Rare Disease Gene Therapy Pricing: uniQure’s Glybera Sets A Record High

Orphan Druganaut Blog

No gene therapy has ever been approved for sale in the United States. In Europe, the EMA (European Medicines Agency) gives regulatory approval in October 2012 to Dutch biotech uniQure, to sell its gene therapy, Glybera, for the treatment of the rare disease, Lipoprotein Lipase Deficiency (LPLD). Glybera has Orphan Drug Designation (ODD) in both the US (May 2007) and the EU (March 2004). Glybera is from uniQure and its unlisted Italian marketing partner Chiesi.

Patients with LPLD have a defect in the gene for lipoprotein lipase. Lipoprotein lipase is an enzyme that breaks down fats. Those with LPLD are usually on a strict low-fat diet and are prone to attacks of pancreatitis, which can be life-threatening.

Glybera is given as a single treatment involving multiple injections into muscles of both the upper and lower legs. Glybera contains a modified virus that carries the lipoprotein lipase gene into the patient’s body’s…

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