ACT EU consolidated advice pilots: information and training webinar for Applicants, Online, European Medicines Agency, Amsterdam, the Netherlands, Broadcast, from 17 July 2024, 15:00 (CEST) to 17 July 2024, 17:00 (CEST)

Orphan designation: 4-(4-Methyl-piperazin-1-yl)-N-{6-[2-(4-trifluoromethyl-benzyloxy)-ethoxy]-1H-indazol-3-yl}-benzamide hemioxalate Treatment of acute myeloid leukaemia, 21/03/2024 Positive

Orphan designation: Mibavademab Treatment of Lawrence syndrome, 21/03/2024 Positive

Orphan designation: Sevasemten Treatment of Becker muscular dystrophy, 21/03/2024 Positive

Orphan designation: zatolmilast Treatment of fragile X syndrome, 21/03/2024 Positive

Orphan designation: Mibavademab Treatment of Berardinelli-Seip syndrome (congenital generalised lipodystrophy), 21/03/2024 Positive

Orphan designation: Autologous CD3-positive T-cells expressing a chimeric antigen receptor against B cell maturation agent Treatment of multiple myeloma, 21/03/2024 Positive

New product information wording – Extracts from PRAC recommendations on signals adopted at the 13-16 May 2024 PRAC

EMEA-003369-PIP01-22

EMEA-003366-PIP01-22