Module 1 2021

30/06/2021

Examples

Nalbuphine hydrochloride for the condition uraemic pruritus* • ODD was refused by COMP and adopted by the EC in 2015:

sponsor failed to establish that uraemic pruritus is a distinct, recognisable medical entity with signs and symptoms that can be distinguished from pruritus caused by other conditions. (EMA/COMP/504453/2015, Sep 2015)

Nieman Pick disease**

Original designation ‘Niemann-Pick disease, type B’ (2001), changed to ‘Niemann-Pick disease’ (2016)

*Itching associated with kidney disease ** Rare, inherited disease that affects the body's ability to metabolize fat (cholesterol and lipids) within cells

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Orphan designation

Recurrent questions • Definition of orphan condition/indication • Nature and quality of data supporting medicinal plausibility • Methodology and sources for prevalence calculations

Not guaranteed • Annual reports • Monitor competitors • Assessment of orphan status following CHMP positive opinion ánd prior to EC-decision (justification in CTD M1.7)  Market exclusivity only for sponsor who first obtains MAA  Co-exclusivity  Similar active ingredient

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