Court of Justice upholds annulment of EMA’s refusal of orphan designation because same ‘active substance’ as an existing orphan drug (EMA v Shire Pharmaceuticals Ireland Ltd, Case C-359/18 P)

Table of contents

• What are the practical implications of this case?
• What was the background?
• What did the court decide?
• Case details

Article summary

Life Sciences analysis: The Court of Justice upheld on appeal the General Court’s decision to annul the EMA’s refusal to validate orphan designation on the basis that Shire’s new product contains the same active substance as an existing orphan product for the same rare disease. The decision is a shift from the position of the European Commission and EMA that companies are entitled only to one ten-year period of orphan designated market exclusivity for each active substance and for each target indication. The judgment clarifies the allocation of decision powers between the EMA and COMP in deciding orphan designations under Regulation No 141/2000 and which legal elements to consider, elucidating the important legal concepts of ‘medicinal product’ versus ‘active substance’ and ‘significant benefit.’