Orphan designation: Parahydroxybenzoic acid Treatment of primary ubiquinone deficiency, 16/04/2025 Positive

Orphan designation: Adeno-associated viral vector serotype LK03 containing the human CFI gene Treatment of primary IgA nephropathy, 16/04/2025 Positive

Orphan designation: Fragment antibody targeting human TfR1 conjugated to phosphorodiamidate morpholino oligomer Treatment of Duchenne muscular dystrophy, 16/04/2025 Positive

EMEA-002924-PIP02-23

Opinion/decision on a Paediatric investigation plan (PIP): Opdualag, nivolumab,Relatlimab, decision type: P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), therapeutic area: Neoplasms benign, malignant and unspecified (incl cysts and polyps), PIP number: P/0169/2024

Opinion/decision on a Paediatric investigation plan (PIP): Rybrevant, amivantamab, decision type: W: decision granting a waiver in all age groups for all conditions or indications, therapeutic area: Neoplasms benign, malignant and unspecified (incl cysts and polyps), PIP number: P/0166/2024

EMEA-002490-PIP01-18

Opinion/decision on a Paediatric investigation plan (PIP): Ztalmy, Ganaxolone, decision type: P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), therapeutic area: Congenital, familial and genetic disorders, PIP number: P/0183/2024

EMEA-002042-PIP03-23

EMEA-001039-PIP04-23