10The new drug breakthrough treatment certification approval fast track can help China's innovation to go global

In order to encourage innovation and meet the urgent clinical needs, this year China set up a variety of rapid review listing channels.
, breakthrough therapeutic drugs target innovative or improved new drugs with significant clinical advantages during clinical trials.
drug, which is included in the breakthrough therapeutic procedure, is expected to accelerate approval.
The industry generally believes that the significance of establishing a breakthrough therapeutic drug approval channel is to explore the advantages of products through early clinical data, focus on multi-resource research and development, shorten the development cycle of advantageous products, improve the number of successful approvals, reduce the number of new molecular entities stopping before clinical, so that patients get better treatment at an early stage.
, the State Drug Administration Drug Review Center "breakthrough treatment publicity" column was updated again, breakthrough therapy drug approval ushered in a speed up.
01 Breakthrough Therapy Re-adding New Army Since the opening of the breakthrough treatment channel in July this year, 10 new drugs including Hengrui, Nanjing Legend, Takeda and other companies have been incorporated into the breakthrough treatment varieties.
according to the latest information on the website of the Drug Review Center (CDE) of the State Drug Administration of China, three new drugs are proposed to be included in the breakthrough treatment varieties, namely Novarma (NVS. US) in the study of antispolytic oligonucleotide (ASO) therapy TQJ230, Yunding Xinyao (01952) introduced the IgA nephropathy in the research drug Nefecon, and Kozi bioBCMA targeted CAR-T cell injection CT053.
CT053 all-human anti-BCMA self-body CAR-T cell injection (Kozi bio) adaptation certificate: recurrence difficult to treat multiple myeloma (R/R MM) CT053 human anti-BCMA self-body CAR T cell injection is The first class of innovative drugs developed by Kozi Bio is a T-cell modified with a chiline antigen -CAR modified with human antibody targeting B-cell mature antigen (BCMA), with the main target adaptive disorder being relapse/difficult to treat multiple myeloma.
ct053 has shown excellent results in an exploratory Phase I clinical study in China.
19 of the 24 patients with recurring multiple myeloma (R/R MM) showed complete remission, with a total remission rate of 79.2%.
, no level 3 or higher cytokine release syndrome (CRS) events were observed.
, CT053 has been awarded the FDA Advanced Therapy for Regenerative Medicine (RMAT) and Orphan Drugs, as well as the European Medicines Agency (EMA) For Priority Medicines (PRIME) and Orphan Drugs.
note that Kozi Bio CT053 is the third CAR-T product to be incorporated into breakthrough therapies.
TQJ230 Injection (Novaral): Adaptation Certificate: Reducing the risk of cardiovascular disease by reducing lipoprotein (a) levels TQJ230 is an antisant oligonucleotide drug developed to inhibit the expression of Lp(a) protein by reducing mRNA levels by combining with mRNA expressing Lp(a).
In February 2019, Novartra acquired the drug from Ionis subsidiary Akcea for $150 million, responsible for the global development and commercialization of TQJ230 and as one of Novartic's key products in the cardiovascular research and development pipeline.
, TQJ230 injections were approved clinically in China to reduce cardiovascular risk.
Nefecon Slow Release Capsules (Calliditas Therapeutics/Yunding Shinyao): Adaptation Certificate: Primary IgA Kidney Disease (IgAN) Nefecon is a slow-release capsule developed by Calliditas using TARGIT technology that releases Budineid only at the source of lgA nephropathy in the lower part of the small intestine, the Pyle collection lymph node region.
TARGIT technology allows active ingredients to pass through the gastrointestinal tract without being absorbed, and the drug is released pulsed only when it reaches the lower part of the small intestine.
will be responsible for nefecon's development and commercialization in Greater China as early as 2019 when Yunding Xinyao and Caliditas reach a partnership.
Caleditas plans to submit an accelerated approval application to the FDA and a conditional approval request to the European EMA in the first quarter of next year.
procedures, the proposed inclusion of breakthrough treatment varieties will be formally incorporated after the public announcement period of breakthrough treatment drug varieties.
generally believed in the industry that for drugs with significant clinical advantages, the resources for priority review by the Drug Review Centre will be tilted upon the identification of breakthrough drugs.
02 Fast track of new drug research and development and approval In July this year, the State Drug Administration organized and formulated a series of documents, such as "Breakthrough Therapeutic Drug Review Procedure (Trial)", which further improved the regulatory path of accelerated review and approval from the system.
In fact, as early as November 2019, the CDE had publicly solicited views from the community on the Breakthrough Therapeutic Drugs Work Procedure (Draft for Comments) and the Priority Review Approval Procedure (Draft for Comments), which has generated tremendous industry concern.
With the new version of the Drug Registration Management Measures clearly set up breakthrough therapeutic drugs, conditional approval, priority review and approval, special approval of the four accelerated channels, as one of the four accelerated channels of breakthrough treatment, will strengthen the whole process of communication, improve the efficiency of the completion of clinical trials, focus on supporting and accelerating the development of high-value new drugs.
based on the relevant content of the Breakthrough Therapeutic Drug Review Process document, Breakthrough Treatment aims to accelerate the development and review of new drugs for serious or life-threatening diseases.
