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
Orphan designation: Omadacycline Treatment of nontuberculous mycobacterial lung disease, 25/07/2024 Positive
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
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
Orphan designation: nicotinamide mononucleotide Treatment of sickle cell disease, 20/06/2023 Positive
Labelling flexibilities for COVID-19 therapeutics
Questions and answers on labelling flexibilities for COVID-19 vaccines
Orphan designation: Cannabidiol Treatment of Leigh syndrome, 25/07/2023 Positive