US $ 2.8 million!"The most expensive medicine in history" is approved by domestic companies in the United States.

China Times (chinatimes.net.cn) reporter Sun Mengyuan Yu Na Beijing report

Recently, the US FDA approved the listing of Bluebird Biological ZyNTEGLO to treat β-thalassemia. This is the first gene therapy for approval and treatment in the United States. It is reported that Zynteglo is priced at $ 2.8 million, which has surpassed Novartis's gene therapy Zolgensma (priced at 2.1 million US dollars), known as "the most expensive medicine in history".

On August 23, Jess Rowlands, head of the Blue Bird Biotechnology media, said in an email of the reporter of the Huaxia Times, "Today, the average lifelong care cost of patients with blood transfusion dependencies β Medica (TDT) is $ 6.4 million. The price of ZyNTEGLO reflects the clinical benefits we showed in the third stage of research, as well as the potential improvement of the quality of life of patients, the expected cost savings of the medical system, and the extensive possible achievement of patients from long -term and heavy blood transfusion. Social benefits. American insurance companies attach great importance to the needs of TDT patients and the huge value that ZyNTEGLO may provide. Blue birds are confident in our ability to realize patient access in the United States. "

Tan Yazhen, an independent commentator of the pharmaceutical industry, told the reporter of the Huaxia Times that drug pricing is so high, and a small number of patients are expected to obtain cost reductions through charity donations or participation in clinical research. Such gene therapy products should be the effect of lifelong cure for one or several treatments, so it can be imagined to imagine the payment arrangement of mortgage loans. The probability of this price cannot be continuous, but it can be recovered as much as possible before the listing of similar products.

"The most expensive medicine in history"

Β-Mediterranean anemia is a hereditary hemolytic disease and is one of the most common monocular diseases. Due to the serious lack of functional β-beadin, a considerable part of patients need to be transfused regularly to survive, resulting in blood transfusion dependent thalassemia (TDT).

Public information shows that Zynteglo belongs to the disposable LVV gene therapy, which is copied by adding the functional copy of the βA-T87Q-pearl protein gene to the patient's own hematopoietic (blood) stem cells, which produces modification. Functional adult hemoglobin (HBAT87Q), which can achieve the purpose of reducing the needs of patients' blood transfusion.

On August 17, the FDA approved the Blue Bird Biological Company's ZyNTEGLO to go public. Because ZyNTEGLO priced at $ 2.8 million in the United States, it exceeded the price of Zolgensma, which had previously priced at the Poor Pharmaceutical's spinal muscle atrophy gene therapy, has also become the "most expensive medicine in history."

On August 23, Jess Rowlands said in the email of the China Times reporter that it is necessary to clarify that the current American media reports pointed out that ZyNTEGLO is the highest price in the United States, but Europe has approved more expensive therapies. The blue bird cannot confirm the price of other therapies.

Bluebird chief business officer publicly stated that although ZyNTEGLO's treatment costs were as high as US $ 2.8 million, the drug pricing regulatory agency stated that the cost of the treatment was as high as $ 3 million per dose. Considering that the blood transfusion of a lifetime may cost $ 6.4 million, the pricing of this therapy is actually reasonable.

The reporter found that in June 2019, Zynteglo was approved in Europe, but in April 2021, Blue Bird Bio announced that Zyteglo was withdrawn from the German market. In subsequent investors exchanges, it also said that it would be withdrawn from the European market in early 2022. Some analysts believe that withdrawal of the market is related to its high pricing, and does not reach a price consensus with German regulators. In this regard, Jess Rowlands replied to the China Times reporter: "We think the price and value of Zynteglo is consistent. There are fundamental differences between the health systems of the United States and Europe. The value of treatment is likely to relieve treatment for patients with chronic blood transfusion. "

However, ZyNTEGLO was postponed by the FDA for security issues. FDA said in the file that patients in the test caused blood cancer due to this treatment, so the therapy may have carcinogenic risk. Jess Rowlands said: "Blue Bird attaches great importance to the safety of patients. Although there is no blood system malignant tumor in patients treated with ZyNTEGLO so far, we have a long -term follow -up study to monitor the results of 15 years in patients in our clinical research. And plan to conduct a registration study to track patients who are treated in the same time in the business environment. "

There are also layouts in China

Public information shows that due to the high cost of blood resources and the cost of iron chelating, only part of domestic TDT patients can maintain standardized blood transfusion and standardized de -iron treatment. The survival rate of TDT patients is significantly lower than that of developed countries. Therefore, domestic enterprises are developing the treatment of poor genetic therapy to improve the availability of drugs.

According to incomplete statistics, clinical trials for thalassemia are carried out in an orderly manner. The products of Suzhou Xinya Gene Biotechnology Co., Ltd. and Beijing Chengnuo Medical Technology Co., Ltd. are all in the pre -clinical research stage. The two research products of technology Co., Ltd. have entered the clinical trial of Phase I. Tan Yayi told the reporter of "Huaxia Times" that whether family and society will choose to give a very small number of severe children with very expensive genetic treatment, on the one hand depends on the actual effect of gene therapy, on the other hand, it depends on the family and society of society and society. Economic resources. If Chinese companies have successfully developed products, the price will definitely be significantly reduced to the financial and scope of Chinese families.

On January 18, 2021, the Boya series had approved the clinical trial application of the CRISPR/CAS9 gene editing therapy for the China Drug Administration of the National Drug Administration of China. The first gene editing therapy products and hematopoietic stem cell products were approved by the State Drug Administration to conduct clinical trials. Boya's album stated that in China, 300,000 patients with medium -sized thalassemia are still there, and blood transfusion dependent β -neighborhood anemia still has huge unsatisfactory medical needs.

On December 30, 2021, Shanghai Integrated Gene Technology Co., Ltd. announced that the "Slow Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus Virus The clinical experiments of the treatment of blood transfusion dependencies β-thalassene anemia "have achieved preliminary results. This is the first domestic report that has reported a successful case based on the treatment of β-thalassemia based on chronic virus carriers.

On August 16, 2022, Bangyao Bio announced that the clinical trial application (IND) of the gene therapy product "BRL-101 autologous hematopoietic ancestor injection" for blood transfusion dependent β-thalassemia (IND) Approval by the Administration of Pharmaceutical Review Center (CDE). Professor Liu Mingyao, the founder and chairman of Bangyao Bio, said, "The approval of this time not only marked the official development of the company's development, but also means that Bangyao Bio has successfully moved closer to genetic and cell therapy."

Bangyao Biological said that in the current traditional therapy of the ground, hematopoietic stem cell transplantation is the only way to cure β-thalassemia, but it is expensive and difficult to match. Only a small number of patients can get transplantation. The hematopoietic stem cells are returned to the patient's body after genetic correction, which can solve the problem of insufficient source of hematopoietic stem cells and difficulty in matching. The progress of CRISPR (gene editing technology) provides possible treatment strategies.

Deng Zhidong, general manager of Hainan Boao Medical Technology Co., Ltd., told the reporter of "Huaxia Times" that gene therapy began to cross the golden age after experiencing the ups and downs of the industry, especially after the rise of gene editing, the development momentum was even more fierce. In terms of domestic gene therapy layout, it is still early, but the national policy is strongly supported, the resources are collected, the prospects are available, and the blue ocean will be used. In the future, there will be more funds, talents, and enterprises.

Xuexue Editor: Editor of Yan Yuan: Chen Yanpeng

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