Glybera, the first gene therapy to go on sale in Europe, is set to cost €1.1m (£870,000) per patient, making it the world's most expensive drug.
The drug, produced by the Dutch biotech firm UniQure, is used to treat lipoprotein lipase deficiency (LPLD), a very rare genetic condition where the body cannot digest fat properly.
At a retail price of €53,000 (£42,000) per vial, a typical LPLD patient with an average weight of 62.5kg would require 21 vials of Glybera for a full course of treatment. This equates to a record €1.1m price tag per patient.
Glybera would initially be sold in Germany 'with the first commercial treatments expected in the first half of 2015', a spokesperson for Chiesi - the Italian company marketing the drug - told Reuters. The drug is also expected to be available in the US market in 2018.
LPLD results in the blood being clogged with fat. This precipitates repeated life-threatening episodes of pancreatic inflammation, early onset diabetes and cardiovascular complications. The disease affects one to two people per million worldwide, who have to carefully manage their condition and adhere to a strict low-fat diet.
Glybera is the first therapeutic cure available for LPLD patients, and acts by replacing defective copies of the gene essential for breaking down fat in the body. Commercial anti-cancer gene therapy is currently available in China but Glybera is the first gene therapy in the western world. It is expected that 150 to 200 LPLD patients will be eligible for the drug in Europe.
Despite its high cost, Chiesi argues Glybera offers a cost-effective treatment for LPLD patients since it permanently cures their disease.
Analysts suggest Glybera's price tag could possibly set a benchmark for other commercial gene therapies. Although it is also possible that future gene therapies targeting more common diseases may be less expensive since manufacturers could recover their research and development costs more efficiently from a larger number of patients.
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