Glybera is the first drug authorized in Europe which is based on gene therapy. It is also known as the most expensive drug in the world – a therapy with the use of it is expected to cost about 1.2 million euros. The drug helps people suffering from familial lipoprotein lipase deficiency (LPLD) which a rare disease of lipid metabolism. In 2012 the European Commission authorized the drug and it will be available in 27 countries of the European Union in a few months (in the second half of 2013).
The familial lipoprotein lipase deficiency (LPLD) is also known under the name of hyperlipoproteinemia type I or a familial hyperchylomicronemia. LPLD is a rare and inherited disease that affects about 1-2 persons per million. Familial hyperchylomicronemia is caused by a defective gene encoding a lipoprotein lipase. Fats cannot be metabolized, thus they accumulate in the form of chylomicrons. Symptoms of the disease include: hepatomegaly, xanthomas, high level of triglycerides in the blood, jaundice and recurrent pancreatitis [1,2].
The disease is diagnosed by examining the level of cholesterol and triglycerides in the blood, performing genetic tests, and (sometimes) by examining the activity of lipoprotein lipase in the blood. Until now, the only treatment was to follow a diet (eating no more than 20 mg of fat per day). [2]
How can we help people suffering from LPLD? A hope is a new drug that will appear at the market soon. Glybera is a drug for a gene therapy which introduces a functional lipoprotein lipase (LPL) gene into the cells of the body. The vector which transfer genes is a virus AAV (called adeno-associated virus). It does not cause infection and cannot replicate in the cells. Instead, it is characterized by a high affinity to the muscle cells in which the lipoprotein lipase is produced [3,4,5].
The drug was tested on a group of 27 patients with severe family lipoprotein lipase deficiency. In each case the drug was well tolerated, lipid levels after meals decreased, and the number of recurrent pancreatitis was reduced (which is associated with fewer hospitalizations and fewer visits to the ICU) [5].
Glybera can be administered to adult patients with a diagnosis of familial lipoprotein lipase deficiency suffering from severe or repeated pancreatitis despite a fat-restricted diet. The diagnosis must be confirmed by a genetic test. Before and after subjecting the patients to the gene therapy, they should receive an immunosuppressant treatment. The drug is administered once, in the form of multiple injections in the thighs and lower leg muscles of both legs. Glybera works for a long time – the level of chylomicrons remained decreased not only for 24, but also 52 weeks after administration. [4,5]
Glybera is the most expensive drug in the world thus far. The cost of the treatment per patient is over 1.2 million euro. Discussions with individual countries about the price and payment are ongoing (it is proposed to spread the payment into instalments for five years -250 thousand euro annually). In 2012 the drug was approved by the European Medicines Agency (EMA). Works on the admission of Glybera for sale in the United States, Canada and other countries are ongoing. The drug will be available in Europe in a few months, in the second half of 2013. [6]
Written by: Magdalena Mroczek
Source:
1.Dugdale, III, David C.; Zieve, David, eds. (May 29, 2011), “Familial lipoprotein lipase deficiency”, A.D.A.M. Medical Encyclopedia (National Center for Biotechnology Information), retrieved October 15, 2012
2.https://www.rareconnect.org/en/community/lipoprotein-lipase-deficiency
3.http://www.echodnia.eu/apps/pbcs.dll/article?AID=/20121107/STYLZYCIA01/121109270
4.http://www.ema.europa.eu/docs/pl_PL/document_library/EPAR__Product_Information/human/002145/WC500135472.pdf
5.http://www.uniqure.com/products/glybera/
6.http://www.oil.org.pl/xml/nil/gazeta/numery/n2013/n201301/n20130114
Would You like to know more? Watch one MEDtube.net: Acute Pancreatitis
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Glybera – a drug that will replace a defective gene
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