AMT reaches another milestone in the official marketing authorisation process for Glybera

Jan 25 2010

Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, has reached another important milestone in the official marketing authorisation process for its lead product Glybera(R), AMT's proprietary product for lipoprotein lipase deficiency (LPLD). The submission of the Glybera(R) Marketing Authorisation Application (MAA), announced earlier, has cleared the validation stage with The European Medicines Agency (EMA, formerly known as EMEA). The EMA will now commence its formal review of Glybera(R).

AMT has concluded two clinical studies for LPLD, in Europe and Canada, and long term follow-up from both of these is ongoing, as is a third clinical study in Canada. In these three studies Glybera(R) has shown a sizeable decrease in the incidence of pancreatitis, or acute inflammation of the pancreas, the most debilitating complication of LPLD. In addition, these studies indicate that Glybera(R) has an excellent safety profile.

"The acceptance of the Glybera(R) dossier by EMA is a significant step towards marketing approval for Glybera(R). Moreover, it demonstrates AMT's development capability. A future approval of the MAA for Glybera would fully validate our gene therapy approach and our adeno-associated viral (AAV) vector delivery platform. We believe this step offers hope to many patients, as gene therapy may become the therapeutic approach of choice for inherited disorders" said Jorn Aldag, Chief Executive Officer of AMT.

The EMA formal review will be conducted via the centralised procedure, which is the standard route for all advanced therapies. During 2010, AMT expects to provide further updates on the results from its follow-up and ongoing studies, in accordance with reporting regulations.