Lee Rannals for redOrbit.com — Your Universe Online
A drug that has the ability to correct errors in a genetic code has been approved for the first time ever in Europe.
The European Commission has given Glybera authorization to sell the drug next summer for treating an ultra rare genetic disease.
The small Dutch biotech company’s drug will cost around $1.6 million per patient, a new record price for medicine.
The drug is a gene therapy for a rare disease that leaves people unable to properly digest fats, which affects one in a million people. As fat builds up in the blood, it leads to abdominal pain and life-threatening inflammation of the pancreas. The only way to manage the condition is with a very low-fat diet.
UniQure‘s drug uses a virus to infect muscle cells with a working copy of the gene that breaks down fats.
Treating patients by replacing a defective gene with a working copy first came to light in 1990, when the world’s first gene therapy clinical tests against a rare condition called severe combined immunodeficiency (SCID) took place.
People who suffer from SCID are unable to cope with infections and usually die in childhood. The researchers trying to use gene therapy against the condition faced a setback in 1999 when an Arizona teenager died in a gene therapy experiment. A few years later, two French boys with SCID developed leukemia after being treated.
The Chinese firm SiBono GeneTech won approval for a gene therapy drug for head and neck cancer in 2003, but no products have been approved in the U.S. or Europe.
UniQure is preparing to apply for regulatory approval for Glybera in the U.S., Canada and other markets.
“The final approval of Glybera from the European Commission marks a major step forward in making gene therapies available not only for lipoprotein lipase deficiency, but also for a large number of rare diseases with a very high unmet medical need.” UniQure chief executive officer Jorn Aldag told BBC News.