Orphan designation: Humanised monoclonal antibody targeting B-cell maturation antigen conjugated with maleimidocaproyl monomethyl auristatin F Treatment of multiple myeloma, 16/10/2017 Withdrawn

Orphan designation: (R)-3-(5-dimethylcarbamoyl-pent-1-enyl)-N-(2-hydroxy-1methylethyl)benzamide Treatment of fragile X syndrome Positive

Orphan designation: 2-(2-(2-[2-(4-Benzothiazol-2-yl-phenoxy)-ethoxy]-ethoxy)-ethoxy)-ethanol Treatment of amyotrophic lateral sclerosis, 20/06/2025 Positive

Orphan designation: sodium 2-(3'(-3-(1-(4-(tert-butyl)benzyl)-4-ethyl-5-oxo-4,5-dihydro-1H-1,2,4-triazol-3-yl)propyl)-4-ethoxy-[1,1'-biphenyl]-3-yl)acetate Treatment of hepatocellular carcinoma, 20/06/2025 Positive

Human medicines European public assessment report (EPAR): Ivabradine Zentiva, ivabradine, Date of authorisation: 11/11/2016, Revision: 7, Status: Authorised

Human medicines European public assessment report (EPAR): Coagadex, human coagulation factor X, Date of authorisation: 16/03/2016, Revision: 11, Status: Authorised

Human medicines European public assessment report (EPAR): Byooviz, ranibizumab, Date of authorisation: 18/08/2021, Revision: 10, Status: Authorised

Human medicines European public assessment report (EPAR): Fymskina, ustekinumab, Date of authorisation: 25/09/2024, Revision: 4, Status: Authorised

PSUSA/00002139/202411

Human medicines European public assessment report (EPAR): Mepact, mifamurtide, Date of authorisation: 06/03/2009, Revision: 21, Status: Authorised