Orphan designation: milademetan tosilate monohydrate Treatment of soft tissue sarcoma, 20/03/2017 Positive

Orphan designation: Recombinant adeno-associated viral vector serotype 9 containing the human N-alpha-acetylglucosaminidase gene Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome), 12/01/2017 Positive

Orphan designation: ganglioside GM1 Treatment of amyotrophic lateral sclerosis, 13/04/2021 Positive

Orphan designation: Rilonacept Treatment of idiopathic pericarditis, 06/01/2021 Positive

Orphan designation: Soticlestat Treatment of Lennox-Gastaut syndrome, 12/11/2021 Positive

European Shortages Monitoring Platform training session on routine shortage reporting for marketing authorisation holders of CAPs, Online, European Medicines Agency, Amsterdam, the Netherlands, Broadcast, from 20 November 2024, 10:00 (CET) to 20 November 2024, 12:00 (CET)

Orphan designation: (S)-N-(1-(3-Fluoro-2'-methoxy-[1,1'-biphenyl]-4-yl)-2-oxopiperidin-3-yl)-5-(pyridin-2-yl)thiophene-2-sulfonamide Treatment of soft-tissue sarcoma, 13/12/2023 Positive

Orphan designation: Recombinant self-complementary adeno-associated viral vector serotype 9 containing the human CLN3 gene Treatment of neuronal ceroid lipofuscinosis, 21/08/2019 Positive

Orphan designation: Recombinant self-complementary adeno-associated viral vector serotype 9 containing the human CLN6 gene Treatment of neuronal ceroid lipofuscinosis, 21/08/2019 Positive

Orphan designation: 11-(2-pyrrolidin-1-yl-ethoxy)-14,19-dioxa-5,7,26-triaza-tetracyclo[19.3.1.1(2,6).1(8,12)] heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaene Treatment of primary myelofibrosis, 25/08/2010 Positive