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

November 26, 2014

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 cells. After injection into the muscles, it corrects LPLD by:

 “ . . . enabling the muscle cells to produce the enzyme. The enzyme produced by these cells can then help to break down fats in the blood, reducing the number of pancreatitis attacks and the severity of the disease.”

Per a Reuter’s online article, the “Western world’s first gene therapy” is ready for sale in Germany with a price of 1.1 million euro ( approximately $1.4 million US). It is a new high price for a medicine for a rare disease, as up until now, Alexion Pharmaceuticals’ Soliris (Eculizumab) was the most expensive at a price of approximately $0.5 million. Chiesi filed a pricing dossier with Germany’s G-BA (Federal Joint Committee), which will issue a benefit assessment by the end of April 2015. In Germany, the launch price is valid for the first year. A final price will be set after the G-BA gives its assessment in April 2015 and “negotiations are held with statutory health insurance funds.”

Glybera is expected to be available the first half of 2015.

Please Note: “DNA Repair” courtesy of Tom Ellenberger, Washington University School of Medicine in St. Louis. [Public domain] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

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