Orphan designation: Metreleptin Treatment of Berardinelli-Seip syndrome, 17/07/2012 Positive

Orphan designation: Metreleptin Treatment of Barraquer-Simons syndrome, 17/07/2012 Positive

Orphan designation: Metreleptin Treatment of familial partial lipodystrophy, 17/07/2012 Positive

Orphan designation: Humanised IgG4 monoclonal antibody against A proliferation-inducing ligand Treatment of primary IgA nephropathy, 21/06/2022 Positive

Orphan designation: Adeno-associated virus serotype 5 containing the human RDH12 gene Treatment of RDH12 mutation associated retinal dystrophy, 19/10/2020 Positive

Orphan designation: adenovirus associated viral vector serotype 2/8 containing the human CNGA3 gene (entacingene turiparvovec) Treatment of achromatopsia, 31/07/2018 Positive

Orphan designation: Adenovirus associated viral vector serotype 5 containing the human RPE65 gene Treatment of Leber's congenital amaurosis, 11/11/2015 Positive

Orphan designation: Adenovirus associated viral vector serotype 8 containing the human AIPL1 gene Treatment of Leber's congenital amaurosis, 12/12/2017 Positive

Orphan designation: Relacorilant Treatment of Cushing's syndrome, 29/05/2019 Positive

Orphan designation: Relacorilant Treatment of pancreatic cancer, 21/08/2019 Positive