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[Pharmaceutical Network Market Analysis] Statistics show that the prevalence of rare diseases in the population is about 3.
5%-5.
9%.
Although the prevalence of 84.
5% of the diseases is less than one millionth, the total number of rare disease patients in China has There are about 20 million people and it is growing at a rate of more than 200,000 new patients every year
.
Driven by the continuous expansion of clinical drug demand, the rare disease drug market is continuing to heat up
.
Some industry organizations pointed out that China's rare disease drug market accounted for 0.
4% and 1% of the global rare disease market in 2016 and 2020, respectively.
43.
8 billion yuan)
.
Facing the growing demand for medicines, in recent years, the state is continuously introducing policies to encourage the research and development and production of drugs for rare diseases
.
For example, in 2022, CDE has successively issued two guidelines related to the clinical practice of rare disease drugs, the "Technical Guidelines for Clinical Research and Development of Rare Disease Drugs" and the "Statistical Guidelines for Clinical Research of Rare Disease Drugs (Trial)", which are the clinical trial plans for rare disease drugs.
The formulation of it provides new ideas, and also forms an important boost from the technical aspect
.
It is understood that in July, the highly selective CSF-1R inhibitor ABSK021 independently developed by Heyu Pharma was recognized as a breakthrough therapy drug by the Center for Drug Evaluation (CDE) of the State Food and Drug Administration of China for the treatment of inoperable tenosynovial giant cells.
This will bring new treatment options for domestic patients with giant cell tumor of the tendon sheath
.
The so-called breakthrough therapy certification is one of the four accelerated procedures for the review of innovative drugs by CDE, which refers to innovative or improved new drugs in the clinical stage, which are used for the prevention and treatment of serious life-threatening diseases or seriously affect the quality of life.
Diseases for which there is no effective prevention and treatment method or sufficient evidence to show that innovative drugs are superior to existing treatment methods
.
In addition to encouraging local companies to develop and innovate, the country is also vigorously promoting the accelerated entry of foreign rare disease drugs into the domestic market
.
Previously, the State Food and Drug Administration and the National Health and Medical Commission jointly issued the "Announcement on Matters Concerning the Review and Approval of Overseas New Drugs Urgently Needed in Clinical Practice", which established a special channel for review and approval of overseas new drugs urgently needed for clinical treatment such as rare disease treatment drugs
.
Recently, the State Drug Administration of China approved the application for changing the origin of Eculizumab injection (trade name: Sulirui®)
.
As a prescription drug approved in China for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) in adults and children, this change in approval marks the first time eculizumab has been approved as a prescription drug in China.
AstraZeneca is the first rare disease drug in the Chinese market, a key step towards filling the gap in this field in China, providing a new treatment option for Chinese patients in related rare disease fields
.
The industry believes that the importance of the rare disease drug track will continue to increase judging from the current national and relevant departments' concerns about children and rare disease patients
.
Facing the huge market potential, more and more domestic and foreign pharmaceutical companies will begin to accelerate their deployment
.
Disclaimer: Under no circumstances does the information or opinions expressed in this article constitute investment advice to anyone
.
5%-5.
9%.
Although the prevalence of 84.
5% of the diseases is less than one millionth, the total number of rare disease patients in China has There are about 20 million people and it is growing at a rate of more than 200,000 new patients every year
.
Driven by the continuous expansion of clinical drug demand, the rare disease drug market is continuing to heat up
.
Some industry organizations pointed out that China's rare disease drug market accounted for 0.
4% and 1% of the global rare disease market in 2016 and 2020, respectively.
43.
8 billion yuan)
.
Facing the growing demand for medicines, in recent years, the state is continuously introducing policies to encourage the research and development and production of drugs for rare diseases
.
For example, in 2022, CDE has successively issued two guidelines related to the clinical practice of rare disease drugs, the "Technical Guidelines for Clinical Research and Development of Rare Disease Drugs" and the "Statistical Guidelines for Clinical Research of Rare Disease Drugs (Trial)", which are the clinical trial plans for rare disease drugs.
The formulation of it provides new ideas, and also forms an important boost from the technical aspect
.
It is understood that in July, the highly selective CSF-1R inhibitor ABSK021 independently developed by Heyu Pharma was recognized as a breakthrough therapy drug by the Center for Drug Evaluation (CDE) of the State Food and Drug Administration of China for the treatment of inoperable tenosynovial giant cells.
This will bring new treatment options for domestic patients with giant cell tumor of the tendon sheath
.
The so-called breakthrough therapy certification is one of the four accelerated procedures for the review of innovative drugs by CDE, which refers to innovative or improved new drugs in the clinical stage, which are used for the prevention and treatment of serious life-threatening diseases or seriously affect the quality of life.
Diseases for which there is no effective prevention and treatment method or sufficient evidence to show that innovative drugs are superior to existing treatment methods
.
In addition to encouraging local companies to develop and innovate, the country is also vigorously promoting the accelerated entry of foreign rare disease drugs into the domestic market
.
Previously, the State Food and Drug Administration and the National Health and Medical Commission jointly issued the "Announcement on Matters Concerning the Review and Approval of Overseas New Drugs Urgently Needed in Clinical Practice", which established a special channel for review and approval of overseas new drugs urgently needed for clinical treatment such as rare disease treatment drugs
.
Recently, the State Drug Administration of China approved the application for changing the origin of Eculizumab injection (trade name: Sulirui®)
.
As a prescription drug approved in China for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) in adults and children, this change in approval marks the first time eculizumab has been approved as a prescription drug in China.
AstraZeneca is the first rare disease drug in the Chinese market, a key step towards filling the gap in this field in China, providing a new treatment option for Chinese patients in related rare disease fields
.
The industry believes that the importance of the rare disease drug track will continue to increase judging from the current national and relevant departments' concerns about children and rare disease patients
.
Facing the huge market potential, more and more domestic and foreign pharmaceutical companies will begin to accelerate their deployment
.
Disclaimer: Under no circumstances does the information or opinions expressed in this article constitute investment advice to anyone
.
