The first breakthrough therapy flower legend What challenges will domestic innovative drugs face in the future?

On August 5th the National Drug Administration's Drug Review Center (CDE) announced the first proposed breakthrough therapy on its official website.
public information shows that the LCAR-B38M CAR-T cell re-transfusion agent (LCAR-B38M cell preparation) reviewed by Nanjing Legendary Biology has been approved by CDE to be included in the breakthrough treatment procedure.
in accordance with the Provisions of the Breakthrough Treatment Drug Review Procedure (Trial) issued by the State Drug Administration on July 8, no objection (drugs) within 5 days shall be incorporated into the Breakthrough Therapeutic Drugs Program.
that LCAR-B38M cell preparations will be the first breakthrough therapeutic drug in China without incident.
01 Why is it? In July 2012, the FDA Breakthrough Therapy Certification (BTD) was approved by Congress as another special channel for rapid approval of new drugs by the FDA, following Fast Track, Priority Review, and Accelerated Approval.
BTD aims to shorten the development and review of new drugs for "serious or life-threatening diseases".
, Two new drugs from Vertex Pharmaceuticals were the first to be certified by BTD, and it took six months from the time the law went into effect to the time the first drug was certified by BTD.
with his mountain stone, the process is much faster in China.
July 8, the State Drug Administration issued the "Breakthrough Therapeutic Drug Review Process (Trial)" and other three documents announced that the application and review of breakthrough treatment drug procedures with provisional standards and feasible workflow, the Chinese version of the "breakthrough therapy" channel officially opened.
10 July, Legendary Creatures submitted its application to the CDE, which, according to the August 5 publicity, took just over 20 days to review, well below the standard of "feedback within 45 days of receiving the application" in the pilot document.
if the application is successfully approved, it will take only one month from the announcement of the State Drug Administration to the time when the first drug is included in the breakthrough treatment drug program.
Although the State Drug Administration has not published the details of the evaluation, but the trial document more detailed application process and clear standards should be an important factor in the successful completion of the enterprise application and evaluation work.
The pilot document states that breakthrough therapeutic drugs are suitable for the prevention and treatment of diseases that are seriously life-threatening or seriously affect the quality of life during clinical trials and that there is no effective means of prevention or that there is sufficient evidence of significant clinical advantage over existing treatments, such as innovative or modified new drugs.
are required to be applied for in phase I and II clinical trials, usually no later than before Phase III clinical trials are conducted.
addition, the pilot document provides for the conditions under which drugs are applied: 1. For the prevention and treatment of diseases that are seriously life-threatening or that seriously affect the quality of life.
2. Where there is no effective means of prevention and treatment, the drug may provide effective means of prevention and treatment, or the drug has a clear clinical advantage over existing treatment methods, i.e. single use or in combination with one or more other drugs, with significant improvements in one or more clinically significant endpoints.
the "effective prevention and control means" and "obvious clinical advantages" of the situation is explained in detail.
contrast to the United States, BTD was sought after by pharmaceutical companies from the start, but at first neither its definition nor the specific criteria were clear.
It wasn't until June 2013 that the FDA issued "Industry Guidelines for Accelerated Approval processes for Drugs and Biologics for Severe Diseases" to detail the conditions for accelerated applications for new drugs.
LCAR-B38M, which is to be incorporated into the breakthrough drug program, is a CAR-T cell therapy developed by Legendary Biology that targets B-cell mature antigens (BCMA).
Legendary Creatures partnered with Janssen in 2017 to develop and promote the innovative treatment for recurrent/incurable multiple myeloma (MM) worldwide.
MM is a plasma cell tumor, which accounts for 13% of malignant tumors in the blood and is considered an incurable disease.
BCMA is the ideal target for MM immunotherapy, and LCAR-B38M has shown excellent efficacy in clinical trials for MM patients.
early results showed an objective remission rate (ORR) of 88%, and the interim trial results released by Jansen at the annual meeting of the American Hematological Society (ASH) in 2019 showed a total reprimission rate of 100%.
