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The world's most expensive genetic drugs will be helpless to withdraw from the market!

  • Author:Jane
  • Source:科技日报
  • Release on :2017-04-27

Glybera is the first gene drug to be marketed in Western countries for the treatment of rare genetic disorders - lipoprotein lipase deficiency (LPLD). However, according to "MIT Technology Review" magazine website recently reported that after 25 years of research and development was difficult to come out of the drug, will be due to high prices and lack of demand and out of the market.

UniQure, a well-known Dutch biotechnology company that developed the drug, said last week that the EU's licensing period for the drug will expire in October and will not be renewed again.

The reason for this is that the market demand for the drug is very limited and there will be no significant increase in the number of patients who have the financial resources to use the drug in the next few years. Grilbella, which was approved by the European Drug Administration in 2012 and listed in 2014, is an important milestone in gene therapy that has undergone a series of frustration and safety challenges. But the price of the drug was beyond the people's imagination, the price of a course of treatment up to more than 100 million dollars, creating a new record of modern prescription drug prices, but also for its later "no market price" market performance hidden.

Lipoprotein Lipase deficiency, also known as familial high chylomicronemia, because the patient's blood can not bear any fat particles, a variety of acute pancreatitis recurrent, very painful and even life-threatening. Before the advent of Gribbella, there is no specific drug for the disease, only through diet to control blood lipid levels. And Gribera through an adeno-associated virus (AAV), will produce a functional lipoprotein lipase gene delivery to the patient skeletal muscle, the patient after treatment significantly reduced the incidence of pancreatitis, and can relax diet restrictions, improve life quality.

But because the drug price is too high, the health insurance agencies on the use of the drug reimbursement of the relevant set a serious obstacle, at present only one patient to accept the drug treatment.

Kathy Quinn, a specialist in the European drug pricing mechanism at the Massachusetts Institute of Technology's Center for Biomedical Innovation, says that the number of lipoprotein lipase deficiency patients is not much, and that one million people are sick, plus the public The validity of the question has been there, "a needle million" pricing lack sufficient argument. "The failure of Gribbella does not mean that other gene therapies are also troublesome, but that will lead to a deep reflection of the industry's pricing mechanism for gene drugs," Quinn said.