Dynamite! Is the first drug in the United States worth more than $1 million about to be born?

What is the "magic drug" of this drug? Its price is so high that the general public can only hold back It is developed by famous pharmaceutical companies biomarin, spark therapeutics and uniqure It is a new type of gene therapy, aiming to affect the ability of blood coagulation and treat hemophilia Its therapeutic mechanism is to provide healthy spare genes in the process of gene replication and expression, so as to make up for hemophilia caused by correction of gene defects and achieve the purpose of curing or effectively improving the disease The birth of new gene therapy has brought hope to patients suffering from diseases, but its price is not affordable for ordinary people Leerink's analysts once wrote in a research report on Monday that the price of the new drug will rise to $1.5 million, especially in the context of rising prices of prescription drugs in the United States It is also reasonable for the value of new gene therapy to enjoy amazing "pricing" The journey of hemophilia treatment is long and difficult According to leerink, there are about 20000 people suffering from hemophilia in the United States Factor replacement therapy is currently the main treatment for hemophilia patients The annual cost of adult patients is about 580000 to 800000 dollars, while for some patients, the annual cost may reach 1 million dollars, or even higher Therefore, the author believes that although the new drug gives most patients "a glimmer of life", its price is unbearable The gene therapy drug is not the first to be approved in the United States Luxturna, developed and produced by spark in December 2017, is the first gene therapy drug to correct gene defects and the first gene therapy transported by AAV to improve patients with retinopathy (IRD) caused by gene defects This therapy uses AAV to introduce healthy RPE65 gene into patients, so that patients can generate normal functional proteins to improve vision It can not only treat Leber's congenital amaurosis, but also treat other eye diseases caused by RPE65 gene mutation But for the immune response caused by virus, the drug can only be effective in a limited time Luxturna's early approval caused a heated debate in the pharmaceutical industry, mainly because there are only two kinds of gene therapy approved and used in Europe, while the United States is extremely cautious about the approval of any gene therapy, and the probability of approval is very small Luxturna's successful entry into the market not only marks its historic significance, but also another milestone after Novartis car-t This directly led to a market value of $850000 after it was approved in December The so-called priceless medicine is because of its existence value When human beings are suffering from rare diseases, these orphan drugs play a huge role in life What's more, in the process of research and development of gene drugs, pharmaceutical enterprises need to spend years continuously on the cultivation of target viruses, the selection and expression of target genes, and the production of final drugs Even if it goes public, the cost of health care and the market cannot bear such a burden Therefore, this is also the key to the birth of rare drugs in the world The arrival of this new gene therapy not only breaks the threshold of drug price in the United States, but also provides impetus and new ideas for global research on gene therapy Content source: https:// Region = World