First gene therapy approved by EU Commission

uniQure's Glybera has become the first gene therapy in the western world to be approved by regulatory authorities.

The European Commission gave the nod for the treatment in an announcement from the firm released today (November 2nd).

Glybera, also known as alipogene tiparvovec, is a therapy for people suffering with a metabolic disorder called lipoprotein lipase deficiency (LPLD) which is also named familial hyperchylomicronemia and causes acute and recurring pancreatitis.

Patients inflicted with the very rare and inherited disease, LPLD, cannot metabolise the fat particles carried in their blood.

This leads to inflammation of the pancreas (pancreatitis) which is an enormously painful, serious and possibly deadly condition.
LPLD can also cause early onset diabetes and cardiovascular complications in many patients.

Presently the only way sufferers can try to keep the illness at bay is by restricting the amount of fat they eat, so the approval of Glybera will make a dramatic difference to the treatment of the illness by helping to normalise the metabolism of fat.

It does this by preventing inflammation of the pancreas, thus avoiding the pain and suffering as well as associated co-morbidities if taken early enough, explained professor John Kastelein of the Department of Vascular Medicine at the Academic Medical Centre of the University of Amsterdam in the Netherlands.

The approval requires that patients will obtain treatment with Glybera through devoted centres of excellence and by specifically trained doctors.

uniQure has stated that it will also build a patient registry to additionally progress the understanding of this "devastating" and "under-researched" illness and the effects of Glybera therapy.

"The final approval of Glybera from the EC 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 Jorn Aldag, chief executive officer at the firm.

uniQure is currently arranging to apply for regulatory approval in the US, Canada and other markets.

Posted by Neil Clark