In accordance with the requirements of the relevant documents, breakthrough therapeutic drugs are applicable: during clinical trials of drugs, used to prevent and treat diseases that are seriously life-threatening or seriously affect the quality of life, and there is no effective means of prevention and treatment, or compared with existing treatments, there is sufficient evidence of a clear clinical advantage of innovative drugs or modified new drugs.
for this class of drugs, applicants can apply for breakthrough therapeutic procedures at the Phase I and II clinical trial stage, usually no later than before phase III clinical trials are conducted.
In recent years, China's mechanism to speed up the review and approval of new drugs is almost identical to the FDA's in terms of rules, and in the reform of China's drug review system, the four accelerated channels set up to encourage innovation and meet clinical needs are easily reminiscent of the FDA's four channels for accelerating approval and encouraging innovation.
2012, the FDA introduced breakthrough therapeutic findings for accelerated drug development and review for serious diseases.
The FDA may take a number of actions to accelerate research and development and apply for reviews for drugs recognized for breakthrough treatments, including comprehensive guidance on efficient drug development programs, organizational commitments to include senior management, rolling reviews, priority reviews under certain conditions, and so on.
data show that between 2012 and 2019, the FDA awarded about 340 breakthrough therapies, of which 150 were approved for market, with a significantly higher probability of approval than the industry average;
It is clear that obtaining breakthrough treatments will shorten the lengthy and expensive development process and allow promising drugs from early research to enter the market as quickly as possible without having to complete the development of traditional Phase I-III clinical studies.
, breakthrough therapies have become the weather for drug approval, representing the latest breakthrough in drug research and development.
in the industry, China's breakthrough therapy will be an important label for the future, but also represents China's level of innovation.
most important of China's breakthrough therapies is to further accelerate the process of listing drugs with clinical advantages in China, and to further meet the clinical treatment needs of major diseases in China.
03 The internationalization of innovative drugs will take time And policy innovation is driving innovation in the pharmaceutical industry.
breakthrough treatment fast track is designed to accelerate the launch of high-value innovative varieties and better meet China's unsolt clinical needs.
At present, China's innovative pharmaceutical companies are rapidly changing their innovation models, from me-too to me-better, to co-introduction of world-class innovation, to best-in-class development, we believe that China's first-in-class original innovation will become more and more common.
it must be acknowledged that China's local innovative pharmaceutical companies are already on their way to that goal.
Since January 2019, when Zebutini of Baiji Shenzhou became the first Chinese innovative drug to be approved by the FDA, less than two years later, China's innovative pharmaceutical companies have achieved 0 breakthroughs in the fields of polymer antibodies, CAR-T cell therapy, etc., and more and more domestic drugs have been certified as orphan drugs in the European and American markets, etc., proving to the world the innovative power of Chinese medicine.
industry sources point out that Thanks to the reform of the State Drug Administration's drug registration and approval review system, the return of scientists and the entry of large amounts of money, China's innovative drug research and development ecosystem is improving and the role of China's pharmaceutical innovation in the world has changed.
In Building a Sustainable Chinese Pharmaceutical Innovation Ecosystem, the U.S. accounted for about half of the world's innovation contribution in 2016, with Japan, The U.K., Germany and other countries in the second tier contributing about 5% to 10% of innovation, and China being classified as the third tier, with innovation contributing only about 4%.
2019, China's contribution to global pharmaceutical research and development will rise to 4% to 8%, making it the second-tier team, behind the United States and Japan.
undeniably, China has become an important position for global pharmaceutical innovation.
, has Chinese pharmaceutical innovation really entered the world's second tier? In the first half of 2020, the number of clinical trials in China has surpassed that of the United States and ranked first in the world, according to CoreLogian.
compared with the United States, the current clinical trials in China show a more concentrated target characteristics.
2019- September 2020, Chinese companies conducted research on 553 targets and conducted clinical trials, up from 1,333 in the United States, according to the U.S. Government.
addition, there are more than 100 clinical trials at 16 targets in China, up from three in the United States.
PD-(L)1 as an example, more than 50 companies in China are currently in this area, but the design, data, process and timing of clinical trials are key factors in the success of the drug.
When drugs in the same field are listed into the "hybrid war", if there is no very good team and unique innovation, it is difficult to have a superior competitive advantage in the current situation, there will be a large number of enterprises difficult to get a piece of the pie.
In the eyes of the industry, the current domestic independent innovation ability is still weak, tending to focus more on Me-too drugs, rather than high uncertainty of new target drugs, which is also a real obstacle to domestic innovation drug research and development.
, it is still very difficult for most local biotech companies to gain more review approvals internationally.
drug review system has changed in recent years, the research and development ecology of innovative drugs driven by clinical value and with patients as the core has not yet been formed.
In the view of the founders of some innovative pharmaceutical companies, Chinese pharmaceutical innovation, as an ecological system, has entered the second tier in some links or subdivided technical levels, but as a complete ecological system, drug innovation all relevant parties need to work together to cultivate innovative drug research and development ecology adapted to innovation.
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