Module232025

12/05/2025

New Section 2 core messages

Unmet need and potential benefit Formulation Administration Safer alternative

Differences and similarity vs adults

Pharmacology and mechanism of action First in class? Proof of concept?

Clinical course? Severity? Applicability of end-points

Markers?

Leveraging existing data in the most effective way – providing evidence foundation supporting extrapolation (as applicable)

13

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Quality and non clinical development

1 4

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