Ascentage Pharma, a biopharmaceutical company focused on developing novel therapies for cancer and other diseases, has announced that the China National Medical Products Administration's Center for Drug Evaluation (CDE) has recommended their lead drug candidate, olverembatinib (HQP1351), for Breakthrough Therapy Designation (BTD). The BTD recommendation is for the treatment of patients with succinate dehydrogenase (SDH)-deficient gastrointestinal stromal tumor (GIST) who have received initial treatment.
This is the second BTD granted to olverembatinib by the CDE. The first BTD 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). The New Drug Application (NDA) for this indication was accepted by the NMPA in July 2022 and received a BTD, which will support the full approval process for olverembatinib. The drug has also been granted two Priority Review Designations, highlighting its potential therapeutic value.
Breakthrough Therapy Designations are typically granted to innovative drugs or modified novel drugs targeting life-threatening diseases or conditions where there are no existing treatments or significant advantages over current options. These designations prioritize communication, guidance, and development progress for the drugs. BTD-designated drugs may also be eligible for Priority Review status and conditional approvals upon NDA submission, expediting the development and review process for drugs addressing critical medical needs.
Olverembatinib has shown promise in the treatment of SDH-deficient GIST, a rare subtype of gastrointestinal stromal tumor. While most GIST patients benefit from tyrosine kinase inhibitors, those with SDH-deficient GIST often do not respond to existing treatments. Surgical treatment is effective for early-stage localized disease, but relapse is common. Currently, there is no standard of care for relapsed or advanced SDH-deficient GIST patients, highlighting the unmet medical need.
Olverembatinib, a third-generation tyrosine kinase inhibitor, is jointly commercialized by Ascentage Pharma and Innovent Biologics in China. It has already gained approval 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. In preclinical and early clinical studies, olverembatinib has demonstrated strong anti-tumor activity against GIST, including promising efficacy in SDH-deficient GIST patients. Ongoing Phase Ib/II studies in China have reported a high clinical benefit rate in this patient subgroup. Olverembatinib has not yet received approval for any indication in the United States.
The BTD recommendation for olverembatinib in SDH-deficient GIST represents a significant milestone in its clinical development for non-hematologic indications. Ascentage Pharma aims to maintain close communication with the CDE to accelerate the drug's clinical development program in China, bringing the potential benefits of this novel therapy to patients as soon as possible.
