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Olverembatinib from Ascentage Pharma Granted Second Breakthrough Therapy Designation in China for SDH-Deficient GIST Treatment

Wednesday, May 31, 2023

Ascentage Pharma, a global biopharmaceutical company specializing in developing innovative therapies for cancer and other diseases, has announced that the Center for Drug Evaluation (CDE) of China's National Medical Products Administration (NMPA) has recommended their lead drug candidate, olverembatinib (HQP1351), for a Breakthrough Therapy Designation (BTD) in the treatment of succinate dehydrogenase (SDH)-deficient gastrointestinal stromal tumor (GIST) patients who have received first-line treatment.

This designation marks the second BTD granted to olverembatinib by the CDE. The first was received in March 2021 for the treatment of patients with chronic-phase chronic myeloid leukemia (CML-CP) who are resistant and/or intolerant to first- and second-generation tyrosine kinase inhibitors (TKIs). An application for this indication was accepted by the NMPA in July 2022 and subsequently granted a BTD, supporting olverembatinib's full approval. Additionally, olverembatinib has been awarded two Priority Review Designations, highlighting its potential as a promising therapeutic option.

Breakthrough Therapy Designations are typically granted to innovative drugs or modified novel drugs intended to treat severe, life-threatening diseases or conditions where no existing treatment options are available or where significant advantages over current treatments exist. These designations prioritize communication, guidance, and expedited drug development and review processes by the CDE. BTD-designated drugs may also be eligible for Priority Review status and conditional approvals upon submission of a New Drug Application (NDA), accelerating their availability to patients in need.

SDH-deficient GIST is a rare subtype of gastrointestinal stromal tumor that currently lacks effective treatment options. While the introduction of TKIs has improved outcomes for most GIST patients, those with SDH-deficient GIST often do not respond to existing TKIs. Surgical treatment can benefit early-stage localized disease, but relapse is common. Currently, there is no standard of care for patients with relapsed or advanced SDH-deficient GIST, with a 5-year event-free survival rate of only 24%.

Olverembatinib, a third-generation TKI, is jointly commercialized by Ascentage Pharma and Innovent Biologics in China. It is the first and only third-generation BCR-ABL inhibitor approved in China for the treatment of TKI-resistant chronic-phase chronic myeloid leukemia (CML-CP) or accelerated-phase CML (CML-AP) with the T315I mutation. Preclinical models and early clinical studies have demonstrated olverembatinib's potent antitumor activity against GIST, particularly in patients with SDH-deficient GIST. Ongoing Phase Ib/II studies have shown an impressive clinical benefit rate of 93.8% in this patient population. These promising results have led to presentations at the American Society of Clinical Oncology (ASCO) Annual Meeting for two consecutive years. It is important to note that olverembatinib is still an investigational drug and has not received approval for any indication in the United States.

Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma, expressed gratitude for the recognition of olverembatinib's clinical potential and described the BTD recommendation as a significant milestone in the drug's nonhematologic clinical development. Ascentage Pharma aims to maintain close communication with the CDE to expedite the clinical development program in China, ensuring that this novel therapeutic can be made available to patients as soon as possible.


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