In addition to the "compliance" of the product itself, LCAR-B38M is also recognized by the European Medicines Agency (EMA) as a Priority Review Drug (PRIME) in 2019 and the BTD awarded by the FDA (codenamed JNJ-4528 for experimental drugs in the United States and Europe).
According to public information, two pharmaceutical companies in addition to Legendary Biology submitted applications for breakthrough treatment drugs to the State Drug Administration: on July 17th Li's Big Pharmaceuticals announced that it was using PD-L1 to treat recurrent and metastatic cervical cancer. Monotherapy ZKAB001 applied to the State Drug Administration for a breakthrough treatment drug, and on July 22, Repolar Pharmaceuticals also announced that it had applied to identify MAX-40279 as a breakthrough treatment for FRT3 wild acute myeloid leukemia (AML).
according to the press release, in previous phase I AML clinical trials conducted simultaneously in Australia and China, MAX-40279 had 36% ORR for patients treated with FRT3 wild AML end-line treatment.
02 is fast-track, and it's just fast-track as the first product to be included in the breakthrough therapeutic drug program, and the development and launch of the LCAR-B38M will give priority to the benefits of the policy.
the pilot document, the Drug Review Center communicates and prioritizes resources for drugs included in breakthrough therapeutic drug programs, strengthens guidance and promotes drug development.
this means that drug development, which incorporates breakthrough treatment procedures, can be guided more efficiently and closely regulated by CDE and, to some extent, will be "cared for" by the authorities.
also means that drugs included in breakthrough drug programs can go fast enough to reduce the time it takes to develop and go to market.
example of BTD in the United States, statistics show that the average time to market approval for BTD drugs is reduced by about 3 months, and the clinical development time is reduced by 2-3 years compared to drugs that do not receive BTD.
the pilot document, the applicant may also apply for conditional approval and priority review and approval when applying for a drug listing permit if the applicant has been assessed to meet the relevant conditions for the inclusion of the breakthrough therapeutic drug procedure.
to the breakthrough therapeutic drug program will undoubtedly increase confidence and confidence in research and development for the pharmaceutical companies themselves.
the breakthrough therapeutic drug program not only stimulates drug research and development, but also helps pharmaceutical companies gain access to capital markets.
shares of pharmaceutical companies rose an average of 6 percent the day after the announcement, according to an analysis by the American Chemical Digest (CAS).
the first immunotherapy drug is certainly important good news for the legendary creature, which just reaped the second-largest mid-year financing in the first half of the year on Nasdaq in June.
, on the other hand, the successful inclusion of breakthrough drug procedures does not mean that approval and listing is 100 percent guaranteed.
breakthrough drug program can only speed up the process of new drugs on the market, and there is no direct relationship with the final approval of the market.
, drugs included in the breakthrough therapeutic drug program may also be terminated.
the pilot document states that CDE will initiate termination proceedings when it is found that clinical trials of drugs included in breakthrough therapeutic drug procedures are no longer eligible for inclusion.
year in the United States, BTD's determination is revoked by the FDA after it is granted.
In addition, many pharmaceutical companies and the media have deliberately or unwittingly raised the "breakthrough" to a high level in their publicity, but it is not known whether these halo drugs or therapies, dubbed "breakthrough", will eventually deliver on their commitment to efficacy and meet patient expectations.
but breakthrough therapy itself may not produce "breakthrough" results, but it does have a positive effect on "encouraging research and the creation of drugs with clear clinical advantages."
as of July 31, 2020, the FDA received 974 BTD applications, of which 377 were BTD certified and 163 BTD drugs were finally approved for market.
believe there will also be more domestic innovative drugs on the road to "breakthrough therapy" to try.
original title: Behind the legend of the first "breakthrough therapy": Home-made innovative drugs to the fast track, what challenges will be faced next?