Glybera, which was the first gene therapy drug put on sale, has been unable to make the expected development in the pharmaceutical industry because of its price above $ 1 million. It is declared that the drug will no longer be commercially available at the end of 2017.
Glybera which was produced as a result of the long-term studies of a Dutch company named UniQure had received the approval for commercial sale by the European Medicines Agency. The drug, which is able to treat the genetic disorder called lipoprotein lipase deficiency (LPLD) by using a single dose, settled first on the list of the world’s most expensive drugs with its $ 1 million price for just one dose. The drug developer UniQure has declared that the medicine will not be on sale next year.
The company explained that the reason of this decision is not related with the risk-benefit concerns. As it can be estimated, there has not been enough demand to keep the drug on sale despite the high costs of its production process. The insurance companies, healthcare providers and government agencies do not provide financial support for the purchase of this drug. And, due to rare occurrence of LPLD, insurance companies, healthcare providers and government agencies have not made any attempt to change their regulations. All these reasons caused the demand to remain well below expectations. The drug was used by just one person in recent five years. UniQure announced that they would not renew the approval by the European Medicines Agency.
There are many other studies going on about the treatment of genetic disorders such as Duchenne muscular dystrophy and lipoprotein lipase deficiency. Gene therapy, which works with the principle of replacing damaged genes in cells with healthy genes using viruses, was currently tested.