Orphan designation: Humanised IgG1 kappa antibody against serum amyloid A and AL amyloid Treatment of amyloid light-chain amyloidosis, 08/02/2013 Positive
Human medicines European public assessment report (EPAR): Abevmy, bevacizumab, Date of authorisation: 21/04/2021, Revision: 9, Status: Authorised
Human medicines European public assessment report (EPAR): Xadago, safinamide, Date of authorisation: 23/02/2015, Revision: 15, Status: Authorised
Orphan designation: Treatment of progressive familial intrahepatic cholestasis, 17/07/2012 Positive
Orphan designation: 2S, 4R ketoconazole Treatment of Cushing's syndrome, 04/07/2012 Positive
Orphan designation: Flucytosine Treatment of glioma, 22/02/2018 Positive
Orphan designation: Vocimagene amiretrorepvec Treatment of glioma, 22/02/2018 Positive
Orphan designation: Reldesemtiv Treatment of spinal muscular atrophy, 28/06/2019 Positive
Orphan designation: Reldesemtiv Treatment of amyotrophic lateral sclerosis, 28/02/2020 Positive
Orphan designation: Enzastaurin hydrochloride Treatment of Ehlers-Danlos syndrome, 24/02/2022 Positive