On June 2, 2023 Innovent Biologics, Inc. ("Innovent") (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of oncology, metabolic, autoimmune, ophthalmology and other major diseases, reported that the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) has granted Breakthrough Therapy Designation (BTD) for olverembatinib for the treatment of patients with succinate dehydrogenase (SDH)-deficient gastrointestinal stromal tumor (GIST) who had received first-line treatment (Press release, Innovent Biologics, JUN 2, 2023, View Source [SID1234632403]).
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Results from an ongoing Phase 1b/2 study of olverembatinib in China showed an impressive clinical benefit rate (CBR) of 93.8% in patients with this subtype of GIST. Based on these promising results, the study was selected for presentations at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting for two consecutive years[1].
Olverembatinib is the first and only third-generation BCR-ABL inhibitor approved in China for the treatment of adult patients with tyrosine kinase inhibitors (TKI)-resistant chronic-phase chronic myeloid leukemia (CML-CP) or accelerated-phase CML (CML-AP) harboring the T315I mutation as confirmed by a validated diagnostic test. Olverembatinib is jointly commercialized in China by Ascentage Pharma and Innovent Biologics.
This marks the second BTD granted to olverembatinib by the CDE, with the first one granted in March 2021 for the treatment of patients with CML-CP resistant and/or intolerant to first- and second-generation TKIs. A New Drug Application (NDA) for the first BTD indication was accepted by the NMPA in July 2022 and subsequently granted Priority Review designation that will support a full approval of olverembatinib.
Dr. Hui Zhou, Senior Vice President of Innovent stated:" We are glad to see the NMPA grants another BTD for olverembatinib, marking a major milestone in its clinical development and demonstrating its therapeutic potentials beyond hematologic malignancies. We look forward to more good news of olverembatinib that allow more patients to benefit from this novel drug as soon as possible."
NMPA Breakthrough Therapy Designation is intended to facilitate and expedite the development and review of an investigational drug to treat a serious disease or condition when preliminary clinical evidence indicates that the drug has demonstrated substantial improvement over current therapies. The BTD will not only qualify a drug candidate to receive status for rapid review by the CDE, but it will also allow the sponsor to obtain timely advice and communication from the CDE to accelerate the approval and launch to address the unmet clinical need of patients at an accelerated pace. Click here for the published list of drugs which have been granted BTD by NMPA.
About Gastrointestinal Stromal Tumor (GIST)
GIST is a type of malignancy that arises in mesenchymal tissues of the gastrointestinal tract, and most patients with GIST harbor KIT or PDGFRA mutations. The introduction of TKIs has significantly improved the prognosis of these patients. However, patients with SHD-deficient GIST, a rare subtype of GIST, still have considerable unmet medical needs. It is known to the research community that patients with SDH-deficient GIST are commonly insensitive to existing TKIs. Although patients with early-stage localized disease can benefit from surgical treatment, most of them eventually experience relapse[2]-[6]. At present, there is no standard of care for patients with relapsed or advanced SDH-deficient GIST, whose 5-year event-free survival (EFS) is only 24%[2]-[6].
About Olverembatinib
Developed by Ascentage Pharma with support from the National Major New Drug Discovery and Manufacturing Program in China, the orally active, third-generation BCR-ABL inhibitor olverembatinib is the first China-approved third-generation BCR-ABL inhibitor targeting drug-resistant chronic myeloid leukemia (CML). Olverembatinib can effectively target a spectrum of BCR-ABL mutants, including the T315I mutation.
In November 2021, olverembatinib was granted a conditional approval through the Priority Review process by the China National Medical Products Administration (NMPA) for the treatment of adult patients with tyrosine kinase inhibitor (TKI)-resistant chronic-phase CML (CML-CP) or accelerated-phase CML (CML-AP) harboring the T315I mutation as confirmed by a validated diagnostic test. Subsequently, Olverembatinib was included into the China 2022 National Reimbursement Drug List (NRDL) for the approved indication. In March 2021, olverembatinib was granted a Breakthrough Therapy Designation (BTD) by the CDE for the treatment of patients with CML-CP who are resistant and/or intolerant of first- and second-generation TKIs.
In overseas, olverembatinib was cleared by the US FDA in July 2019 to directly enter a Phase Ib study. Since 2018, the clinical results of olverembatinib have been selected for oral presentations at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meetings for five consecutive years, and was nominated for "Best of ASH (Free ASH Whitepaper)" in 2019. To date, olverembatinib has been granted one Fast Track Designation (FTD) and four Orphan Drug Designations (ODDs) from the US Food and Drug Administration (FDA) for the treatment of CML, acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), and gastrointestinal stromal tumour (GIST); and one Orphan Designation from the European Medicines Agency (EMA) of the European Union for the treatment of CML.
In July 2021, Ascentage Pharma (6855.HK) and Innovent Biologics (1801.HK) reached the agreement regarding the joint development and commercialization of olverembatinib in China.
*Olverembatinib has not been approved for any indication in the U.S.