Orphan designation: adeno-associated virus serotype rh.10 containing the human PKP2 gene Treatment of arrhythmogenic cardiomyopathy caused by pathogenic mutations in the PKP2 gene, 26/02/2025 Positive

Orphan designation: (R)-(3-(2'-cyclopropyl-3-(hydroxymethyl)-[1,1'-biphenyl]-4-yl)pyrrolidin-1-yl)(5-hydroxy-6-methylpyridin-2-yl)methanone Treatment of Olmsted syndrome, 26/02/2025 Positive

Orphan designation: Venetoclax Treatment of diffuse large B-cell lymphoma, 14/10/2016 Withdrawn

Orphan designation: Treatment of X-linked hypohidrotic ectodermal dysplasia (Christ-Siemens-Touraine Syndrome), 26/02/2025 Positive

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

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

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