Gene therapy Archives

What could you buy with €1,1M euros? A house? Or a single prescription for a new drug... The first gene therapy drug "Glybera" is set to go on sale in Germany with a €1.1 million price tag, a new record for a medicine to treat a rare disease. And this drug from UniQure is not the only one in the pipelines since other major Biotech/Pharma companies are working on. 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. And yes, it is easily the most expensive medicine ever developed. However, if you have the rare disease which this newly developed gene therapy treats, you might be willing to sell everything you have to get your hands on it. Maybe that price offsets the immense cost required to make this drug, but nobody really knows, though, and that has every other pharmaceutical company a bit confused and very irate. According to Reuters, the new drug fights an ultra-rare genetic disease called lipoprotein lipase deficiency (LPLD) which results in the body not producing an enzyme and, as a result, the body cannot break down fat digested from food. It consists of a harmless modified virus that carries a corrective gene into the body’s cells. With only 150 to 200 patients likely to be eligible for Glybera across Europe, the impact on health-care budgets will be small, even at a very high price, but this case will be watched closely as a benchmark for future gene therapies. UniQure also has plans to seek approval for Glybera in the United States, which it hopes to get in 2018. Rivals in the gene therapy market include privately owned Spark Therapeutics, which has an eye drug in late-stage clinical tests, and Bluebird Bio, which is working on drugs for neurological and blood disorders. Bluebird Bio and UniQure both staged successful floats on the Nasdaq market in the past 18 months, reflecting growing investor interest in the field. Also, Among major pharmaceutical companies, Bayer struck a gene therapy deal with Dimension Therapeutics in June, while Novartis recently established a new cell and gene therapies unit. This new drug will reopen the debate concerning the growing price of therapeutics. Last January, the $84,000 Gilead Hepatitis C drug sets off payer revolt who didn't understand this price. When it comes to orphan drugs like LPLD the price is even higher. We'll see how patient and payers will welcome this treatment but one thing is sure, the current European Healthcare system can't sustain those costs indefinitely.