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
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