Russian leukemia drug recognized in the USA
For the first time, the drug, fully developed in Russia, received orphan status in the USA
The US Food and Drug Administration (FDA) has recognized the uniqueness of the drug PF-114 for the treatment of myeloid leukemia (including its resistant forms with the T315I mutation), developed by the resident of the biomedical technologies cluster of the Skolkovo Foundation, Fusion Pharma, as well as the significance of the ongoing research. The status obtained by the drug guarantees increased attention to it and assistance in the further development process from the FDA.
Kirill Kaem, Acting Senior Vice President for Innovation, Executive Director of the biomedical Technologies cluster of the Skolkovo Foundation, stressed that the development of innovative medicines is a long process and overcoming each stage is a victory for the development team. "I am happy to congratulate Fusion Pharma on the recognition of the importance of the project by the FDA. The development of the Skolkovo resident was the first innovative Russian development to receive such a status in the United States. We have been supporting this project for a long time and wish the company a successful completion of all stages of drug registration," says Kirill Kayem.
The recognition of orphan status by the regulator is one of the key points for the clinical stage of PF–114 development, says Hermes Chilov, PhD, CEO of Fusion Pharma and one of the developers of PF-114. He also stressed the uniqueness of the project: "PF-114 is the first targeted drug developed entirely in Russia, fully in accordance with modern international standards."
Currently, the company is conducting the 1st phase of a clinical study of PF-114 molecule, a selective third-generation tyrosine kinase inhibitor that suppresses the activity of the chimeric protein BCR-ABL, specific for chronic myeloid leukemia (CML). The safety and efficacy of the drug in resistant patients is being studied, and the optimal tolerated dose of the drug is being searched. The study was launched in August 2016 on the basis of the Hematology Research Center of the Ministry of Health of the Russian Federation in Moscow, and since the beginning of 2017, the Almazov Medical Research Center of the Ministry of Health of the Russian Federation in St. Petersburg has joined the study. "In the future, we plan to conduct phase II clinical trials around the world with subsequent registration of the drug in Europe, the USA and Russia," Hermes Chilov shares his plans.
Currently, there is only one drug in the world that is effective in patients with the T315I mutation, which, unfortunately, is not registered in Russia, says Anna Turkina, MD, Professor, Head of the Scientific Advisory Department of Myeloproliferative Diseases of the National Research Center of the Ministry of Health of the Russian Federation, chief researcher of PF-114. According to her, preliminary preclinical studies have convincingly shown that PF-114 is highly effective for blocking the growth of cells with mutations that cause cell resistance to the effects of drugs, and is also effective against the T315I mutation.
"There is reason to hope that the drug PF-114 will demonstrate a low level of toxicity, which will significantly expand the range of its use in patients with CML," emphasizes Professor Turkina
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20.07.2017