EMA Orphan Approval Path Seems More Twisty After Pfizer/Protalix’s Elelyso Stumbles

The European Medicines Agency will not recommend Pfizer/Protalix’s Elelyso for type 1 Gaucher, maintaining that it is too similar to Shire’s Vpriv, despite the firms’ argument that their product offers better bone marrow data and alleviates shortage concerns.