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What Orphan Product Is Estimated To Cost 1 Million Dollars When Launched ?

January 13, 2013

Clarification Posted 02/12/13 : Per the Reuters article referenced below, the cost of Glybera is “$1 million per patient” and not “$1 million/patient/year”.
Per a July 2012 article from, Jorn Aldag, UniQure’s Chief Executive, said the drug probably will cost €250,000 a year for 5 years, with a total bill of €1.25 million per patient.

UniQure’s lead orphan product Glybera (Adeno-associated vector expressing the human lipoprotein lipase protein), for the treatment of Lipoprotein Lipase Deficiency (LPLD), is estimated to cost close to $1 million/patient when it launches in the Summer of 2013 in Europe, according to Reuters. UniQure is a European company based in The Netherlands. Glybera is the 1st gene therapy to receive approval in the West.

Up to this point in time, Alexion Pharmaceuticals’ orphan drug Soliris, which costs about $410,000/patient/year, has been the most expensive orphan drug in the world.  Per the Reuters article, over the past 10 years, the orphan drug market has been growing at an estimated 25.8% pace, compared to 20.1% for the rest of the drug market.

The only reason the drug costs so much is because UniQure can charge as much as it wants without penalty, as there are no current government-approved alternatives. This is big business for biopharmaceutical companies when it bills insurance companies enormous large amounts of money for very specialized drug treatments that are not available anywhere else, and are also the only available option patients may have.

Facts about Glybera

–  Receives FDA Orphan Drug Designation (ODD) May 21, 2007

–  Receives European Commission (EC) orphan designation on March 8, 2004

–  EC approves Glybera’s marketing authorization on October 25, 2012, for LPLD under exceptional circumstances in all 27 EU member states

–  1st in a class of gene therapy products to receive approval in the Western world to treat orphan diseases

–  As part of the approval, it will be offered through “dedicated centers of excellence with expertise in treating LPLD & by specially trained doctors to ensure ongoing safety of this novel treatment paradigm”

–  Company getting ready to apply for regulatory approval in the US, Canada, and other markets.

Facts about LPLD

–  Estimated # is approximately 0.02/10,000 EU population; Approximately 1,013 patients.

References January 3, 2013 article titled, “Million-dollar therapy will test payers’ tolerance of orphan drug prices” November 2, 2012 article titled, “European Commission stamps approval on 1st gene therapy for rare disease”

Molecular Therapy, Editorial, October 2012, “Endgame: Glybera Finally Recommended for Approval as the 1st Gene Therapy Drug in the European Union”

Motley Fool article titled, “Pfizer adds to Orphan Drug Momentum” January 3, 2013 article titled, “Analysis: Entering the age of the $1 million medicine”

UniQure Pipeline

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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