Orphan designation: Recombinant adeno-associated viral vector encoding a human micro-dystrophin gene under the control of a muscle specific promoter Treatment of Duchenne muscular dystrophy, 14/10/2016 Positive

Orphan designation: Olorofim Treatment of invasive Scopulariopsis, 14/01/2022 Positive

Orphan designation: melatonin Treatment of perinatal asphyxia, 02/04/2012 Positive

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