This Dutch Gene Therapy is on the shelf and seems to Work. But nobody is buying…

06/05/2016 - 3 minutes

uniQure‘s Glybera was approved in Europe in 2012, making it the first gene therapy on the market in the West. But so far this €1M therapy has only been bought once… 

mit_technology_review_uniqure_glyberaThe approval of Glybera was an important milestone for Biotech. After years of being deemed too risky (after the death of Jesse Gelsinger in 1999), a gene therapy treatment finally made it to the European (and Western) market.

Glybera also holds another record. At €1M, it’s the most expensive drug ever. However, according to the MIT Technology Review the drug is a flop. Since its approval, only one patient has received it.

The therapy was developed by Amsterdam-based uniQure, and is a treatment for lipoprotein lipase deficiency (LPLD) – a genetic defect that affects only 1 out of 1 million people. It interferes with fat digestion, and can lead to diabetes and life-threatening acute pancreatitis.

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