Germany is going to be the first country in Europe where gene therapy drug will be marketed. Sales of the drug for the treatment of a rare genetic disease — lipoprotein lipase deficiency, will be launched in the first half of next year. The cost of the drug will be 1.1 million euros for the course.

The drug is called Glybera and was developed by Danish company UniQure and received a marketing authorization of the European Medicines Agency (EMA) in 2012. pharmaceutical market experts point out that currently there are 150-200 patients with lipoprotein lipase deficiency in EU, so the purchase of the drug will not have much impact on the budget of the national health systems, despite the high price of drugs.

It is expected that in the US a new therapy to be approved in 2018. Currently the drug is being sold only in China.

Lipoproteinaza enzyme promotes the breakdown of fat entering the body with food. Due to a mutation in the gene encoding the enzyme LPL production it is not produced in the desired amount. Such patients are forced to adhere to a very strict diet with a severe restriction of the amount of fat. Glybera is a gene therapy drug based on adeno-associated virus (AAV), which is carries healthy LPL gene.