EMEA-003274-PIP01-22

EMEA-003271-PIP01-22

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: Antisense oligonucleotide complementary to the exonic splicer enhancer sequence at intron 26 of the centrosomal protein 290 pre-mRNA Treatment of Leber's congenital amaurosis, 28/04/2016 Positive

Orphan designation: ultevursen Treatment of retinitis pigmentosa, 22/02/2018 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: erlotinib Treatment of patients with Olmsted syndrome, 09/12/2020 Positive

Orphan designation: Elamipretide Treatment of Barth syndrome, 20/05/2021 Positive

Orphan designation: Ibrexafungerp Treatment of invasive candidiasis, 12/11/2021 Positive

Orphan designation: Epetraborole Treatment of nontuberculous mycobacterial lung disease, 10/08/2022 Positive