5 November 2012

Dr Lucy Spain By

Appeared in BioNews 680

The European Medicines Agency (EMA) has approved Glybera, a gene therapy to treat the rare genetic condition, lipoprotein lipase deficiency (LPLD), for sale across Europe. Glybera is the first gene therapy to be approved in Europe or North America and offers the first therapeutic treatment for people with LPLD.

'The final approval of Glybera from the [European Commission] marks a major step forward in making gene therapies available not only for LPLD but also for a large number of rare diseases with a very high unmet medical need', said Mr Jörn Aldag, CEO of uniQure, which developed Glybera.

At a cost of €1.2million per patient, Glybera is the most expensive medicine currently available. However, Mr Aldag said this is a fair price to pay for a therapy that restores natural body function and is not just a short-term fix.

In an interview with Reuters he said, '[Glybera] provides higher benefit to patients than the classical protein replacement strategy and this is why we think we should be fairly and adequately compensated'.

After the first successful treatment of severe combined immunodeficiency (SCID) using gene therapy in 1990, development of the technique stumbled. One gene therapy patient in Arizona died and two study participants in France developed leukaemia during the SCID gene therapy trial. Although approval for a cancer gene therapy was granted in China in 2003, no European approval has been granted until now.

LPLD is a disease which results in an inability to digest fat properly, leading to repeated bouts of inflammation of the pancreas, early onset diabetes and cardiovascular complications. It affects up to one to two people per million worldwide. Protein replacement therapy provides a temporary fix for many genetic diseases, however it is not available for the treatment of LPLD.

Professor John Kastelein from the Department of Vascular Medicine at the University of Amsterdam, the Netherlands commented, '[Glybera] will have a dramatic impact on the lives of these patients. Currently their only recourse is to severely restrict the amount of fat they consume. By helping to normalise the metabolism of fat, Glybera prevents inflammation of the pancreas thereby averting the associated pain and suffering and, if administered early enough, the associated co-morbidities'.

UniQure is currently planning to apply for regulatory approval for Glybera in other markets including the USA and Canada. Mr Aldag said, 'The final approval of Glybera from the European Commission marks a major step forward in making gene therapies available not only for lipoprotein lipase deficiency, but also for a large number of rare diseases with a very high unmet medical need'.

Clinical trials of gene therapy in Parkinson's disease (reported in BioNews 652) and cystic fibrosis (reported in BioNews 649) are already underway in the UK.