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According to the definition of the World Health Organization (WHO), a rare disease is a disease in which the number of patients accounts for 0.
65‰-1‰ of the total population
.
At present, there are more than 7,000 rare diseases confirmed in the world, and about 80% are caused by genetic defects
.
However, it is worth noting that due to the small number of patients, low market demand, and high R&D costs for drugs for rare diseases, there are few pharmaceutical companies paying attention and R&D; therefore, there are still huge unmet needs, as well as barriers to approval and payment
.
However, with the advancement of technology and the huge market prospects, more and more investors and pharmaceutical companies have begun to deploy in the rare disease field in recent years, especially some large foreign pharmaceutical companies
.
In September of this year, AstraZeneca announced the formal establishment of its China Rare Diseases Business Unit.
In July, it had just completed an astronomical acquisition of a rare disease drug company, with an acquisition price of US$39 billion
.
In addition to AstraZeneca, there are also many major global pharmaceutical companies such as Takeda, Roche, Sanofi, etc.
that are also rushing to enter the rare disease drug field
.
Specifically, in December 2020, AstraZeneca acquired rare disease giant Alexion for US$39 billion and officially entered the rare disease market
.
Prior to this, in February 2019, Roche also acquired gene therapy Spark Therapeutics for US$4.
3 billion
.
Earlier, in April 2018, Takeda Pharmaceuticals spent US$65 billion to acquire the rare disease giant Shire, which became an ultra-large M&A case in the pharmaceutical field of the year
.
In addition to large foreign pharmaceutical companies that continue to increase their deployment in the field of rare diseases, in recent years, many domestic pharmaceutical companies have also increased their deployment in this field, and some rare disease treatment companies have gradually been favored by more and more investors
.
For example, on February 26th, Jinlan Gene, a gene therapy new drug research and development biotechnology company, announced that it has completed an angel round of financing of nearly 100 million yuan.
The SME Development Fund, Yuansheng Venture Capital, Saiying Capital, and Xie Nuo Chentu Fund managed by Oriental Fuhai The joint investment is completed
.
This round of financing is mainly used to promote the first three gene therapy projects in the company's product pipeline to enter the IND stage
.
It is reported that Jinlan Gene focuses on the treatment of rare diseases and has a rich pipeline of rare diseases
.
On the whole, in recent years, problems such as the difficulty in diagnosis of rare diseases, the high price of medicines and the lack of available medicines have attracted more and more attention in the industry
.
Especially in China, in order to promote the development of this industry, the state has issued a series of policy documents
.
For example, rare disease catalogues, rare disease diagnosis and treatment guidelines, etc.
These documents have accelerated the approval of rare disease drugs into the domestic market and benefited patients, while also continuously encouraging pharmaceutical companies to actively invest in the research and development of rare disease drugs
.
Among them, it is worth mentioning that on September 15 this year, the National Medical Security Administration’s website released the "National Medical Security Administration on the Fourth Session of the 13th CPPCC National Committee No.
4468 (Social Management Category No.
371) Proposal Reply The letter clearly mentioned that it will further explore the procurement mechanism suitable for "orphan drugs", and will combine the needs of clinical treatment medications, medical insurance financing capabilities and other factors to include eligible rare disease drugs into the scope of medical insurance payment according to procedures
.
In this regard, the analysis believes that the emergence of more diversified payment methods and supporting policies may make rare disease drugs popular among pharmaceutical companies, and even become a development strategy for R&D pipelines
.
In general, as China's economic and social development enters a new stage, the establishment of a new drug review and approval system and the need for innovative development of the pharmaceutical industry, the unmet clinical needs of patients with rare diseases have received more and more attention
.
In this context, companies that have entered the market need to strengthen basic research on rare diseases, and at the same time, combine the characteristics of rare diseases and use scientific tools to innovate research and design to improve research and development efficiency, so as to provide more accessible treatments for patients with rare diseases.
.
65‰-1‰ of the total population
.
At present, there are more than 7,000 rare diseases confirmed in the world, and about 80% are caused by genetic defects
.
However, it is worth noting that due to the small number of patients, low market demand, and high R&D costs for drugs for rare diseases, there are few pharmaceutical companies paying attention and R&D; therefore, there are still huge unmet needs, as well as barriers to approval and payment
.
However, with the advancement of technology and the huge market prospects, more and more investors and pharmaceutical companies have begun to deploy in the rare disease field in recent years, especially some large foreign pharmaceutical companies
.
In September of this year, AstraZeneca announced the formal establishment of its China Rare Diseases Business Unit.
In July, it had just completed an astronomical acquisition of a rare disease drug company, with an acquisition price of US$39 billion
.
In addition to AstraZeneca, there are also many major global pharmaceutical companies such as Takeda, Roche, Sanofi, etc.
that are also rushing to enter the rare disease drug field
.
Specifically, in December 2020, AstraZeneca acquired rare disease giant Alexion for US$39 billion and officially entered the rare disease market
.
Prior to this, in February 2019, Roche also acquired gene therapy Spark Therapeutics for US$4.
3 billion
.
Earlier, in April 2018, Takeda Pharmaceuticals spent US$65 billion to acquire the rare disease giant Shire, which became an ultra-large M&A case in the pharmaceutical field of the year
.
In addition to large foreign pharmaceutical companies that continue to increase their deployment in the field of rare diseases, in recent years, many domestic pharmaceutical companies have also increased their deployment in this field, and some rare disease treatment companies have gradually been favored by more and more investors
.
For example, on February 26th, Jinlan Gene, a gene therapy new drug research and development biotechnology company, announced that it has completed an angel round of financing of nearly 100 million yuan.
The SME Development Fund, Yuansheng Venture Capital, Saiying Capital, and Xie Nuo Chentu Fund managed by Oriental Fuhai The joint investment is completed
.
This round of financing is mainly used to promote the first three gene therapy projects in the company's product pipeline to enter the IND stage
.
It is reported that Jinlan Gene focuses on the treatment of rare diseases and has a rich pipeline of rare diseases
.
On the whole, in recent years, problems such as the difficulty in diagnosis of rare diseases, the high price of medicines and the lack of available medicines have attracted more and more attention in the industry
.
Especially in China, in order to promote the development of this industry, the state has issued a series of policy documents
.
For example, rare disease catalogues, rare disease diagnosis and treatment guidelines, etc.
These documents have accelerated the approval of rare disease drugs into the domestic market and benefited patients, while also continuously encouraging pharmaceutical companies to actively invest in the research and development of rare disease drugs
.
Among them, it is worth mentioning that on September 15 this year, the National Medical Security Administration’s website released the "National Medical Security Administration on the Fourth Session of the 13th CPPCC National Committee No.
4468 (Social Management Category No.
371) Proposal Reply The letter clearly mentioned that it will further explore the procurement mechanism suitable for "orphan drugs", and will combine the needs of clinical treatment medications, medical insurance financing capabilities and other factors to include eligible rare disease drugs into the scope of medical insurance payment according to procedures
.
In this regard, the analysis believes that the emergence of more diversified payment methods and supporting policies may make rare disease drugs popular among pharmaceutical companies, and even become a development strategy for R&D pipelines
.
In general, as China's economic and social development enters a new stage, the establishment of a new drug review and approval system and the need for innovative development of the pharmaceutical industry, the unmet clinical needs of patients with rare diseases have received more and more attention
.
In this context, companies that have entered the market need to strengthen basic research on rare diseases, and at the same time, combine the characteristics of rare diseases and use scientific tools to innovate research and design to improve research and development efficiency, so as to provide more accessible treatments for patients with rare diseases.
.
