Decision made due to lack of demand
Dutch biotech UniQure made headlines in 2012 when it won approval for its Glybera gene therapy, which at more than €1m is the world's most expensive drug. Now just five years later, it is pulling it off the market.
UniQure said there was simply not enough demand for Glybera (alipogene tiparvovec) - a treatment for patients with the ultra-rare inherited disease lipoprotein lipase deficiency (LPLD) - to warrant keeping it on sale. As a result, it will not try to get its marketing licence renewed when it expires on October 25 this year. Glybera is marketed by Chiesi in Europe.
The decision "involved a thoughtful and careful evaluation of patient needs and the clinical use of the therapy, and is not related to any risk-benefit concern", said UniQure's chief executive Matthew Kapusta in a statement. "Glybera's usage has been extremely limited and we do not envision patient demand increasing materially in the years ahead."
Only one patient has been treated with Glybera commercially since its approval, and the German physician who oversaw the procedure said it was tough getting approval from regulators and insurers as the price was simply too high. The one recipient has however seen a dramatic improvement in LPLD symptoms.
The company says it has already started discussions with the European Medicines Agency (EMA) about withdrawing Glybera and continuing to monitor patients who have received the therapy. In 2015 it dropped plans to try to get the therapy approved in the US
The experience with Glybera is a stark reminder of the challenges in developing an effective business model for gene therapy, particularly when it consists of a single treatment that should last indefinitely. It's readily accepted that such therapies should have a high price to recoup R&D expenses, but UniQure's experience shows there is clearly a limit and a new mechanism is needed to accommodate this new generation of one-off treatments.
GlaxoSmithKline (GSK), which was the second company to bring a gene therapy to the EU market when it launched Strimvelis (GSK2696273) for immune-deficiency disease ADA-SCID last year, set its price at $665,000, with a money-back guarantee if the therapy was unsuccessful.
That was deemed acceptable by the Italian government which agreed to reimbursements for the drug, since prior treatments could cost as much as $4m over a patients' life. As a result the 15 or so patients per year who are diagnosed with ADA-SCID will all be offered treatment at an Italian hospital.