FDA grants orphan drug designation to HQP1351 for chronic myeloid leukemia
The FDA granted orphan drug designation to HQP1351 for treatment of patients with chronic myeloid leukemia, according to the agent’s manufacturer.
HQP1351 (Ascentage Pharma) is a novel, oral BCR-ABL tyrosine kinase inhibitor. The agent — in development for treatment of patients with CML whose disease is resistant to first- and second-generation TKIs — is designed to target BCR-ABL mutants, including T315I.
BCR-ABL kinase mutations are a key mechanism of acquired drug resistance in CML, and T315I — the most common drug-resistant mutation — occurs in approximately one-quarter of patients with drug-resistant disease.
A phase 2 study of HQP1351 as treatment for CML is underway in China, and Ascentage Pharma officials intend to submit a new drug application for the agent this year.
“There is significant unmet clinical need in the treatment of CML globally. This orphan drug designation from FDA marks a major milestone for HQP1351, which will bring about the incentives and support that will enable us to further accelerate the global development and commercialization of this drug candidate,” Dajun Yang, MD, PhD, chairman and CEO of Ascentage Pharma, said in a company-issued press release. “Given the favorable safety and efficacy data obtained thus far, we will expedite the development and are hopeful that HQP1351 will soon benefit patients worldwide.”
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.