Drug to fix faulty DNA gets go-ahead in landmark move that may alter medicine forever



Gene therapy drug given green light for rare disease

Glybera to cost £1m ($1.6m) for each patient



Drug is first to get approval in Western world

EU go-ahead paves way for many more treatments



Revolution: Gene therapies can change a person's DNA

Regulators yesterday approved the first therapy in the western world that can correct errors in a person’s genetic code.

Europe has approved Glybera to be used against a rare inherited disorder which disrupts fat production in the body.

The treatment uses a virus to counteract LPLD, lipoprotein lipase deficiency, which can led to acute inflammation of the pancreas.

The illness affects around one or two people per million and sufferers have damaged copies of a gene which is essential for breaking down fats. The virus infects muscle cells with a copy of a healthy gene and a one-off treatment is effective.

The treatment was backed by an advisory panel to the European Commission in July and full approval was granted yesterday. The medicine should be available next year.

Professor John Kastelein, of the University of Amsterdam, said the therapy would have a ‘dramatic impact’ on patients.

Only two other gene therapies have previously been approved for sale, both in China.

It will cost around £1 million ($1.6 million) per patient, a new record for pricey modern medicines.

Joern Aldag, chief executive of Amsterdam-based uniQure, said more such treatments would follow and argued a high price was justified because gene therapy restored natural body function and was not just a short-term fix.

'This provides higher benefit to patients than the classical protein replacement strategy and this is why we think we should be fairly and adequately compensated,' he said in a telephone interview on Friday

Patients with LPLD, which affects no more than one or two people per million, are unable to handle fat particles in their blood and are at risk of acute and potentially fatal inflammation of the pancreas.

The approval follows a positive recommendation from the European Medicines Agency in July.

WHAT DOES GLYBERA DO?

Glybera treats the genetic disorder lipoprotein lipase deficiency (LPLD) by altering sufferers' DNA.

LPLD affects about one in a million people, leaving them without a vital gene that breaks down fat particles in their blood. It means fat builds up in the blood leading to abdominal pain and life-threatening inflammation of the pancreas (pancreatitis). The only other way to manage the disease is by having a very low-fat diet. The therapy uses a virus (pictured) to infect muscle cells with a working copy of the gene.



The privately owned firm is now working with governments on potential pricing strategies, which are likely to vary from country to country, ahead of the commercial roll-out from the second half of 2013.

Aldag said some countries preferred the idea of a one-off payment at the time of treatment but others were interested in an annuity system, which would probably involve charging around £200,000 a year for five years.

That kind of annual charge would put Glybera in a similar price range to expensive enzyme replacement therapies for other rare diseases, such as Cerezyme for Gaucher disease from Sanofi's Genzyme unit.

UniQure is also preparing to apply for regulatory approval for Glybera in the United States, Canada and other markets.

The idea of treating disease by replacing a defective gene with a working copy gained credence in 1990 with the success of the world's first gene therapy clinical tests against a rare condition called severe combined immunodeficiency (SCID).

People with SCID - also known as 'bubble boy disease' - cannot cope with infections and usually die in childhood.

The field then suffered a major setback when an Arizona teenager died in a gene therapy experiment in 1999 and two French boys with SCID developed leukaemia in 2002.

In China, Shenzhen SiBiono GeneTech won approval for a gene therapy drug for head and neck cancer in 2003 but no products have been approved until now in Europe or the United States.