700,000 "high-priced drugs" dropped to 33,000!

February 28 is International Rare Disease Day.
There are more than 20 million patients with rare diseases in China, with more than 200,000 new patients every year
.
There are more than 7,000 confirmed rare diseases in the world, and the number of rare diseases is increasing at a rate of 250 to 280 per year.
The world's population base is huge, and rare diseases are actually not "rare"
.
Recently, Chinese hemophiliacs have called for Roche to cut the price of Emicizumab, which has sparked heated discussions
.
At present, most of the domestic rare disease treatment relies on imported drugs.
According to statistics, more than 40 rare disease drugs have been included in the medical insurance catalog.
About 100 of its drugs have been granted orphan drug qualifications by the FDA.
Among them, there are many domestic pharmaceutical giants such as Innovent Bio, Hengrui Medicine, Fosun Pharma, and CSPC.
Beihai Kangcheng has continued to grow with the "License-in" model
.
700,000 "high-priced drugs" dropped to 33,000! Over 40 Rare Disease Drugs Entered Medical Insurance Recently, an "Open Letter from Chinese Hemophilia Patients to Roche Pharmaceuticals" sparked heated discussions.
The price of the injection has remained high since its launch, and hemophiliacs have called on Roche to cut prices
.
According to statistics, there are currently more than 60 rare disease drugs approved for marketing in China, of which more than 40 have been included in the National Medical Insurance Drug List, involving 25 diseases
.
Among them, multiple sclerosis, pulmonary hypertension, Parkinson's disease (young-onset, early-onset), and hemophilia are relatively more than 5 drugs included in the medical insurance
.
Rare disease drugs that have been included in medical insurance The entry of rare disease drugs into the medical insurance catalog is one of the main paths for price reduction.
In 2021, there are 7 rare disease varieties that have entered the medical insurance catalog for the first time through medical insurance negotiation, including ilovolumab injection, Nosinason Sodium Injection, Human Coagulation Factor IX, Agalsidase Alpha Concentrated Solution for Injection, Chlorfloxacin Soft Capsules, Icitibant Acetate Injection and Fampridine Sustained-Release Tablets
.
Among them, Nosinagen Sodium Injection is the first high-value rare disease drug to be included in the national medical insurance list.
The price of this drug for the treatment of spinal muscular atrophy was once as high as 700,000 yuan per injection.
/ Needle successfully passed the medical insurance negotiation, and the price reduction rate was as high as 95%
.
Over 10 billion market players are competing! Domestic pharmaceutical companies are catching up.
According to data, China's rare disease market is expected to reach 2.
4 billion US dollars (about 15.
2 billion yuan) in 2022, and it will increase to 25.
9 billion US dollars (about 164.
2 billion yuan) in 2030.
The market growth rate is significant and the prospects are broad.
As the rare disease market continues to heat up in recent years, more and more companies are targeting the Chinese market
.
At present, domestic rare disease drugs mainly rely on imported drugs, and multinational pharmaceutical companies have been "sieging cities and looting territories" here
.
Since 2018, more than 80% of the rare disease drugs that have been marketed are imported drugs
.
Sanofi, Roche, Takeda, and Biojian are all old faces in the domestic rare disease market.
After AstraZeneca acquired the rare disease pharmaceutical company Alexion at the end of 2020, it also officially entered the rare disease field
.
At the same time, multinational pharmaceutical companies are gradually increasing the proportion of their business in the field of rare diseases
.
For example, Pfizer readjusted its organizational structure in China at the end of last year, and rare diseases were included as one of the six independent business units; AstraZeneca announced in September 2021 that it would formally establish a rare disease business unit in China
.
Domestic pharmaceutical companies are catching up.
Up to now, about 100 drugs developed by domestic pharmaceutical companies have been granted orphan drug qualifications by the FDA, involving more than 50 pharmaceutical companies, including Innovent Bio, Hengrui Medicine, and Fosun.
Domestic pharmaceutical giants such as Pharma and CSPC, Beihai Kangcheng have grown continuously with the "License-in" model
.
In January of this year, Beihai Kangcheng's rare disease drug macibat oral liquid was declared for listing.
It is planned to be included in priority review for "new varieties, dosage forms and specifications of children's drugs that meet the physiological characteristics of children" for Alagille syndrome 1 year old and above.
(ALGS) patient with cholestatic pruritus
.
Moxibut Oral Liquid was approved for marketing in the United States in September 2021, and is the world's first and only drug for the treatment of ALGS rare diseases
.
In April 2021, Beihai Kangcheng and Mirum, the original researcher, reached a cooperation to obtain the exclusive license to develop and commercialize macibat in Greater China for ALGS, progressive familial intrahepatic cholestasis and bile duct atresia
.
For ALGS, there is no domestic drug for this indication on the market, Beihai Kangcheng is expected to occupy the leading position in the field of rare diseases with the oral liquid of macibat
.
It is worth mentioning that Maxibut oral liquid is not the only case of Beihai Kangcheng's "License-in".
On the contrary, Beihai Kangcheng can be regarded as a veteran of the "License-in" model
.
In the 10 years since its establishment, Beihai Kangcheng has approved 3 products for the market, all of which were imported from abroad
.
