Orphan designation: ultevursen Treatment of retinitis pigmentosa, 22/02/2018 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
Orphan designation: Fosmanogepix Treatment of invasive aspergillosis, 18/07/2022 Positive
Orphan designation: Fosmanogepix Treatment of invasive candidiasis, 18/07/2022 Positive
Orphan designation: erlotinib Treatment of pachyonychia congenita, 18/07/2022 Positive
Orphan designation: Fosmanogepix Treatment of scedosporiosis, 20/03/2023 Positive
Orphan designation: Fosmanogepix Treatment of lomentosporiosis, 15/02/2023 Positive