Drug to fix faulty DNA shown green flag in Europe
London: Regulators have approved the first therapy in the western world that can correct errors in a person’s genetic code, it has been revealed.
Europe has approved Glybera to be used against a rare inherited disorder which disrupts fat production in the body, the Daily Mail reported.
The treatment uses a virus to counteract LPLD, lipoprotein lipase deficiency, which can lead to acute inflammation of the pancreas.
The treatment was backed by an advisory panel to the European Commission in July and full approval was granted this week, and the medicine is expected to be available next year.
Professor John Kastelein, of the University of Amsterdam, said that 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.
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.
Glybera treats the genetic disorder lipoprotein lipase deficiency (LPLD) by altering sufferers’ DNA.