Orphan designation: paltusotine Treatment of acromegaly, 26/02/2025 Positive
Submitted by Anonymous (not verified) on 11 March 2025 - 10:55
Orphan designation: paltusotine Treatment of acromegaly, 26/02/2025 Positive
Orphan designation: adeno-associated virus vector serotype SNY001 containing the human PAH gene Treatment of hyperphenylalaninaemia, 26/02/2025 Positive
Submitted by Anonymous (not verified) on 11 March 2025 - 10:47
Orphan designation: adeno-associated virus vector serotype SNY001 containing the human PAH gene Treatment of hyperphenylalaninaemia, 26/02/2025 Positive
Orphan designation: volixibat potassium Treatment of primary sclerosing cholangitis, 26/02/2025 Positive
Submitted by Anonymous (not verified) on 11 March 2025 - 10:42
Orphan designation: volixibat potassium Treatment of primary sclerosing cholangitis, 26/02/2025 Positive
Orphan designation: epertinib Treatment of amyotrophic lateral sclerosis, 26/02/2025 Positive
Submitted by Anonymous (not verified) on 11 March 2025 - 10:38
Orphan designation: epertinib Treatment of amyotrophic lateral sclerosis, 26/02/2025 Positive
Orphan designation: Autologous CD34+ cell enriched population containing haematopoietic stem and progenitor cells transduced ex vivo with a lentiviral vector encoding the human ADA2 gene Treatment of adenosine deaminase 2 deficiency (DADA2), 27/02/2025 Po
Submitted by Anonymous (not verified) on 11 March 2025 - 10:34
Orphan designation: Autologous CD34+ cell enriched population containing haematopoietic stem and progenitor cells transduced ex vivo with a lentiviral vector encoding the human ADA2 gene Treatment of adenosine deaminase 2 deficiency (DADA2), 27/02/2025 Positive
Human medicines European public assessment report (EPAR): Emcitate, tiratricol, Date of authorisation: 12/02/2025, Status: Authorised
Submitted by Anonymous (not verified) on 11 March 2025 - 10:30
Human medicines European public assessment report (EPAR): Emcitate, tiratricol, Date of authorisation: 12/02/2025, Status: Authorised
Orphan designation: adeno-associated virus sector serotype rh74 containing the human SGCG gene Treatment of limb-girdle muscular dystrophy, 27/02/2025 Positive
Submitted by Anonymous (not verified) on 11 March 2025 - 10:28
Orphan designation: adeno-associated virus sector serotype rh74 containing the human SGCG gene Treatment of limb-girdle muscular dystrophy, 27/02/2025 Positive
Cancer Medicines Forum: December 2024, Online, European Medicines Agency, Amsterdam, the Netherlands, from 16 December 2024, 13:00 (CET) to 16 December 2024, 15:00 (CET)
Submitted by Anonymous (not verified) on 11 March 2025 - 10:18
Cancer Medicines Forum: December 2024, Online, European Medicines Agency, Amsterdam, the Netherlands, from 16 December 2024, 13:00 (CET) to 16 December 2024, 15:00 (CET)
Presentation - Optimizing the dosage of human prescription drugs and biological products for the treatment of oncologic diseases: FDA guidance (Mirat Shah)
Submitted by Anonymous (not verified) on 11 March 2025 - 10:16
Presentation - Optimizing the dosage of human prescription drugs and biological products for the treatment of oncologic diseases: FDA guidance (Mirat Shah)
Minutes of the Cancer Medicines Forum - December 2024
Submitted by Anonymous (not verified) on 11 March 2025 - 10:14
Minutes of the Cancer Medicines Forum - December 2024