EMEA-003379-PIP01-22

Opinion/decision on a Paediatric investigation plan (PIP): Rinvoq, upadacitinib, decision type: P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), therapeutic area: Dermatology, PIP number: P/0446/2023

EMEA-002705-PIP05-23

Opinion/decision on a Paediatric investigation plan (PIP): Reblozyl, Luspatercept, decision type: P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), therapeutic area: Haematology-Hemostaseology, PIP number: P/0441/2023

EMEA-003307-PIP01-22

EMEA-003033-PIP02-22

EMEA-003363-PIP01-22

Orphan designation: H-L-Tryptophanyl-L-seryl-glycyl-L-tryptophanyl-L-seryl-L-seryl-L-cysteinyl-L-seryl-L-arginyl-L-seryl-L-cysteinyl-glycyl-OH (disulfide bond), acetate salt Treatment of amyotrophic lateral sclerosis, 10/11/2024 Positive

Orphan designation: Navenibart Treatment of hereditary angioedema, 10/11/2024 Positive

Orphan designation: Genetically modified human adenovirus encoding human PH20 hyaluronidase Treatment of retinoblastoma, 10/11/2024 Positive