Orphan designation: vonafexor Treatment of Alport syndrome, 25/07/2023 Positive
Submitted by Anonymous (not verified) on 1 February 2024 - 9:31
Orphan designation: vonafexor Treatment of Alport syndrome, 25/07/2023 Positive
Orphan designation: Certepetide Treatment of pancreatic cancer, 13/10/2023 Positive
Submitted by Anonymous (not verified) on 1 February 2024 - 9:21
Orphan designation: Certepetide Treatment of pancreatic cancer, 13/10/2023 Positive
Orphan designation: taldefgrobep alfa Treatment of spinal muscular atrophy, 25/07/2023 Positive
Submitted by Anonymous (not verified) on 1 February 2024 - 9:19
Orphan designation: taldefgrobep alfa Treatment of spinal muscular atrophy, 25/07/2023 Positive
Orphan designation: Indole-3-carboxaldehyde Treatment of primary CTLA-4 checkpoint related immunodeficiencies, 20/03/2023 Positive
Submitted by Anonymous (not verified) on 1 February 2024 - 9:13
Orphan designation: Indole-3-carboxaldehyde Treatment of primary CTLA-4 checkpoint related immunodeficiencies, 20/03/2023 Positive
Orphan designation: sildenafil citrate Treatment of Leigh syndrome, 13/10/2023 Positive
Submitted by Anonymous (not verified) on 1 February 2024 - 9:10
Orphan designation: sildenafil citrate Treatment of Leigh syndrome, 13/10/2023 Positive
Orphan designation: Allogeneic peripheral blood-derived haematopoietic stem and progenitor cells, regulatory T cells and conventional T cells Treatment in haematopoietic stem cell transplantation, 25/07/2023 Positive
Submitted by Anonymous (not verified) on 1 February 2024 - 9:00
Orphan designation: Allogeneic peripheral blood-derived haematopoietic stem and progenitor cells, regulatory T cells and conventional T cells Treatment in haematopoietic stem cell transplantation, 25/07/2023 Positive
Orphan designation: Omadacycline Treatment of nontuberculous mycobacterial lung disease, 25/07/2024 Positive
Submitted by Anonymous (not verified) on 1 February 2024 - 9:00
Orphan designation: Omadacycline Treatment of nontuberculous mycobacterial lung disease, 25/07/2024 Positive
EMEA-002999-PIP01-21
Submitted by Anonymous (not verified) on 1 February 2024 - 8:35
EMEA-002999-PIP01-21
Products Management Services (PMS) - Implementation of International Organization for Standardization (ISO) standards for the identification of medicinal products (IDMP) in Europe - Chapter 7
Submitted by Anonymous (not verified) on 30 January 2024 - 16:38
Products Management Services (PMS) - Implementation of International Organization for Standardization (ISO) standards for the identification of medicinal products (IDMP) in Europe - Chapter 7
Orphan designation: 5-(3,4-Dichloro-phenyl)-N-((1R,2R)-2-hydroxy-cyclohexyl)-6-(2,2,2-trifluoro-ethoxy)-nicotinamide Treatment of Alport syndrome, 20/06/2023 Positive
Submitted by Anonymous (not verified) on 30 January 2024 - 16:21
Orphan designation: 5-(3,4-Dichloro-phenyl)-N-((1R,2R)-2-hydroxy-cyclohexyl)-6-(2,2,2-trifluoro-ethoxy)-nicotinamide Treatment of Alport syndrome, 20/06/2023 Positive