Pharma Focus Europe

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

Wednesday, May 31, 2023

Ascentage Pharma (6855.HK), a global biopharmaceutical company focused on developing innovative therapies for cancer, chronic hepatitis B (CHB), and age-related diseases, has recently announced that the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA) has recommended their lead drug candidate, olverembatinib (HQP1351), for Breakthrough Therapy Designation (BTD). The BTD is intended for the treatment of patients with succinate dehydrogenase (SDH)-deficient gastrointestinal stromal tumor (GIST) who have received first-line treatment.

This marks the second BTD granted to olverembatinib by the CDE. The first BTD was granted in March 2021 for the treatment of patients with chronic-phase chronic myeloid leukemia (CML-CP) who are resistant 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 olverembatinib received a BTD to support full approval.

Breakthrough Therapy Designations are typically granted to innovative drugs or modified novel drugs that target serious, life-threatening diseases or conditions for which there is no existing treatment or demonstrate advantages over current treatment options. Drugs with BTDs receive priority in communication, guidance, and development progress from regulatory authorities. They are also eligible for Priority Review status and conditional approvals upon submission of a New Drug Application (NDA). BTDs help expedite the development and review process of drugs with significant clinical value or that address urgent unmet medical needs.

GIST is a type of malignancy that arises in the gastrointestinal tract's mesenchymal tissues, with most patients having KIT or PDGFRA mutations. While tyrosine kinase inhibitors (TKIs) have significantly improved the prognosis for GIST patients, those with SDH-deficient GIST, which is a rare subtype, still face significant unmet medical needs. Existing TKIs are generally ineffective for SDH-deficient GIST, and there is currently no standard of care for relapsed or advanced SDH-deficient GIST patients. Olverembatinib, a third-generation TKI jointly commercialized by Ascentage Pharma and Innovent Biologics in China, has shown promising antitumor activity against GIST in preclinical models and early clinical studies. In a Phase Ib/II study in China, olverembatinib demonstrated an impressive clinical benefit rate (CBR) of 93.8% in patients with SDH-deficient GIST.

Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma, expressed appreciation for the regulatory authority's recognition of olverembatinib's clinical potential. The BTD for olverembatinib in SDH-deficient GIST represents a significant milestone in its clinical development for nonhematologic indications. Ascentage Pharma intends to maintain close contact with the CDE to expedite the clinical development program in China, with the aim of providing this novel therapeutic option to patients as quickly as possible.

Thermo Fisher Scientific - mRNA ServicesWorld Orphan Drug Congress 2024World Vaccine Congress Europe 2024Advanced Therapies USA 2024