Stockwatch: When rare is too expensive
This article was originally published in Scrip
Executive Summary
When the European Medicines Agency (EMA) declined to approve Amsterdam Molecular Therapeutics’ (AMT’s) Glybera (alipogene tiparvovec) for the rare genetic disease lipoprotein lipase deficiency (LPDL) back in June (scripintelligence.com, 24 June 2011), the CEO of AMT was ‘convinced and confident’ of the approval of Europe’s first gene therapy within five months. At the end of this week, AMT completed the first, and easiest part of that process by filing an appeal against the EMA’s rejection and stated that they expected that the re-examination of the dossier would be completed by the end of 2011 (scripintelligence.com, 8 July 2011). Behind the backdrop of the appeal for re-examination which, history has shown does not have a high chance of success, the AMT annual report for 2010 reveals that the regulatory path for Glybera has been far from smooth.