Orphan designation: Autologous CD34+ cells transduced with a lentiviral RNA vector that results in integrated cDNA encoding for functional cystinosin Treatment of cystinosis, 19/02/2021 Positive
Orphan designation: imatinib Treatment of pulmonary arterial hypertension, 21/06/2021 Positive
Orphan designation: Alisitol, retinol palmitate, zinc gluconate Treatment of microvillus inclusion disease, 13/11/2020 Positive
Orphan designation: Adeno-associated viral vector serotype 8 encoding B-domain deleted liver specific codon optimized bioengineered chimeric human porcine factor VIII, under a synthetic hepatic combinatorial bundle promoter Treatment of haemophilia A, 13/04/2022 Positive
Orphan designation: rovatirelin Treatment of spinocerebellar ataxia, 11/11/2022 Positive
EMA systems disruption expected from 11 to 17 April
EMEA-001821-PIP01-15-M06
Opinion/decision on a Paediatric investigation plan (PIP): Trixeo Aerosphere, Budesonide,Formoterol (fumarate),Glycopyrronium bromide, decision type: PM: decision on the application for modification of an agreed PIP, therapeutic area: Pneumology-allergology, PIP number: P/0068/2023
Opinion/decision on a Paediatric investigation plan (PIP): Evrysdi, Risdiplam (RO7034067), decision type: PM: decision on the application for modification of an agreed PIP, therapeutic area: Neurology, PIP number: P/0086/2023
EMEA-002079-PIP01-16-M03