FRANKFURT\/LONDON The Western worlds first gene-therapy drug is set to go on sale in Germany with a price of 1.1 million ($1.4 million), a new record for a medicine to treat a rare disease. <\/p>\n
The sky-high cost of Glybera, from the Dutch biotechnology firm UniQure and its unlisted Italian marketing partner, Chiesi, shows how targeted therapies to fix faulty genes may upend the conventional pharmaceutical business model. <\/p>\n
After a quarter of a century of experiments and several setbacks, gene therapy is finally throwing a lifeline to patients by inserting corrective genes into malfunctioning cells but paying for it poses a challenge. <\/p>\n
The new drug fights an ultra-rare genetic disease called lipoprotein lipase deficiency (LPLD), which clogs the blood with fat. The medicine was approved in Europe two years ago, but its launch was delayed to allow for the collection of six-year follow-up data on its benefits. <\/p>\n
Now Chiesi has filed a pricing dossier with Germanys Federal Joint Committee (G-BA), which will issue an assessment of the drugs benefits by the end of April 2015. The company is seeking a retail price of 53,000 (66,000) per vial, or 43,870 ($54,800) ex-factory. <\/p>\n
That equates to 1.11 million for a typical LPLD patient, who will need 42 injections from 21 vials. This price will be subject to a standard 7 percent discount under Germanys drug pricing system. <\/p>\n
Under German rules, the launch price for a new drug is valid for the first 12 months. <\/p>\n
A Chiesi spokeswoman confirmed the launch price. She added that a final figure would be set after the G-BA gives its verdict and negotiations are held with statutory health insurance funds. <\/p>\n
First commercial treatments are expected in the first half 2015, she said. <\/p>\n
UniQure, which will get a net royalty of between 23 and 30 percent on sales, said EU pricing is a matter for its Italian partner, although the Dutch firm does plan to discuss Glybera pricing during an investor meeting in New York on Dec. 1. <\/p>\n
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