The US Food and Drug Administration (FDA) assigned an orphan status to a drug for the treatment of myeloid leukemia, which is being developed by Fusion Pharma, a resident of Skolkovo Foundation’s biomed cluster. This means that the regulator recognized the uniqueness of the drug and importance of its trials, which guarantees increased attention and assistance in the process of further development from FDA. This is the first time when a drug fully developed in Russia received such status in the United States.
Fusion Pharma develops innovative medicinal products, including those used for the treatment of resistant forms of chronic myeloid leukemia (CML), including its resistant forms with T315I mutation. Currently, the company is studying PF-114 molecule, a selective third-generation tyrosine kinase inhibitor that suppresses the activity of the chimeric BCR-ABL protein, which is specific for chronic myeloid leukemia (CML).
CML is one of the most common types of leukemia in adults. Each year, there are 1-2 registered cases of this disease per 100 thousand people. A characteristic feature of CML is the presence of the so-called “Philadelphia chromosome” in the leukemia cells, which is formed as a result of a reciprocal chromosomal translocation, a special type of mutation. This leads to the formation of BCR-ABL, a chimeric oncogene which plays a key role in the uncontrolled division of cells and development of the disease. The product of this defective gene is the target for the advanced targeted therapy, which allowed to significantly improve patients survival.
Currently, Fusion Pharma conducts clinical study of PF-114 Phase 1 aiming to assess safety and efficacy of the drug in patients who do not respond to therapies, and to find the optimal tolerated dose of the drug for further trials. The clinical trials started in August 2016 on the premises of the Hematology Research Center of the Russian Ministry of Health in Moscow and, since the beginning of 2017, V.A. Almazov Medical Research Center of the Russian Ministry of Health in St. Petersburg joined the research.