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Orphan drugs are also called rare drugs, which refer to drugs used to prevent, treat, and diagnose rare diseases
.
For a long time, rare diseases have been difficult to diagnose, difficult to buy medicines, and expensive to use.
In recent years, with the advancement of the reform of the drug review and approval system, a large number of rare disease drugs have entered the "green channel" and accelerated their listing in China.
Patients with rare diseases in China bring good news
.
But at present, a large part of the more than 20 million rare disease patients in China are still unable to afford or have no medicines available.
Therefore, there is still a large unmet demand in the domestic rare disease drug market, and the market space is vast
.
The industry predicts that in the context of the successive release and implementation of relevant supporting policy documents, the rare disease drug market in the Mainland is expected to increase to US$25.
9 billion in 2030
.
Facing the huge blue ocean of the market, coupled with policies encouraging pharmaceutical companies to develop rare disease drugs, for example, on October 11 this year, the "Technical Guidelines for the Clinical Research and Development of Rare Disease Drugs (Draft for Comment)" was released to improve the clinical research and development of rare diseases.
Efficiency: On November 18, the "Guiding Principles for Clinical Research of Rare Disease Drugs (Draft for Comment)" was released, which also encourages pharmaceutical companies to develop rare disease treatment drugs to improve the efficiency and quality of clinical research and development
.
In this context, many pharmaceutical companies are deploying on the rare disease drug track
.
Currently, listed companies such as Junshi Biologics and other innovative drugs are actively deploying drugs for rare diseases
.
Among them, Junshi Bio's Teriplizumab has successively obtained multiple orphan drug qualifications.
On November 16 this year, Junshi Bio issued an announcement stating that Teriplizumab has obtained FDA orphan drug qualifications for the treatment of esophageal cancer.
.
This is also the fourth FDA orphan drug designation obtained by teriprizumab.
The indications previously obtained orphan drug designation are mucosal melanoma, nasopharyngeal carcinoma and soft tissue sarcoma
.
In July this year, Junshi Bio also announced a cooperation with Jiachen Xihai.
The two parties will jointly establish a joint venture company to jointly develop and commercialize new drug projects in the field of rare diseases and other diseases based on the mRNA technology platform and other technology platforms on a global scale.
.
It is understood that Jiachen Xihai’s pipeline covers tumor immunotherapy, infectious disease vaccines, rare diseases, etc.
, among which tumor immunity and infectious disease vaccine projects are rapidly entering the clinical trial stage
.
Ascent Pharmaceuticals announced on the evening of November 22 that the European Commission (EC) recently granted its Class 1 new drug Oribatinib orphan drug designation for the treatment of chronic myeloid leukemia (CML)
.
This is also the second orphan drug certification for the drug under research after it was certified by the US FDA as an orphan drug
.
According to employment statistics, as of now, Yasheng Pharmaceutical has obtained 9 FDA orphan drug certifications for 4 new drugs under research
.
For another example, Beihai Kangcheng, which was listed on the Hong Kong stock market not long ago, has attracted much attention from the capital market.
It is reported that the company has a rich R&D pipeline.
Through independent research and development and cooperation, the company has a comprehensive differentiation covering 13 drug assets with huge market potential.
Products, of which 3 products have been successfully marketed, 4 are in the clinical stage, 1 is in the IND preparation stage, 2 are in the preclinical stage, and 3 are gene therapy products in the lead identification stage
.
In addition, pharmaceutical companies such as Kangdini Pharmaceuticals, Deyi Sunshine, Shufang Pharmaceuticals, Langyu Group, and Faith Pharmaceuticals are also actively deploying drugs for rare diseases, but most companies are in a relatively early stage of financing
.
It is foreseeable that, as many pharmaceutical companies accelerate their deployment on the rare disease drug track, superimpose the support of a series of powerful policies such as speeding up the review of new drugs, there will be more new rare disease drugs on the market in the future, further satisfying the 20 million rare disease patients Medication needs
.
With the advancement of medical reforms such as medical insurance negotiations and centralized procurement, the cost of drugs for rare diseases will also be further reduced, reducing the burden on the families of related patients
.
