PSUSA/00001912/202509
Submitted by Anonymous (not verified) on 14 April 2026 - 15:36
PSUSA/00001912/202509
Orphan designation: mezagitamab Treatment of primary IgA nephropathy, 20/10/2025 Positive
Submitted by Anonymous (not verified) on 14 April 2026 - 15:30
Orphan designation: mezagitamab Treatment of primary IgA nephropathy, 20/10/2025 Positive
Orphan designation: sutacimig Treatment of Glanzmann thrombasthenia, 20/10/2025 Positive
Submitted by Anonymous (not verified) on 14 April 2026 - 15:30
Orphan designation: sutacimig Treatment of Glanzmann thrombasthenia, 20/10/2025 Positive
Orphan designation: ozisiran Treatment of Glanzmann thrombasthenia, 20/10/2025 Positive
Submitted by Anonymous (not verified) on 14 April 2026 - 15:30
Orphan designation: ozisiran Treatment of Glanzmann thrombasthenia, 20/10/2025 Positive
Orphan designation: ceruloplasmin Treatment of congenital aceruloplasminemia, 20/10/2025 Positive
Submitted by Anonymous (not verified) on 14 April 2026 - 15:30
Orphan designation: ceruloplasmin Treatment of congenital aceruloplasminemia, 20/10/2025 Positive
List of industry subject matter experts and list of planned calls for industry subject matter experts
Submitted by Anonymous (not verified) on 14 April 2026 - 15:20
List of industry subject matter experts and list of planned calls for industry subject matter experts
European Medicines Agency meeting with senior representatives of pharmaceutical companies, European Medicines Agency, Amsterdam, the Netherlands, 17 April 2026
Submitted by Anonymous (not verified) on 14 April 2026 - 15:15
European Medicines Agency meeting with senior representatives of pharmaceutical companies, European Medicines Agency, Amsterdam, the Netherlands, 17 April 2026
Orphan designation: nangibotide Treatment of sickle cell disease, 21/11/2025 Positive
Submitted by Anonymous (not verified) on 14 April 2026 - 15:00
Orphan designation: nangibotide Treatment of sickle cell disease, 21/11/2025 Positive
Orphan designation: Felzartamab Treatment of primary membranous nephropathy, 21/11/2025 Positive
Submitted by Anonymous (not verified) on 14 April 2026 - 15:00
Orphan designation: Felzartamab Treatment of primary membranous nephropathy, 21/11/2025 Positive
Orphan designation: Infigratinib Treatment of hypochondroplasia, 21/11/2025 Positive
Submitted by Anonymous (not verified) on 14 April 2026 - 15:00
Orphan designation: Infigratinib Treatment of hypochondroplasia, 21/11/2025 Positive