Recently, two domestically produced new drugs have been certified as orphan drugs overseas

"Orphan drugs" are also known as rare drugs, which refer to drugs used to prevent, treat, and diagnose rare diseases
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For a long time, due to the small population of rare diseases, low market demand, and high research and development costs, few pharmaceutical companies have paid attention to the research and development of their therapeutic drugs, so these drugs have been vividly called "orphan drugs
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" According to statistics, there are currently about 7,000 known rare diseases in the world, and only 5%-10% of those diseases where medicines are actually available
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Moreover, less than 10% of patients with rare diseases are still facing a situation where medicines are available but it is difficult to afford medical expenses
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However, it is worth noting that although few pharmaceutical companies have paid attention to the field of "orphan drugs" in the past, in recent years, with the advent of the tide of pharmaceutical innovation in China, many local companies have begun to actively promote the development of "orphan drugs"
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For example, in the near future, two more domestically produced new drugs have obtained orphan drug qualifications overseas
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Genxi Bio-GC012F obtained FDA orphan drug certification.
Recently, GRCL.
US announced that the U.
S.
Food and Drug Administration (FDA) has awarded it a CAR-T cell therapy orphan based on the development of the FasTCAR technology platform and the BCMA/CD19 dual target.
Drug Qualification (ODD) for the treatment of multiple myeloma
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It is understood that this new dual-targeting autologous CAR-T therapy product code-named GC012F based on the FasTCAR platform can simultaneously target malignant plasma cell sites expressing BCMA and early progenitor cell sites expressing CD19.
Previous studies have shown that BCMA/CD19 dual-target CAR-T can more effectively eliminate the cells formed by multiple myeloma clones and reduce recurrence
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At present, although the drug has not yet been declared in China, the FIC Intelligence database of Zhongkang shows that 3 international clinical registration numbers indicate that GC012F has carried out a multi-center, phase I clinical trial initiated by the investigator in China, including for multiple bone marrow Clinical trials for new tumors and high-risk patients
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Yasheng Pharmaceutical’s new drug HQP1351 was recognized as an orphan drug by the European Commission.
On November 22, Yasheng Pharmaceutical issued an announcement that the European Commission (EC) recently granted its Class 1 new drug oribatinib (HQP1351) orphan drug qualification for treatment Chronic Myeloid Leukemia (CML)
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It is reported that this is the first orphan drug certification that Yasheng Pharmaceutical has obtained in the European Union, and it is also the second orphan drug certification for the drug under development after the orphan drug certification of the U.
S.
Food and Drug Administration (FDA)
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Data show that CML is a rare hematological malignancy, with an annual incidence rate of 2.
43/10000 in the 27 EU countries
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HQP1351 is an original Class 1 new drug of Ascent Pharmaceuticals, a new third-generation BCR-ABL TKI, which is used to treat CML resistant to the first and second-generation TKIs, especially for CML patients with T315I mutations.
It has appeared in clinical trials.
Good curative effect
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At present, the new drug application (NDA) of this drug in China is in the approval process for the treatment of CML chronic phase (CML-CP) and accelerated phase (CML-AP) patients with T315I mutation
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Conclusion In general, under the influence of China's favorable policies, the research and development of domestic orphan drugs will gradually start to go on track, and more and more domestic pharmaceutical companies will also invest in the field of orphan drugs
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With the emergence of more new drugs, the dilemma that patients with rare diseases can choose from few or no drugs will be changed, benefiting more patients
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