Orphan designation: 6-(4-(tert-butyl)phenoxy)pyridin-3-amine Treatment of acute lymphoblastic leukaemia, 10/12/2021 Withdrawn
Orphan designation: Marstacimab Treatment of haemophilia B, 13/12/2023 Withdrawn
Orphan designation: H-Lys-Lys-Gly-Asp-Asn-Ile-Met-Val-Thr-Phe-Arg-Asn-Gln-Ala-Ser-Arg-Pro-Tyr-Gly-Lys-Lys-OH Treatment of haemophilia A, 10/08/2022 Withdrawn
Orphan designation: glecirasib Treatment of pancreatic cancer, 10/11/2024 Positive
Orphan designation: octreotide hydrochloride Treatment of autosomal dominant polycystic liver disease, 10/11/2024 Positive
Nitrosamine impurities in specific medicines
Scientific review on the risk of nitrosamine impurities in human medicines
Nitrosamine impurities
Orphan designation: N-[(1R)-1-phenylethyl]-6-{1H-pyrazolo[3,4-d]pyrimidin-4-yl}quinazolin-2-amine Treatment of fragile X syndrome, 20/04/2017 Withdrawn
Orphan designation: Live attenuated Listeria monocytogenes transfected with plasmids encoding the HPV-16E7 protein fused to a truncated fragment of the Lm protein listeriolysin O Treatment of anal cancer, 11/01/2016 Withdrawn