Gene therapy in Europe: the first swallow
The first gene therapy drug in the West will appear in 2015
Copper news
Soon, a gene therapy drug will appear for the first time in Western countries, which will be sold in Germany. The cost of one full course of treatment will be about 1.4 million dollars (the price of one ampoule of the drug is 42 thousand dollars), reports The Guardian (Germany poised to say yes to €1.1m a patient gene therapy drug). The drug is intended for the treatment of a rare genetic disease – lipoprotein lipase deficiency.
The enzyme lipoprotein lipase is involved in regulating the level of lipids in the blood, but in people with a mutant gene that should be responsible for the formation of this enzyme, there is a lack of it, which is why there is a high risk of blockage of blood vessels with lipids (fats). The drug Glybera, developed jointly by the Dutch biotech company UniQure and the Italian marketing firm Chiesi, makes up for its deficiency.
The total number of potential patients in Europe is approximately 150-200 people, so the drug will not significantly affect the health budget. Nevertheless, Glybera is the first gene therapy drug in Western countries. It has already received approval in 2012, but final approval for its sale is expected by April next year.
The world's first gene therapy scheme was approved for commercial use in 2003 in China, where an adenoviral vector was developed that delivers the p53 protein gene, one of the most important inhibitors of the formation of malignant tumors, to tumor cells.
According to proponents of gene therapy for lipoprotein lipase deficiency, the drug is effective because it is able to completely normalize the health of patients. According to the developers of the drug, the course of Glybera treatment will cost no more than several courses of expensive enzyme replacement therapy, which must be performed periodically.
Portal "Eternal youth" http://vechnayamolodost.ru01.12.2014