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Posted by on Nov 6, 2012 in Science |

Europe approves the first drug to fix defective genes

Europe approves the first drug to fix defective genes

This is an idea that has on the table over three decades. Treat genetic diseases by replacement of a defective gene by a well working copy . A difficult road towards creating an effective therapy, safe and effective that it is now a reality thanks to a Dutch biotechnology company under the name uniQure. The first therapy in the West led to the correction of errors in the of a person has been approved in Europe.

A story that could open the way to similar pathways. The drug is called Glybera and is limited to the treatment of a rare genetic disorder only under the name of lipoprotein lipase deficiency (LPLD), a rare inherited disease that makes it impossible to metabolize fatty acids found in the blood , which is inflammation of the pancreas. A disorder that affects about one in every million people.

The problem with this is that first pioneering treatment is very expensive. Every therapy will cost 1.2 million euros per patient, which is obviously a problem. Despite this, the company justifies the price of the product, clearly exorbitant. According Joern Aldag, CEO of the company, will take similar treatments to market, and the high price is justified because it is a therapy to restore the natural function of the body and not a short-term solution:

This medication provides a greater benefit to patients than any classical protein substitution, and that is why we think we should be fairly and adequately compensated.

The question then becomes clear, how can patients access to treatment? uniQure speaks of an agreement with the European governments to lower prices which include potential variations in payment plans subsidized by the governments themselves. Thus, the European Commission has approved the drug and will go on sale next summer.

Glybera operates by introducing a gene in normal healthy patient’s body so that it can make LPL protein function.

After Europe, it is expected that the treatment will also adopt in the U.S. and Canada. A first pathway to repair the genetic code which might sumársele more in the future, similar therapies that might be able to cure other genetic disorders.

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