Orphan designation: macitentan Treatment of idiopathic pulmonary fibrosis, 28/01/2010 Withdrawn
Orphan designation: macitentan Treatment of functional single ventricle congenital heart disease, 20/05/2021 Withdrawn
Orphan designation: macitentan Treatment of chronic thromboembolic pulmonary hypertension, 10/12/2021 Withdrawn
Orphan designation: Mavoglurant Treatment of fragile X syndrome, 10/10/2012 Withdrawn
Orphan designation: humanised IgG2k Fc-modified bispecific monoclonal antibody against CD3 and BCMA Treatment of multiple myeloma, 19/07/2021 Withdrawn
Orphan designation: vutrisiran Treatment of Stargardt's disease, 10/08/2022 Withdrawn
Orphan designation: cedazuridine,decitabine Treatment of myelodysplastic syndromes, 16/08/2023 Withdrawn
Orphan designation: ovine anti-colchicine polyclonal antibody fragments Treatment of colchicine poisoning, 17/12/2010 Withdrawn
Orphan designation: genetically modified serotype 5/3 adenovirus coding for granulocyte-macrophage colony-stimulating factor Treatment of soft tissue sarcoma, 19/06/2013 Withdrawn
Orphan designation: genetically modified serotype 5/3 adenovirus coding for granulocyte macrophage colony-stimulating factor Treatment of malignant mesothelioma, 16/12/2014 Withdrawn