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.
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.
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.
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
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.
its high cost, Chiesi argues Glybera offers a cost-effective
treatment for LPLD patients since it permanently cures their disease.
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.