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"Orphan drugs" are also known as rare disease drugs, which refer to drugs used to prevent, treat, and diagnose rare diseases
.
Due to the small population of rare diseases, low market demand, and high research and development costs, few pharmaceutical companies pay attention to the research and development of their therapeutic drugs, so these drugs are vividly called "orphan drugs
.
" Although there are few companies concerned, strengthening the research and development of "orphan drugs" cannot be ignored
.
Recently, the Center for Drug Evaluation of the State Food and Drug Administration publicly solicited opinions on the "Guiding Principles for Clinical Research of Rare Disease Drugs (Draft for Comment)" (hereinafter referred to as the Draft for Comment)
.
The draft for comments on the design and analysis of clinical research involving rare disease drugs, precautions in clinical research implementation, evidence evaluation, etc.
, focuses on ensuring the quality of the research and the reliability of the results and improving the efficiency of research and development, and elaborates on key statistical issues
.
Related content also proposes that due to the small overall patient population, clinical research on rare disease drugs may consider appropriately relaxing the entry and exclusion criteria to allow relatively large numbers of patients to enter the study
.
For rare diseases that affect both adults and children, sponsors should consider including pediatric patients in clinical studies as soon as possible
.
It is understood that Yasheng Pharmaceutical is a representative enterprise in the development of "orphan drugs" in China
.
The company is positioned in the clinical development stage of the original innovative drug research and development, focusing on the treatment of tumors, hepatitis B and aging-related diseases, and developing corresponding drugs
.
According to statistics, Yasheng Pharmaceutical has a total of 4 new drugs under development that have obtained a total of 12 "orphan drug" qualifications granted by the U.
S.
Food and Drug Administration (FDA).
It is a pharmaceutical company with a large number of orphan drug pipelines in China
.
On November 22, Yasheng Medicine once again received new news that the European Commission (EC) recently awarded the company a new class 1 drug, Olverembatinib (HQP1351, formerly known as Nectinib and Oribatinib) orphans.
Drug qualification certification for the treatment of chronic myeloid leukemia (CML)
.
This is the first orphan drug certification that Ascent Pharmaceuticals has obtained in the European Union, and it is also the second orphan drug certification of the drug under research after the US FDA orphan drug certification.
.
In addition, the author was informed that Yasheng Pharmaceutical also announced recently that it has reached a clinical cooperation and drug supply agreement with Pfizer.
According to the terms of the agreement, Yasheng Pharmaceutical will be responsible for carrying out the research, and Pfizer will be responsible for providing the drugs needed for the research and joint research and treatment of breast.
New plan for cancer
.
That is to say, the two parties will jointly develop the Bcl-2 selective inhibitor Lisaftoclax (APG-2575) of Ascent Pharmaceuticals combined with Pfizer's CDK4/6 inhibitor Aiboxin (pibecili) for the treatment of recurrent, locally advanced or metastatic disease Treatment of estrogen receptor positive (ER+) and human epidermal growth factor receptor 2 negative (HER2-) breast cancer
.
Drugs used to prevent, treat, and diagnose rare diseases are called "orphan drugs
.
" Although not many pharmaceutical companies pay attention to the field of "orphan drugs", many companies are actively promoting the development of "orphan drugs"
.
For example, in addition to Yasheng Pharmaceuticals, the author has learned that Junshi Biotech has also received new news in the field of "orphan drugs" recently, that the anti-PD-1 monoclonal antibody drug Teriprizumab independently developed by Junshi Biotech has been awarded the FDA orphan drug Qualified for the treatment of esophageal cancer (EC)
.
It is reported that this is the fourth orphan drug qualification that Teriplizumab has obtained.
Previously, this product has been approved by the FDA for the treatment of mucosal melanoma, nasopharyngeal carcinoma and soft tissue sarcoma
.
It is understood that teriprizumab injection is the first domestically produced monoclonal antibody drug targeting PD-1 that has been approved for marketing in China.
The first approved indication is for unresectable use in previous systemic treatment failures.
Or the treatment of metastatic melanoma
.
In December 2020, Teriplimumab successfully passed the national medical insurance negotiations and was included in the new version of the catalog
.
Today, Teriplimumab has carried out more than 30 clinical studies covering more than 15 indications worldwide
.
In addition, Genxi Biotech recently announced that the U.
S.
Food and Drug Administration (FDA) has granted it the BCMA/CD19 dual target CAR-T cell therapy orphan drug designation (ODD) based on the FasTCAR technology platform for the treatment of multiple diseases.
