For help on how to get the results you want, see our search tips.
8 results
Categories
Human Remove Human filter
-
List item
Orphan designation: 2-(hydroxymethyl)-2-(methoxymethyl)-1-azabicyclo[2.2.2]octan-3-one for: Treatment of myelodysplastic syndromes
Date of designation: 25/07/2019, Positive, Last updated: 13/10/2020 -
List item
Orphan designation: Mavorixafor for: Treatment of WHIM syndrome
Date of designation: 25/07/2019, Positive, Last updated: 13/10/2021 -
List item
Orphan designation: N-((R)-2,3-dihydroxypropoxyl)-3,4-difluro-2-(2-fluoro-4-iodo-phenylamino)-benzamide for: Treatment of neurofibromatosis type 1
Date of designation: 25/07/2019, Positive, Last updated: 13/10/2021 -
List item
Orphan designation: Pevonedistat for: Treatment of acute myeloid leukaemia
Date of designation: 25/07/2019, Withdrawn, Last updated: 18/02/2022 -
List item
Orphan designation: Recombinant mutated extracellular domain of the human acetylcholine receptor subunit alpha1 for: Treatment of myasthenia gravis
Date of designation: 25/07/2019, Positive, Last updated: 24/09/2019 -
List item
Orphan designation: Elafibranor for: Treatment of primary biliary cholangitis
Date of designation: 25/07/2019, Positive, Last updated: 08/09/2022 -
List item
Orphan designation: Parsaclisib for: Treatment of marginal zone lymphoma
Date of designation: 25/07/2019, Withdrawn, Last updated: 13/12/2022 -
List item
Orphan designation: 7-ethyl-10-hydroxy-camptothecin for: Treatment of soft tissue sarcoma (updated)
Date of designation: 25/07/2019, Positive, Last updated: 09/06/2023