Europe’s first gene therapy drug receives regulator recommendation

The first gene therapy treatment in the Western world has moved closer to being prescribed to patients, as the European drugs regulator recommended the drug for approval across member states.

The therapy is called Glybera and will be used to treat patients whose bodies fail to make certain enzymes, meaning fat does not break down and the pancreas expands. The illness affects one or two people in every million.

The European Medicines Agency have failed to recommend the drug for approval three times before assessing the treatment in a particular groups of patients suffering with pancreatitis.

It was made particularly difficult for the EMA to make their decision as only 27 people were available for trials, such is the rarity of the condition. However they have now accepted that the drug could be used for the worst-affected patients on condition that they are closely monitored following the one-off treatment.

The decision now passes to the European Commission for official endorsement, which it is likely to give.

Glybera will be the first gene therapy – outside of those used in clinical trials – in Europe or the US. China approved a gene therapy for head and neck cancer in 2004.

Gene therapy works by replacing faulty or unhealthy genes with healthy copies, and earlier trials were often unsuccessful because not enough genes were replaced, or the wrong genes were “switched on”.

Glybera works by using a virus to smuggle healthy genes into a patient, which when in place will help create the missing enzyme.

A former director of the US Food and Drug Administration praised the decision, saying: “This is not an isolated example; this is the beginning of something. It paves the way for the approval of other treatments of this kind. There are about 3,000 of these rare genetic diseases out there… gene therapy is the way we’re going to cure those 3,000 diseases.”

Abbie Cavendish