Orphan designation: roginolisib Treatment of uveal melanoma, 13/12/2024 Positive
Submitted by Anonymous (not verified) on 13 January 2025 - 14:53
Orphan designation: roginolisib Treatment of uveal melanoma, 13/12/2024 Positive
Human medicines European public assessment report (EPAR): Pepaxti, melphalan flufenamide, Date of authorisation: 17/08/2022, Revision: 5, Status: Authorised
Submitted by Anonymous (not verified) on 13 January 2025 - 14:50
Human medicines European public assessment report (EPAR): Pepaxti, melphalan flufenamide, Date of authorisation: 17/08/2022, Revision: 5, Status: Authorised
Procedure for the preparation of European Union herbal monographs and European Union list entries and appointment of HMPC rapporteurs and peer-reviewers
Submitted by Anonymous (not verified) on 13 January 2025 - 14:40
Procedure for the preparation of European Union herbal monographs and European Union list entries and appointment of HMPC rapporteurs and peer-reviewers
Minutes - PDCO minutes of the 12-15 November 2024 meeting
Submitted by Anonymous (not verified) on 13 January 2025 - 14:28
Minutes - PDCO minutes of the 12-15 November 2024 meeting
Orphan designation: Vosoritide Treatment of hypochondroplasia, 13/12/2024 Positive
Submitted by Anonymous (not verified) on 13 January 2025 - 14:21
Orphan designation: Vosoritide Treatment of hypochondroplasia, 13/12/2024 Positive
Orphan designation: votoplam Treatment of Huntington's disease, 13/12/2024 Positive
Submitted by Anonymous (not verified) on 13 January 2025 - 13:59
Orphan designation: votoplam Treatment of Huntington's disease, 13/12/2024 Positive
Orphan designation: mRNA encoding Cas9-deaminase, single guide RNA against the human TGM1 gene Treatment of autosomal recessive congenital ichthyosis, 13/12/2024 Positive
Submitted by Anonymous (not verified) on 13 January 2025 - 13:51
Orphan designation: mRNA encoding Cas9-deaminase, single guide RNA against the human TGM1 gene Treatment of autosomal recessive congenital ichthyosis, 13/12/2024 Positive
Orphan designation: elebsiran Treatment of hepatitis delta virus infection, 13/12/2024 Positive
Submitted by Anonymous (not verified) on 13 January 2025 - 13:33
Orphan designation: elebsiran Treatment of hepatitis delta virus infection, 13/12/2024 Positive
Orphan designation: adeno-associated virus serotype 5 containing the human RORA gene Treatment of inherited retinal dystrophies due to dysfunction in the ABCA4 gene, 13/12/2024 Positive
Submitted by Anonymous (not verified) on 13 January 2025 - 13:13
Orphan designation: adeno-associated virus serotype 5 containing the human RORA gene Treatment of inherited retinal dystrophies due to dysfunction in the ABCA4 gene, 13/12/2024 Positive
Orphan designation: diazoxide choline Treatment of glycogen storage disease type I, 13/12/2024 Positive
Submitted by Anonymous (not verified) on 13 January 2025 - 11:04
Orphan designation: diazoxide choline Treatment of glycogen storage disease type I, 13/12/2024 Positive