Marketing authorisation application (MAA) - pre-submission interactions form

European Medicines Agency procedural advice for users of the centralised procedure for similar biological medicinal products applications

European Medicines Agency procedural advice for users of the centralised procedure for similar biological medicinal products applications: document with track changes

Orphan designation: Adeno-associated virus serotype 9 containing the human RPE65 gene inherited retinal dystrophy due to defects in the RPE65 gene, 16/01/2025 Positive

Orphan designation: N-(4-(4-amino-5-(3-fluoro-4-((4-methylpyrimidin-2-yl)oxy)phenyl)-7-methyl-7H-pyrrolo[2,3-d] pyrimidin-6-yl)phenyl)methacrylamide hydrochloride Treatment of biliary tract cancer, 10/11/2022 Positive

Orphan designation: Arsenic trioxide Treatment of acute promyelocytic leukaemia, 13/12/2024 Positive

Biosimilar medicines: marketing authorisation

Orphan designation: human anti-promyostatin monoclonal antibody (apitegromab) Treatment of spinal muscular atrophy, 14/12/2018 Positive

Orphan designation: Lomitapide Treatment of familial chylomicronaemia, 17/12/2010 Positive

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