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
Orphan designation: mitapivat sulfate Treatment of thalassaemia intermedia and major, 12/01/2024 Positive
Orphan designation: Thiophene methylimidazole pentahydrogen Diagnosis of glioma, 12/01/2024 Positive