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

By Jenny Hope

PUBLISHED: 08:19 EST, 2 November 2012 | UPDATED: 21:14 EST, 2 November 2012

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 persons 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.

More:
First gene therapy drug given European license

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