hormone (afamelanotide) for: Treatment of erythropoietic protoporphyria
Date of designation: 08/05/2008, Positive, Last updated: 11/04/2016 -
List item
Orphan designation: Recombinant human tripeptidyl-peptidase 1 for: Treatment of neuronal ceroid lipofuscinosis type 2
Date of designation: 12/03/2013, Positive, Last updated: 09/02/2016 -
List item
Orphan designation: Recombinant human nerve growth factor (cenegermin) for: Treatment of neurotrophic keratitis
Date of designation: 14/12/2015, Positive, Last updated: 02/02/2016 -
List item
Orphan designation: carfilzomib for: Treatment of multiple myeloma
Date of designation: 03/06/2008, Positive, Last updated: 02/12/2015 -
List item
Orphan designation: Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] (ponatinib) for: Treatment of chronic myeloid leukaemia
Date of designation: 02/02/2010, Positive, Last updated: 23/03/2015 -
List item
Orphan designation: Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] (ponatinib) for: Treatment of acute lymphoblastic leukaemia
Date of designation: 02/02/2010, Positive, Last updated: 23/03/2015