I am curious to know the difference between an orphan designation and a marketing authorisation. Does an orphan designation imply that the product is currently registered as a drug?
Hi,
It’s a tricky one! There’s three concepts that are relevant in this context
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Marketing authorisation - The approval to market a medicine. This can be in one, several or all European Union Member States depending on the kind of marketing authorisation that you requested.
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Orphan Designation - Before obtaining marketing authorisation, one may apply for an Orphan Designation at EMA. This will give several advantages during the rest of the development of the drug - but it is not authorised yet!
- Orphan Medicinal Product - If you got an Orphan Designation (and if you comply to some other rules) the drug will be an Orphan Medicinal Product once authorised and also benefit from the orphan market exclusivity which is currently 10 years + possible 2-year extension if you complete the Pediatric Investigation Plan.
So to answer your question: No, an orphan designation does not mean that a product is registered (=has marketing authorisation).
It can get tricky if the following occurs. Multiple people/companies can apply for an orphan designation for the same active substance and the same orphan indication at the same time. In that case, only one of them can eventually get marketing authorisation as an Orphan Medicinal Product (unless the next one is clinically superior).
If that is the case, there can be both an active Orphan Designation and an Orphan Medicinal Prodoct containing the same active pharmaceutical ingredient.
Please see the EMA website for exact rules and definitions
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