Yasheng pharmaceutical won the 9th FDA orphan drug qualification, the highest in China's Biopharmaceutical Enterprises

On January 5, 2021, AXA announced that the US Food and Drug Administration (FDA) recently granted the company the qualification of the orphan drug apg-2575, an original Bcl-2 inhibitor, for the treatment of acute myeloid leukemia (AML). This is apg-2575's fourth orphan drug qualification granted by FDA after the indications of Fahrenheit macroglobulinemia (WM), chronic lymphoblastic leukemia (CLL) and multiple myeloma (mm). Up to now, four new drugs under development of AXA Pharmaceutical have obtained 9 FDA orphan drug certifications, which is the highest among Chinese enterprises.

AML is a kind of hematological malignancy with high heterogeneity, and it is mainly a disease of elderly patients. The median age of diagnosis is 68 years old. The latest SEER (Surveillance Epidemiology and end results program) estimated that there will be 19940 newly diagnosed AML cases and 11180 deaths in the United States in 2020. Although some progress has been made in the treatment of AML in recent years, the 5-year survival rate of the disease is 25% - 30%, so there is an urgent need for safer, more lasting and effective treatment.

Orphan drugs, also known as rare drugs, refer to drugs used to prevent, treat and diagnose rare diseases. In the United States, rare diseases are defined as diseases that affect less than 200000 people. Since 1983, the orphan drug act has been implemented in the United States to provide policy support to enterprises to encourage the research and development of drugs for rare diseases. The orphan drug qualification granted by FDA will help the drug to enjoy certain policy support in the follow-up R & D and commercialization in the United States, including tax exemption of clinical trial fees, exemption of NDA application fees, and access to R & D funding. In particular, the drug can obtain 7-year exclusive rights in the U.S. market after its indication is approved for marketing.

With the rise of China's pharmaceutical innovation power, the trend of Chinese pharmaceutical enterprises to "go to sea" in recent years has become increasingly obvious, and obtaining FDA Orphan Drug Certification and other qualifications has also been regarded as one of the important indicators of global innovation strength. As a leader in global innovation, AXA pharmaceutical is undoubtedly one of the best. Up to now, there are 4 new drugs in research and development of Yasheng pharmaceutical, which have obtained 9 FDA orphan drug certifications (see the table below). According to public information, this number is the most leading among Chinese enterprises.

Public data on the number of innovative drugs approved by FDA show that the proportion of orphan drugs approved by FDA in the approved innovative therapies has gradually increased in the past 10 years. In 2020, more than 60% of the new drugs approved will be qualified as orphan drugs, a new high in 10 years. This data shows that FDA's orphan drug qualification policy has a very practical incentive and promotion significance for accelerating the R & D and marketing of rare disease drugs.