According to a story from BioPortfolio, the biopharmaceutical company CStone Pharmaceuticals has recently announced that China’s National Medical Product Administration (NMPA) has issued approval for the company to conduct a phase 1 bridging study of the company’s drug ivosidenib, which is currently approved in the US as a treatment for acute myeloid leukemia presenting with an IDH1 mutation. The study will evaluate the drug in Chinese patients with the same indication.
About Acute Myeloid Leukemia (AML)
Acute myeloid leukemia, also known as acute myelogenous leukemia, is a type of blood cancer which affects myeloblasts, stem cells that would normally develop into myeloid white blood cells. There are a number of risk factors for this cancer, including other blood disorders such as myelodysplastic syndrome, family history, certain genetic variants, chemical exposure (including chemotherapy agents), and radiation. Symptoms include an increased risk of infection, easy bruising and bleeding, fatigue, shortness of breath, fever, weight and appetite loss, anemia, and bone/joint pain. Treatment for this cancer is most often chemotherapy or stem cell transplant; there are very limited options for patients with relapsed disease. The five year survival rate for acute myeloid leukemia is only 27 percent in the US. There is a clear need for more effective treatments for this cancer. To learn more about acute myeloid leukemia, click here.
Treating Acute Myeloid Leukemia in China
China has about 30,000 new cases of this blood cancer within its population every year. In addition, the five year survival rate for patients is lower than in the US at around 20 percent. With this deficiency in mind, it is clear that the introduction of new treatment options like ivosidenib could help bridge the gap and improve survival. The IDH1 mutation that ivosidenib acts on is present in anywhere from six to ten percent of cases. The approval for the trial from the NMPA is a significant milestone for Cstone, with ivosidenib being the first and only product from the company having obtained public approval anywhere.
The drug more recently earned Breakthrough Therapy designation from the FDA when used in combination with azacitidine for newly diagnosed patients with the relevant mutation.
