Meeting of the Medicine Shortages Single Point of Contact (SPOC) Working Party, Online, from 15 April 2026, 09:30 (CEST) to 15 April 2026, 13:00 (CEST)
Orphan designation: risvutatug rezetecan Treatment of pulmonary neuroendocrine carcinoma, 20/10/2025 Positive
Orphan designation: fenfluramine hydrochloride Treatment of Rett syndrome, 20/10/2025 Positive
Paediatric investigation plans: questions and answers
Orphan designation: glial cell line-derived neurotrophic factor,sodium butyrate Treatment of Hirschsprung's disease, 20/10/2025 Positive
Orphan designation: Unasnemab Treatment of amyotrophic lateral sclerosis, 20/10/2025 Positive
PSUSA/00001912/202509
Orphan designation: mezagitamab Treatment of primary IgA nephropathy, 20/10/2025 Positive
Orphan designation: sutacimig Treatment of Glanzmann thrombasthenia, 20/10/2025 Positive
Orphan designation: ozisiran Treatment of Glanzmann thrombasthenia, 20/10/2025 Positive