Ascentage Pharma announced today that its novel drug olverembatinib has secured Breakthrough Therapy Designation (BTD) from China's National Medical Products Administration for a new indication. The designation covers the drug's use in combination with low-intensity chemotherapy as first-line treatment for newly diagnosed Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL) patients.
The latest regulatory milestone represents a significant advancement in addressing an urgent therapeutic need in China's hematological cancer landscape. Ph+ ALL, which accounts for 20-30% of adult ALL cases, has historically been associated with poor outcomes, particularly affecting elderly patients who often struggle with treatment tolerance.
Clinical Impact and Unmet Need
Prior to the advent of tyrosine kinase inhibitors (TKIs), patients with Ph+ ALL faced a grim prognosis, with five-year overall survival rates below 20% when treated with chemotherapy alone. While the introduction of TKIs has transformed the treatment paradigm, existing first- and second-generation options have shown limitations, including high relapse rates and short disease-free survival periods.
Notably, China currently lacks any approved TKI for first-line Ph+ ALL treatment, highlighting the significant therapeutic void that olverembatinib aims to address. The incidence rate of ALL in China stands at approximately 0.69 per 100,000 people.
Development Status and Future Direction
A global Phase III registrational study comparing olverembatinib plus chemotherapy against imatinib plus chemotherapy in treatment-naïve Ph+ ALL patients received clearance from Chinese regulators in July 2023. This trial positions olverembatinib to potentially become China's first approved TKI for first-line Ph+ ALL treatment.
"This BTD underscores the therapeutic potential and utility of olverembatinib in Ph+ ALL, a hematologic malignancy with dismal prognosis," stated Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma. "Moving forward, we will strive to accelerate this clinical development program for olverembatinib and bring it to more patients as quickly as possible."
Regulatory Context and Previous Approvals
This marks olverembatinib's third BTD in China, building on previous designations received for:
- Treatment of chronic-phase chronic myeloid leukemia resistant/intolerant to first- and second-generation TKIs (March 2021)
- Treatment of SDH-deficient gastrointestinal stromal tumor after first-line treatment (June 2023)
As China's first approved third-generation BCR-ABL inhibitor, olverembatinib is currently marketed jointly by Ascentage Pharma and Innovent Biologics. Its approved indications, which include treatment for specific mutations in chronic myeloid leukemia, are covered under China's National Reimbursement Drug List.
The BTD pathway in China prioritizes promising therapies for serious conditions, potentially accelerating their development and review process. Drugs receiving this designation may qualify for priority review and conditional approvals, facilitating faster access to innovative treatments for patients with urgent medical needs.
