On 22 March 2018, the European General Court handed down its judgment in Case T-80/16 Shire Pharmaceuticals Ireland v EMA.  Shire had sought annulment of the EMA’s decision refusing to validate its 2015 application for designation of its medicinal product Indursulfase-IT as an orphan medicinal product for the treatment of Hunter Syndrome. This decision is particularly important for innovator companies who invest in R&D for rare diseases, and will likely have an impact on incentivizing this type of research.

Background

In 2001, a Shire product containing the active substance idursulfase was designated as an orphan medicinal product for the treatment of Hunter Syndrome. This was followed in 2007 by the grant of a marketing authorisation for the medicinal product Elaprase, containing the active substance idursulfase, and administered as a solution for intravenous infusion. In parallel, Shire started developing another medicinal product containing the same active substance. However this product, Idursulfase-IT, could be delivered intrathecally, whereas Elaprase did not cross the blood-brain barrier. Intrathecal administration of the new product allowed treatment of cognitive disorders associated with a severe form of Hunter Syndrome.Continue Reading Shire v EMA: European General Court provides clarification for designation of orphan medicinal products