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

Orphan designation: Asp-Arg-Val-Tyr-Ile-His-Pro (Angiotensin 1-7),talfirastide Treatment of Duchenne muscular dystrophy, 19/02/2014 Positive

Orphan designation: Adenovirus associated viral vector serotype 8 containing the human CNGB3 gene Treatment of achromatopsia caused by mutations in the CNGB3 gene, 11/11/2015 Positive

Orphan designation: 2-Hydroxypropyl-β-cyclodextrin Treatment of Niemann-Pick's disease, type C, 26/04/2013 Positive

Orphan designation: Donidalorsen Treatment of hereditary angioedema, 19/02/2024 Positive