Weigh! The first gene therapy drug will be on the market with an average cure price of 1.11 million euros

Source: on November 28, 2014, the first gene therapy drug in the western world will be launched in Germany, with a price of 1.1 million euros (about US $1.4 million) setting a new record for the price of drugs for treating rare diseases The drug, glybera, is developed and manufactured by Dutch biotechnology company uniqure and its market partner is Cayce Pharmaceutical Group in Italy, Reuters reported The priceless glybera shows how a single treatment can repair defective genes, which may subvert the traditional medical business model After 25 years of experiments and several twists and turns, gene therapy finally throws out the lifeline to patients through the method of transferring correction genes to defective cells, but its high price is a huge challenge to patients In October 2012, glybera was approved in the European Union, but the listing date was delayed for two years, which was used to track and collect the follow-up data of patients for up to six years after treatment All of these patients met the labeling requirements of glybera in the European Union Now, Casey has submitted the pricing materials to the g-ba, and will release the evaluation of the drug by the end of April 2015 Drug manufacturers believe that the retail price of each bottle should be 53000 euros, and the factory price should be 4387 euros In the clinical trial, a typical lpld patient with an average weight of 62.5kg needs 21 bottles of glybera and 42 injections, so a patient needs to spend 1.11 million euros on the drug Under the German drug price system, the final price will enjoy a 7% discount According to the relevant regulations of Germany, the validity period of new drug price is the first 12 months A spokeswoman for Casey confirmed the listing price "The first commercial use is expected in the first half of 2015." She added that the final price of the drug would be determined after g-ba's conclusion and after negotiations with the statutory medical insurance fund Uniqure will receive a net royalty of 23% to 30% of sales The company claims that the EU's pricing is the business of its partner Casey, but uniqure plans to discuss glybera's pricing at an investor meeting in New York on December 1 As there are only 150-200 patients in Europe who may meet the requirements of using glybera, even if the price of the drug is very high, the impact on the health care budget will be very small, but the drug still gets close attention from all walks of life, because it is regarded as a benchmark for gene therapy in the future At present, uniqure is also seeking approval from glybera in the United States, and expresses the hope that glybera can successfully pass the customs in 2018 In 2004, China became the first country to approve gene therapy products for commercial use to treat cancer, but before that, European and American countries have not approved any gene therapy, and glybera is the first in the western world Proponents of gene repair technology insist that it is a cost-effective treatment that, although costly, can permanently cure many patients As for glybera, Casey said the annual cost is not higher than some of the expensive enzyme replacement therapy used in other rare diseases, and the drug has proved effective for at least six years Glybera is a harmless modified virus that transmits an effective gene copy to generate lipoprotein lipase after injection into patients At present, uniqure is also carrying out gene therapy research on hemophilia and an early project on heart failure research If successful, gene therapy may not be limited to rare diseases Some industry analysts predict that once the trial is successful, gene therapy will have more common diseases and lower costs, because manufacturers should be able to recover R & D investment from a larger population of patients In the gene therapy market, uniqure's competitors include spark therapeutics and Bluebird bio On June 6, the U.S Food and Drug Administration (FDA) granted spark therapeutics product spk-rpe65 a breakthrough therapeutic status, which is used to treat night blindness caused by gene mutation Bluebird is committed to the development of drugs for the treatment of nervous system and blood system diseases In the past year and a half, Bluebird and uniqure have successfully listed on Nasdaq, reflecting the growing interest of investors in this field Among the major pharmaceutical companies in the world, Bayer announced to obtain the gene therapy project of dimension therapeutics for hemophilia for us $252 million this year, while Novartis just set up a cell and gene therapy department.