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EU Ruling Raises New Questions For Orphan Designation

 
• By Ian Schofield

An EU court has upheld a decision to revoke the orphan designation awarded to GMP-Orphan's trientine product for Wilson's disease, saying that the product no longer met the “significant benefit” criterion on the grounds of lack of availability of another drug. The case has raised questions about the level of evidence needed to justify a significant benefit. 

Benefit
The concept of 'significant benefit' for orphan designation is again under scrutiny • Source: Shutterstock

“Significant benefit” is a key criterion to be taken into account when deciding whether a drug for a rare disease should be awarded orphan designation in the EU.

It is a controversial one too, as has just been shown again after a European court ruled that GMP-Orphan’s trientine tetrahydrochloride product, Cuprior, should lose its orphan status because it did not offer a

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