Compasso, which prevents and treats oral mucositis caused by chemotherapy, was introduced from EUSA Pharmaceutical Company in the United Kingdom; neratinib, which is used for intensive adjuvant treatment of HER2-positive early breast cancer, was introduced from Puma Biotechnology Company in the United States; and China's first mucopolysaccharide The type II enzyme replacement therapy drug for storage diseases, Haris (Idosulfatase β), was introduced from GC Pharma in South Korea
.
Judging from the varieties under research, in addition to the macibut oral liquid that has been declared for marketing, Beihai Kangcheng has 3 products in the clinical stage, and the other 3 products are in the preclinical stage
.
Among them, including its core product is CAN008 (Asunercept)
.
CAN008 is a CD95-Fc glycosylation fusion protein being developed for the treatment of glioblastoma (GBM), and is currently in phase II clinical trials in China
.
It is understood that CAN008 is from Apogenix, and was introduced into China by Beihai Kangcheng in June 2015
.
In the field of rare diseases, due to the narrow audience and uncertain income, early research and development is more difficult.
Beihai Kangcheng uses the "License-in" path to form a rich product pipeline with its own research and development products to make up for the shortcomings of its own product pipeline
.
Furthermore, the successful use of this model brings considerable returns to the enterprise
.
In December 2021, Beihai Kangcheng successfully listed in Hong Kong
.
Ascentage, CSPC, BeiGene.
.
.
Sailing overseas, rare disease drugs are more often referred to as "orphan drugs".
In order to stimulate the research and development of orphan drugs, the United States promulgated the "Orphan Drug Act" 30 years ago.
, Drugs that have obtained orphan drug qualification can enjoy tax relief for clinical trial fees, exemption of NDA application fees, access to research and development funding, 7-year exclusive rights in the US market and other dividends, which are valid so far
.
For domestic pharmaceutical companies, obtaining orphan drug qualification from the FDA is a recognition of their own research and development strength
.
More importantly, orphan drug designation can become a stepping stone for new drugs to enter the US market, and it is a shortcut for China's innovative drugs to go international
.
On February 14, IBI326, an innovative drug targeting B cell maturation antigen (BCMA) chimeric antigen receptor autologous T cells (CAR-T) jointly developed by Innovent and Reindeer Medical, was granted orphan drug designation by the US FDA , which is mainly used for the treatment of relapsed/refractory multiple myeloma, which is the second drug of Innovent to be granted orphan drug designation by the FDA
.
Status of TOP3 companies that have obtained FDA orphan drug qualifications Judging from the number of FDA orphan drug qualifications, Ascentage Pharmaceuticals, CSPC, and BeiGene are at the forefront
.
Ascentage Pharma has a total of 4 drugs under development that have obtained a total of 13 orphan drug qualifications granted by the U.
S.
FDA, continuously breaking records of Chinese pharmaceutical companies and becoming the leader of the orphan drug pipeline in China; CSPC has 7 drugs under development, a total of 9 2 orphan drug qualifications; BeiGene has also won 9 orphan drug qualifications with tislelizumab and zanubrutinib
.
Ascentage Pharma's third-generation BCR-ABL inhibitor orebatinib was approved by the NMPA in November 2021 for the treatment of resistance to any tyrosine kinase inhibitor (TKI) using a well-validated assay Methods Adult patients diagnosed with chronic myeloid leukemia (CML) with T315I mutation in chronic phase (CP) or accelerated phase (AP)
.
At present, orebatinib has obtained 2 orphan drug designations in the United States for the treatment of chronic myeloid leukemia and for the treatment of acute myeloid leukemia
.
The first-generation BCR-ABL inhibitor Gleevec (imatinib) is an important reference for the commercial value of orabatinib.
According to the data from Minet.
com, in 2020, China's urban public hospitals, county-level public hospitals, urban community centers and The sales of terminal imatinib in township health centers (referred to as Chinese public medical institutions) exceeded 2 billion yuan
.
CSPC's mitoxantrone liposome was approved in China in January 2022 for the treatment of relapsed or refractory peripheral T-cell lymphoma (PTCL)
.
In the United States, mitoxantrone liposome has also received orphan drug designation for the treatment of peripheral T-cell lymphoma
.
In addition, CSPC's Butylphthalide Soft Capsule was launched in China in 2005.
In 2020, the sales of Butylphthalide in China's public medical institutions exceeded 6 billion yuan.
With the qualification of orphan drug, CSPC will promote this Chinese Class 1 new drug to the international market
.
BeiGene's Tislelizumab and Zanubrutinib are blockbuster drugs that have been approved in China
.
Tislelizumab is the sixth PD-1 drug to be marketed in China.
At present, tislelizumab has obtained 3 orphan drug qualifications for esophageal cancer, hepatocellular carcinoma, and gastric cancer (including esophagogastric junction cancer)
.
Zanubrutinib is also the first domestic anticancer drug approved for marketing in the United States, making history
.
On February 23, 2022, the marketing application for the new indication of zanubrutinib was accepted by the US FDA
.
At present, zanubrutinib has obtained 6 orphan drug qualifications, which is the drug with the most orphan drug qualifications by domestic pharmaceutical companies
.