Take stock of 23 breakthrough therapies by 2020 The most innovative pharmaceutical companies Traditional pharmaceutical companies only...

In July 2020, the Drug Review Center (CDE) of the State Drug Administration proposed the first breakthrough drug review procedure, and the Chinese version of "breakthrough therapy" was officially launched.
in the past six months, 23 drugs have been identified as breakthrough drugs.
more than 20 enterprises from both inside and outside China.
U.S. version of Breakthrough Therapy (BTD) was created by the FDA in July 2012 to accelerate the development and review of new drugs for serious or life-threatening diseases.
As another FDA drug review channel following fast-track, accelerated approval, and priority review, fda-certified drug development can be further guided by closer guidance, including senior FDA officials, to ensure that new treatment options are available to patients in the shortest possible time.
as new drug research and development has become more difficult in recent years, the FDA's increasingly stringent applications for new drugs are common problems faced by the world's major drug companies.
can get the FDA's favor and support is undoubtedly equivalent to a strong needle, encouraging the development of enterprises.
"breakthrough therapy" has also made communication between governments and businesses closer, while also making it easier for the market to market drugs that are in dire need.
In July 2020, CDE issued a breakthrough therapeutic drug review procedure, which makes it clear that during drug clinical trials, there is sufficient evidence to support the prevention and treatment of diseases that seriously endanger life or seriously affect the quality of life, such as innovative drugs or modified new drugs with clear clinical advantages, and can be applied for the application of breakthrough therapeutic drug procedures in phase I. and II.
China Drug Audit Center will also communicate and exchange priority resources for drug priorities included in breakthrough therapeutic drug procedures, strengthen guidance and promote drug research and development.
in just 6 months, CDE awarded 23 breakthrough therapeutic drugs covering the more popular innovative therapies of the day, including CAR-T therapy, ADC, dual-specific antibodies, ASO therapy, and many other small molecule inhibitors, involving targets such as RET, EGFR, JAK, ROCK2, PI3K, etc.
these products are from large international pharmaceutical companies such as Takeda, Novarlor, Pfizer, but also from Chinese companies such as Hengrui Pharmaceuticals, Kangfang Bio, Koji Bio, Wanchun Brin and other .01 Emerging innovative pharmaceutical companies: license in and CAR-T On July 10, three companies submitted applications, including Nanjing's legendary CAR-T therapy, which was the first to get the first breakthrough treatment drug identification from the China Drug Administration in August.
LCAR-B38M is a CAR-T therapy that targets B-cell mature antigens (BCMA) developed by Legendary Bio for adult patients with relapsed or refractic multiple myeloma (MM).
Three years ago, Legendary Creatures' LCAR-B38M surprised ASCO with a 100% objective mitigation rate at its annual meeting, which also attracted global pharmaceutical giant Jansen Heavy Gold to buy the joint development and commercialization of the product, setting a record for the largest down payment authorized by Chinese pharmaceutical companies for foreign patents at that time.
three years later, at the end of 2020, Jansen/Legendary Bio announced that it had begun rolling applications for biological agent licensing (BLA) for this product for the treatment of adult recurrence and/or refragrant multiple myeloma.
this listing is based on a key Ib/II clinical study.
99 percent of the 97 subjects recruited for the study received end-line treatment, 88 percent of whom had received at least three-line treatment in the past.
study found that after 12.4 months of middle follow-up, the independent reviewed 97 per cent of ORRs, including 67 per cent of sCR (strict total remission), 26 per cent of VGPRs (very good partial mitigation) and 4 per cent of PR.
overseas markets, LCAR-B38M has been recognized by the FDA and EMA as a breakthrough therapy and a priority drug.
, at present, the number of CAR-T products for MM is high, the future MM CAR-T treatment market will be more competitive intensity.
Wanlian Securities believes that LCAR-B38M has a certain first-mover advantage and clinical effect advantage, is expected to further catalyz commercial operation to good, in the domestic breakthrough therapy will also further attract the attention of the innovative drug market and the related construction of the payment system.
In addition, in 2020 by virtue of independent research and development products to obtain CDE breakthrough therapeutic drugs companies are Wanchun Brin, Li Pharmaceuticals, Kangfang Bio, Kozi Pharmaceuticals, Heyuan Biologicals, Rongchang Biologicals, Microcore Biology and Dizhe Pharmaceuticals.
among them, Kozi Pharmaceuticals, Mingju Biologicals (Pharmaceuticals) and Heyuan Bio's products are also car-T therapies.
Wanchun's Punabrin has been recognized by breakthrough therapies in both countries almost simultaneously as a product used to prevent chemotherapy-induced neutral granulocytosis (CIN) of non-myelin malignancies.
since 1991, only G-CSF has been approved for the prevention of severe CINs, and Punablin is expected to accelerate its launch as the first breakthrough treatment in 30 years in terms of standards and clinical benefits for severe CIN adaptation.
, a microcore organism, is also an independently developed product, a novel mechanism that targets three-way selective kinase inhibitors and belongs to small molecule anti-tumor originals.
domestic and foreign products are not listed.
addition, some of the products identified as from China's local innovative pharmaceutical companies are imported overseas, such as Keystone Pharmaceuticals' RET inhibitor BLU-667 was introduced in 2018 from Blueprinte, the upr for blood tumors. Oleselan injections were introduced from GlycoMimetics, BN101 from Kadmon, USA, in 2019, and Nefecon slow-release capsules from Yunding, a new drug that set a three-year listing record.
It is worth noting that there are two other products identified as related to the research and development center of multinational pharmaceutical companies in China, the first of which is the new drug AK0529 as a new RSV fusion protein inhibitor, which originated at Roche China Research and Development Center, and which obtained AK0529's global development rights license from Roche in June 2014.
