FDA grants Axa Pharma's third-generation TKI inhibitor HQP1351 orphan drug for the treatment of chronic granulocytic leukemia

Ascentage Pharma has announced that the U.SFood and Drug Administration (FDA) has awarded it the HQP1351 Orphan Drug (ODD) for the treatment of chronic granulocytic leukemia (CML)This is the first ODD obtained by Ascentage PharmaCML is a malignant tumor of the blood system with an annual incidence of about 1.9 cases / 100,000BCR-ABL tyrosine kinase inhibitors (TKI) have significantly improved the treatment of CMLHowever, many patients develop edgy resistance after the first generation of BCR-ABL TKI's Imartininib (Grevi) and some second-generation TKI treatmentsThis acquired TKIs resistance is a major challenge in the treatment of CMLBCR-ABL kinase mutation is a key mechanism for acquired drug resistance, of which T315I is the most common drug-resistant mutation, with about 25% of drug-resistant CML patients occurring T315IPatients with T315I mutations are resistant to both first- and second-generation BCR-ABL inhibitors, so the next generation of BCR-ABL inhibitors is urgently needed for more effective treatmentHQP1351 is a new type of oral third-generation BCR-ABL inhibitor that effectively targets BCR-ABL mutants, including T315I, and is being developed to treat CML patients resistant to first- and second-generation TKIIn July 2019, HQP1351 was approved by the FDA for Phase Ib research, and an important Phase II study is currently under way in ChinaDrDajun Yang, Chairman and CEO of Ascentage Pharma, said: 'There is a large unmet clinical demand for CML treatment worldwideODD from the FDA marks an important milestone for HQP1351, which will inspire and support us to further accelerate the global development and commercialization of this drug candidate'