Orphan designation: Autologous CD3-positive T-cells expressing a chimeric antigen receptor against B cell maturation agent Treatment of AL amyloidosis, 19/02/2024 Positive

Orphan designation: Human IgG1 monoclonal antibody targeting amyloid transthyretin Treatment of ATTR amyloidosis, 19/02/2024 Positive

Orphan designation: Recombinant human heparan-N-sulfatase Treatment of mucopolysaccharidosis type IIIA (Sanfilippo A syndrome), 12/01/2024 Positive

Orphan designation: Ziftomenib Treatment of acute myeloid leukaemia, 12/01/2024 Positive

Orphan designation: Apilimod dimesilate Treatment of amyotrophic lateral sclerosis, 12/01/2024 Positive

Orphan designation: Adeno-associated viral vector serotype 9 containing the human MECP2 gene, an intron encoding a miRNA generating sequence, and complementary miRNA binding sites Treatment of Rett syndrome, 12/01/2024 Positive

Orphan designation: cutamesine alpha-thalassaemia X-linked intellectual disability syndrome (due to mutations in the ATRX gene), 12/01/2024 Positive

Orphan designation: Tarlatamab Treatment of small cell lung cancer, 12/01/2024 Positive

Human medicines European public assessment report (EPAR): Entecavir Viatris (previously Entecavir Mylan), entecavir, Date of authorisation: 18/09/2017, Revision: 8, Status: Authorised

Orphan designation: motixafortide Treatment of patients undergoing haematopoietic stem cell transplantation, 12/01/2024 Positive