Fin24.com | First gene therapy drug sets price record

London - The Western world's first gene therapy drug is set to go on sale in Germany with a 1.1m ($1.4m) price tag, a new record for a medicine to treat a rare disease.

The sky-high cost of Glybera, from Dutch biotech firm UniQure and its unlisted Italian marketing partner Chiesi, shows how single curative therapies to fix faulty genes may upend the conventional pharmaceutical business model.

After a quarter century of experiments and several setbacks, gene therapy is finally throwing a life-line to patients by inserting corrective genes into malfunctioning cells - but paying for it poses a challenge.

The new drug fights an ultra-rare genetic disease called lipoprotein lipase deficiency (LPLD) that 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.

Now Chiesi has filed a pricing dossier with Germany's Federal Joint Committee, or G-BA, which will issue an assessment of the drug's benefits by the end of April 2015. The company is seeking a retail price of 53 000 per vial, or 43 870 ex-factory.

That equates to 1.11m for an typical LPLD patient, averaging 62.5 kg in clinical trials, who will need 42 injections from 21 vials. This price will be subject to a standard 7% discount under Germany's drug pricing system.

Under German rules, the launch price for a new drug is valid for the first 12 months.

A Chiesi spokeswoman confirmed the launch price, in response to inquiries from Reuters, prompted by information from health insurance sources. 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.

"First commercial treatments are expected in the first half 2015," she said.

UniQure, which will get a net royalty of between 23 and 30% on sales, said EU pricing was a matter for its Italian partner, although the Dutch firm does plan to discuss Glybera pricing during an investor meeting in New York on December 1.

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Fin24.com | First gene therapy drug sets price record