Orphan designation: 11-(2-pyrrolidin-1-yl-ethoxy)-14,19-dioxa-5,7,26-triaza-tetracyclo[19.3.1.1(2,6).1(8,12)] heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaene Treatment of post-polycythaemia vera myelofibrosis, 25/08/2010 Positive

Orphan designation: Treprostinil sodium Treatment of idiopathic pulmonary fibrosis, 16/03/2022 Positive

Orphan designation: 11-(2-pyrrolidin-1-yl-ethoxy)-14,19-dioxa-5,7,26-triaza-tetracyclo[19.3.1.1(2,6).1(8,12)] heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaene Treatment of post-essential thrombocythaemia myelofibrosis, 25/08/2010 Positive

Orphan designation: Omaveloxolone Treatment of Friedreich’s ataxia, 27/06/2018 Positive

Information on the Member States requirement for the nomination of a pharmacovigilance (PhV) contact person at national level

Orphan designation: Atrasentan Treatment of primary IgA nephropathy, 10/12/2021 Positive

Annual report of the Pharmacovigilance Inspectors Working Group for 2023

Annual report of the Pharmacovigilance Inspectors Working Group for 2023

Orphan designation: humanised IgG1 monoclonal antibody against annexin-A1 Treatment of pancreatic cancer, 15/02/2023 Positive

Orphan designation: dry extract from birch bark (DER 5-10 : 1), extraction solvent n-heptane 95% (w/w) (birch bark extract) Treatment of epidermolysis bullosa, 23/02/2011 Positive