Myeloma
.
.
Due to the small population of rare diseases, low market demand, and high research and development costs, few pharmaceutical companies pay attention to the research and development of their therapeutic drugs, so these drugs are vividly called "orphan drugs
.
" Although there are few companies concerned, strengthening the research and development of "orphan drugs" cannot be ignored
.
Recently, the Center for Drug Evaluation of the State Food and Drug Administration publicly solicited opinions on the "Guiding Principles for Clinical Research of Rare Disease Drugs (Draft for Comment)" (hereinafter referred to as the Draft for Comment)
.
The draft for comments on the design and analysis of clinical research involving rare disease drugs, precautions in clinical research implementation, evidence evaluation, etc.
, focuses on ensuring the quality of the research and the reliability of the results and improving the efficiency of research and development, and elaborates on key statistical issues
.
Related content also proposes that due to the small overall patient population, clinical research on rare disease drugs may consider appropriately relaxing the entry and exclusion criteria to allow relatively large numbers of patients to enter the study
.
For rare diseases that affect both adults and children, sponsors should consider including pediatric patients in clinical studies as soon as possible
.
It is understood that Yasheng Pharmaceutical is a representative enterprise in the development of "orphan drugs" in China
.
The company is positioned in the clinical development stage of the original innovative drug research and development, focusing on the treatment of tumors, hepatitis B and aging-related diseases, and developing corresponding drugs
.
According to statistics, Yasheng Pharmaceutical has a total of 4 new drugs under development that have obtained a total of 12 "orphan drug" qualifications granted by the U.
S.
Food and Drug Administration (FDA).
It is a pharmaceutical company with a large number of orphan drug pipelines in China
.
On November 22, Yasheng Medicine once again received new news that the European Commission (EC) recently awarded the company a new class 1 drug, Olverembatinib (HQP1351, formerly known as Nectinib and Oribatinib) orphans.
Drug qualification certification for the treatment of chronic myeloid leukemia (CML)
.
This is the first orphan drug certification that Ascent Pharmaceuticals has obtained in the European Union, and it is also the second orphan drug certification of the drug under research after the US FDA orphan drug certification.
.
In addition, the author was informed that Yasheng Pharmaceutical also announced recently that it has reached a clinical cooperation and drug supply agreement with Pfizer.
According to the terms of the agreement, Yasheng Pharmaceutical will be responsible for carrying out the research, and Pfizer will be responsible for providing the drugs needed for the research and joint research and treatment of breast.
New plan for cancer
.
That is to say, the two parties will jointly develop the Bcl-2 selective inhibitor Lisaftoclax (APG-2575) of Ascent Pharmaceuticals combined with Pfizer's CDK4/6 inhibitor Aiboxin (pibecili) for the treatment of recurrent, locally advanced or metastatic disease Treatment of estrogen receptor positive (ER+) and human epidermal growth factor receptor 2 negative (HER2-) breast cancer
.
Drugs used to prevent, treat, and diagnose rare diseases are called "orphan drugs
.
" Although not many pharmaceutical companies pay attention to the field of "orphan drugs", many companies are actively promoting the development of "orphan drugs"
.
For example, in addition to Yasheng Pharmaceuticals, the author has learned that Junshi Biotech has also received new news in the field of "orphan drugs" recently, that the anti-PD-1 monoclonal antibody drug Teriprizumab independently developed by Junshi Biotech has been awarded the FDA orphan drug Qualified for the treatment of esophageal cancer (EC)
.
It is reported that this is the fourth orphan drug qualification that Teriplizumab has obtained.
Previously, this product has been approved by the FDA for the treatment of mucosal melanoma, nasopharyngeal carcinoma and soft tissue sarcoma
.
It is understood that teriprizumab injection is the first domestically produced monoclonal antibody drug targeting PD-1 that has been approved for marketing in China.
The first approved indication is for unresectable use in previous systemic treatment failures.
Or the treatment of metastatic melanoma
.
In December 2020, Teriplimumab successfully passed the national medical insurance negotiations and was included in the new version of the catalog
.
Today, Teriplimumab has carried out more than 30 clinical studies covering more than 15 indications worldwide
.
In addition, Genxi Biotech recently announced that the U.
S.
Food and Drug Administration (FDA) has granted it the BCMA/CD19 dual target CAR-T cell therapy orphan drug designation (ODD) based on the FasTCAR technology platform for the treatment of multiple diseases.
Myeloma
.
