Steep price for first gene therapy
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Steep price for first gene therapy

28.11.2014 - The first gene therapy for a rare genetic condition — UniQure's Glybera — is on its way to the European market. But there is a hefty price tag: Chiesi, responsible for the marketing of the orphan drug, is asking for a record price of €1.1m per patient.

Only a few hundred patients suffer from lipoprotein lipase (LPL) deficiency in Europe. The metabolic disorder leads to the patient's inability to process fatty acids in the blood, which can lead to pancreatitis. There was no effective treatment for the rare disorder, until Glybera was granted marketing authorisation in the EU two years ago— the first gene therapy to be approved in Europe. The drug, developed by Dutch biotech firm UniQure with the help of their insect cell-based manufacturing process, aims to restore the LPL enzyme activity. However, the market launch of the drug was delayed until longer-term data was available. Italian commercialisation partner Chiesi has now priced the drug at €53.000 per vial, bringing the total cost of one patient's treatment to over €1m.

Currently, the German Federal Joint Committee (G-BA), the highest decision-making body of the joint self-government of physicians, hospitals and health insurance funds in Germany, is assessing the benefits of Glybera. A decision is expected by April 2015 at the latest. The high price asked for the orphan drug will likely be subject to some debate, especially as it will serve as a benchmark for future gene therapies. In a next step, UniQure plans to seek approval for Glybera in the US.


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