Gene therapy nears approval as European regulator makes recommendation

The European Medicines Agency (EMA) has recommended European Union market approval for a gene therapy, Glybera, to treat patients with severe cases of a rare genetic condition called lipoprotein lipase deficiency (LPLD). If the treatment is approved by the European Commission, which usually follows the recommendations of the EMA, Glybera will be the first commercially available gene therapy in Europe and the USA.

'This is a watershed moment. Gene therapy holds incredible promise for people, especially those with rare disease. It paves the way for the approval of other treatments of this kind', said Dr Tim Coté, former director of the US Food and Drug Administration's office of orphan products development.

Due to mutations in the LPL gene, patients with LPLD lack the necessary enzyme to break down fat from digested food, which can cause fat globules to build up in the blood resulting in severe abdominal pain and pancreatitis. The only way to manage the disease at present is for patients to control the fat content in their diet. Glybera works by using a virus vector to introduce a working copy of the gene.

Jörn Aldag, chief executive at UniQure, the company that is developing the drug, said: 'Now, for the first time, a treatment exists for these patients that not only reduces this risk, but also has a multi-year beneficial effect after just one treatment'.

The approval of Glybera has not been without problems. LPLD is very rare affecting one or two people per million, so it is difficult to assess the risks and safety of the drug in the usual way. Glybera originally received a negative response from the EMA, and the recent recommendation is only for use in patients with a severe form of the disease.

Gene therapy is aimed at patients with genetic diseases and essentially replaces their faulty genes with working ones. The only other commercially available gene therapy is Gendicine, which was approved for the Chinese market in 2004 to treat cancer.

Gene therapy has received bad press in the past after the trial of a drug to treat children with severe combined immunodeficiency was suspended due to some participants developing leukaemia. Another setback to the development of gene therapies was the death of a participant after he reacted adversely to the injected virus in a gene therapy trial.