The European Medicines Agency's draft guideline on investigational advanced therapy medicinal products makes a clear distinction between exploratory and confirmatory clinical trials as the classical approach of distinguishing between various stages of a clinical trial is not as well defined for such products.

In an exclusive interview, Alexion R&D head John Orloff takes In Vivo on a tour around the company’s rejuvenated pipeline and explains how the business is innovating to pre-empt the Soliris-shaped hole in its balance sheet.

Europe’s patchwork of reimbursement agencies make it a daunting place for a small US biotech to do business. Traditionally large pharma partners or CMSOs have provided an experienced hand to hold, but as a new wave of gene therapies and orphan drugs get the EMA green light, smaller drug developers are applying innovation to commercialization and choosing to go solo.