Timetable accelerated assessment request for initial marketing authorisation applications
Orphan designation: vatiquinone Treatment of RARS2 syndrome, 17/01/2018 Withdrawn
Orphan designation: vatiquinone Treatment of Alpers-Huttenlocher syndrome, 10/12/2021 Withdrawn
Orphan designation: vatiquinone Treatment of mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes, 24/02/2022 Withdrawn
Orphan designation: perflubron Treatment of respiratory distress syndrome, 09/12/2020 Withdrawn
Orphan designation: C1 esterase inhibitor (human) Treatment in solid organ transplantation, 14/12/2018 Withdrawn
Orphan designation: Treatment of sickle cell disease, 05/08/2013 Withdrawn
Timetable: Extension application - ATMP
HMPC meetings in 2025 and 2026
HMPC: overview of assessment work - priority list