Pharmaceuticals, which is based in China Investment Innovation and AstraZeneta's Innovation Research and Development Center in Asia and Emerging Markets, will lead Lilly Asia in its Round A financing in 2020.
All in all, whether by independent research and development, or from overseas, in recent decades, emerging local innovative pharmaceutical companies accounted for half of the Chinese version of breakthrough drugs, 15 of the 23 drugs from them, including 5 in license in, CAR-T therapy four.
same time, in 2020 in the United States to obtain breakthrough therapy, orphan drugs and other recognized Chinese enterprises, innovative pharmaceutical companies still occupy the majority, such as Asheng Pharmaceuticals in a year alone nine FDA orphan drug certification, the momentum.
02 Multinational pharmaceutical companies: Innovative and obviously with the Nanjing legend at the same time to submit a breakthrough treatment drug applications are Yang Sen's lung cancer EGFR targeted drug JNJ-61186372 and Takeda's lung cancer EGFR targeted drug TAK-788.
both products are targeted at EGFR targets, but each has its own characteristics.
TAK-788 was also recognized as a breakthrough between China and the United States in 2020, and is the first foreign-funded enterprise product in China to be recognized as a breakthrough therapeutic drug.
is used to treat patients with partial late stage or metastasis non-small cell lung cancer (NSCLC) who have received at least one systemic chemotherapy in the past with exon 20 exon insertion mutations.
for this particular type of NSCLC, there are currently no approved treatment options worldwide.
Jansen's JNJ-61186372 is a dual-specific antibody that targets EGFR and cMET and is currently clinically relevant to lung cancer and other solid tumors worldwide.
The adaptations to be incorporated into the breakthrough therapy are to treat patients with platinum-containing dual-drug chemotherapy who progress during or after chemotherapy, or who are insulated with platinum chemotherapy, or who have a metastasis or surgically unseistable NSCLC mutation inserted into the exon no. 20 of the platinum-containing chemotherapy.
addition to Takeda and Janssen, Novaral, Pfizer, Novarma, AstraZeneta and Bayer have also made big in the market.
Novart's TQJ230 is a specific targeted lipoprotein (a) elevated antonym drug (RNA therapy), and its Phase II clinical findings, presented at the American Heart Association's (AHA) annual general meeting in 2018, raise concerns about the effects of lipoprotein (a) in patients with cardiovascular disease.
year, Novartic introduced the drug, which has just begun phase III clinically and was declared clinically in China a year ago.
Pf-06651600 tablet is an oral acid kinase (JAK3) inhibitor, approved in March 2020 in China clinical, adaptive for the treatment of ≥12-year-old patients with severe baldness, which is also the first approved clinical treatment of bald new drugs.
AstraZeneta and Phase 1 developed DS-8201a is a new ADC drug, this time the breakthrough treatment drug was identified as benefiting from the excellent performance in HER2-positive stomach cancer, which is also her2-positive gastric cancer the first ADC drug.
the drug was previously awarded three breakthrough therapies by the FDA for the treatment of HER2-positive breast, stomach and non-small cell lung cancer patients.
be included in the breakthrough treatment publicity list for the treatment of adult patients with marginal lymphoma using freeze-dried preparations for Bayer copanlisib injections.
copaanlisib injection freeze-dried preparation is a PI3K inhibitor, which can induce tumor cell death, and inhibit the proliferation of primary malignant B cells, and thus achieve the goal of controlling the development of lymphoma.
, according to data released by Bayer, patients with lymphoma in marginal areas who had received at least two treatments had a total remission rate of 69.6 percent after copanlisib treatment.
the FDA's accelerated approval in September 2017 for the treatment of relapsed follicle lymphoma (FL).
it has also been granted FDA-granted orphan drug qualifications for the treatment of FL and MZL.
03 Local old drug companies: Only Hengrui multinational pharmaceutical companies a total of 6 products were identified, less than half of the local innovative pharmaceutical companies, but the local old drug companies in this six months to harvest less.
Hengrui has two products to obtain breakthrough treatment drug identification, but for the same adaptation certificate of the joint drug, that is, injection with carellijudan anti-combined nephloric acid famini capsule treatment after first-line treatment failure of relapsed metastatic cervical cancer.
is also the only PD-1 therapy in China that has received this recognition, while Hengrui is also the only traditional old drug companies.
in the first six months of CDE's breakthrough drug identification, local innovative pharmaceutical companies clearly dominated, followed by multinational pharmaceutical companies, while traditional companies appeared slightly behind.
this recognition also means that PDC will prioritize resources for communication, enhanced guidance and promotion of drug development, with more efficient CDE guidance and close supervision.
, it also helps the drug to get on the fast track and reduce the time it takes to develop and get to market.
example, statistics show that the time to market for BTD drugs is reduced by an average of about 3 months, and the clinical development time is reduced by 2-3 years compared to drugs that do not have BTD.
CDE's trial document stipulates that applicants who have been assessed as eligible for inclusion in the breakthrough therapeutic drug procedure may also apply for conditional approval and priority review and approval when applying for a drug market license.
the inclusion of breakthrough therapeutic drug procedures will undoubtedly increase confidence and confidence in research and development for the pharmaceutical companies themselves.
addition, the inclusion of breakthrough therapeutic drug procedures can also help pharmaceutical companies gain access to capital markets.
But on the other hand, successful inclusion in a breakthrough treatment drug program does not mean that there is a 100 percent guarantee of approval for listing, and CDE will initiate termination procedures when it is found that clinical trials of drugs incorporated into the breakthrough therapeutic drug program no longer meet the inclusion criteria.
in the U.S., BTD determinations are revoked by the FDA every year.