.
For a long time, rare diseases have been difficult to diagnose, difficult to buy medicines, and expensive to use.
In recent years, with the advancement of the reform of the drug review and approval system, a large number of rare disease drugs have entered the "green channel" and accelerated their listing in China.
Patients with rare diseases in China bring good news
.
But at present, a large part of the more than 20 million rare disease patients in China are still unable to afford or have no medicines available.
Therefore, there is still a large unmet demand in the domestic rare disease drug market, and the market space is vast
.
The industry predicts that in the context of the successive release and implementation of relevant supporting policy documents, the rare disease drug market in the Mainland is expected to increase to US$25.
9 billion in 2030
.
Facing the huge blue ocean of the market, coupled with policies encouraging pharmaceutical companies to develop rare disease drugs, for example, on October 11 this year, the "Technical Guidelines for the Clinical Research and Development of Rare Disease Drugs (Draft for Comment)" was released to improve the clinical research and development of rare diseases.
Efficiency: On November 18, the "Guiding Principles for Clinical Research of Rare Disease Drugs (Draft for Comment)" was released, which also encourages pharmaceutical companies to develop rare disease treatment drugs to improve the efficiency and quality of clinical research and development
.
In this context, many pharmaceutical companies are deploying on the rare disease drug track
.
Currently, listed companies such as Junshi Biologics and other innovative drugs are actively deploying drugs for rare diseases
.
Among them, Junshi Bio's Teriplizumab has successively obtained multiple orphan drug qualifications.
On November 16 this year, Junshi Bio issued an announcement stating that Teriplizumab has obtained FDA orphan drug qualifications for the treatment of esophageal cancer.
.
This is also the fourth FDA orphan drug designation obtained by teriprizumab.
The indications previously obtained orphan drug designation are mucosal melanoma, nasopharyngeal carcinoma and soft tissue sarcoma
.
In July this year, Junshi Bio also announced a cooperation with Jiachen Xihai.
The two parties will jointly establish a joint venture company to jointly develop and commercialize new drug projects in the field of rare diseases and other diseases based on the mRNA technology platform and other technology platforms on a global scale.
.
It is understood that Jiachen Xihai’s pipeline covers tumor immunotherapy, infectious disease vaccines, rare diseases, etc.
, among which tumor immunity and infectious disease vaccine projects are rapidly entering the clinical trial stage
.
Ascent Pharmaceuticals announced on the evening of November 22 that the European Commission (EC) recently granted its Class 1 new drug Oribatinib orphan drug designation for the treatment of chronic myeloid leukemia (CML)
.
This is also the second orphan drug certification for the drug under research after it was certified by the US FDA as an orphan drug
.
According to employment statistics, as of now, Yasheng Pharmaceutical has obtained 9 FDA orphan drug certifications for 4 new drugs under research
.
For another example, Beihai Kangcheng, which was listed on the Hong Kong stock market not long ago, has attracted much attention from the capital market.
It is reported that the company has a rich R&D pipeline.
Through independent research and development and cooperation, the company has a comprehensive differentiation covering 13 drug assets with huge market potential.
Products, of which 3 products have been successfully marketed, 4 are in the clinical stage, 1 is in the IND preparation stage, 2 are in the preclinical stage, and 3 are gene therapy products in the lead identification stage
.
In addition, pharmaceutical companies such as Kangdini Pharmaceuticals, Deyi Sunshine, Shufang Pharmaceuticals, Langyu Group, and Faith Pharmaceuticals are also actively deploying drugs for rare diseases, but most companies are in a relatively early stage of financing
.
It is foreseeable that, as many pharmaceutical companies accelerate their deployment on the rare disease drug track, superimpose the support of a series of powerful policies such as speeding up the review of new drugs, there will be more new rare disease drugs on the market in the future, further satisfying the 20 million rare disease patients Medication needs
.
With the advancement of medical reforms such as medical insurance negotiations and centralized procurement, the cost of drugs for rare diseases will also be further reduced, reducing the burden on the families of related patients
.
