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
EMEA-003358-PIP01-22