Latest news with #TKIs
Cision Canada
30-07-2025
- Health
- Cision Canada
Health Canada expands approval of Scemblix®, making it an option for newly diagnosed and previously treated chronic myeloid leukemia (CML) patients Français
Scemblix ® is first to show superior efficacy and a favourable safety and tolerability profile in a Phase III trial vs. all standard of care (SoC) therapies 1,2 Fifty percent of CML patients do not meet efficacy milestones (MMR) with current SoC and almost 25% discontinue or switch therapies within one year of treatments 2 Scemblix ®, a new first-line option for adults with CML, is now approved for newly diagnosed and previously treated CML MONTREAL, July 30, 2025 /CNW/ - Novartis Canada is pleased to announce that Health Canada has granted a Notice of Compliance for Scemblix ® (asciminib tablets) for adult patients with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP) who are newly diagnosed or who have previously received one or more tyrosine kinase inhibitors (TKIs). 1 In Canada, Scemblix ® was previously approved for the treatment of adult patients with Ph+ CML-CP previously treated with two or more TKIs. Newly diagnosed patients will now have access to a treatment that has shown superior efficacy versus all standard of care (SoC) therapies and a favourable safety and tolerability profile. "The approval of a new treatment option for newly diagnosed and previously treated Canadians living with CML is an important milestone," said Lisa Machado, founder, Canadian CML Network. "As a person living with CML and an advocate, I am hopeful that expanded access to this innovative treatment option will offer patients not only continued positive outcomes, but also provide the opportunity to maintain a quality of life that meets their expectations as they and their families navigate the complexities of CML management." CML that is diagnosed in the chronic phase has a more favourable prognosis than CML that is diagnosed in the accelerated or blast phase. Although the Ph chromosome is present in everyone with CML, in rare cases it can't be found during testing. In general, Ph+ CML has a more favourable prognosis than Ph- CML. 3 While TKIs have transformed CML into a chronic disease, efficacy, safety and tolerability challenges continue to hinder long-term treatment success for many patients. Many patients do not meet molecular response goals, and many discontinue or change treatment due to intolerance. 4,5,6 "The approval of asciminib represents a significant step forward, expanding treatment options for CML patients," said Dr. Dennis Kim, Professor of Medicine, Princess Margaret Cancer Centre. "Having a diverse range of therapies available allows care teams to keep the unique needs of the patient at the centre of treatment plans, optimizing outcomes. The ability to prescribe asciminib to newly diagnosed and previously treated patients offers a promising new pathway in our efforts to manage this complex disease effectively and safely." "We are proud that Health Canada has expanded its approval of Scemblix ®, making it a new option for all Canadians with chronic myeloid leukemia, whether they are newly diagnosed or have been previously treated," said Mark Vineis, Country President, Novartis Canada. "This approval means patients and their physicians now have more choices when deciding on the best course of treatment, offering renewed hope for individuals living with CML, their families, and the healthcare teams dedicated to their care." The clinical effectiveness, safety and cost-effectiveness of Scemblix ® is currently under review by Canada's Drug Agency (CDA) and Institut National d'Excellence en Santé et Services Sociaux (INESSS). Novartis looks forward to communicating their recommendations with the CML community, when available. About Chronic Myeloid Leukemia (CML) Chronic myeloid leukemia (CML) is a type of cancer that develops in the blood-forming cells of the bone marrow. In 95% of patients with CML, a genetic mutation produces an abnormal chromosome in bone marrow stem cells known as the Philadelphia chromosome ("Ph chromosome"). When the Ph chromosome is present, CML is classified as Philadelphia chromosome-positive (Ph+). 7 The Ph chromosome produces the BCR-ABL1 protein, which causes bone marrow to make too many abnormal white blood cells. These cells overcrowd healthy blood cells, which can be fatal if untreated. 7 CML has three stages: chronic, accelerated, and blast phases. Most patients are diagnosed in the chronic phase, and with proper treatment, can often stay in this early stage without advancing further. 7 According to the most recently available data, 665 Canadians were diagnosed with CML in 2019 and 140 Canadians died from CML in 2022. 8 About Scemblix ® (asciminib tablets) Scemblix ® is the first CML treatment that works by Specifically Targeting the ABL Myristoyl Pocket (referred to as a STAMP inhibitor in scientific literature). 9,10 The current approved CML treatments are TKIs that target the adenosine triphosphate (ATP)-binding site (ATP-competitive). 10 About the ASC4FIRST Trial The approval of Scemblix ® is based on results from the ongoing Phase III ASC4FIRST trial in patients newly diagnosed with Ph+ CML-CP. 1 Data has shown: Nearly 20% more patients treated with Scemblix ® achieved MMR versus investigator-selected (IS) SoC TKIs (imatinib, nilotinib, dasatinib and bosutinib) (68% vs. 49%, p < 0.001) and nearly 30% more patients achieved MMR versus imatinib alone (69% vs. 40%, p < 0.001) at week 48. 1,2 Scemblix ® is the first CML treatment to show superior efficacy along with a favourable safety and tolerability profile verus imatinib and second generation TKIs. 1,2 In newly diagnosed Ph+ CML-CP patients, the most common adverse reaction (≥ 20%) was musculoskeletal pain. Serious adverse events occurred in 11% of patients who received Scemblix ®. Serious adverse reactions in ≥ 1% included pancreatitis (1%). 1, 2 About Novartis Novartis is a focused innovative medicines company. Every day, we work to reimagine medicine to improve and extend people's lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach more than 250 million people worldwide. In Canada, Novartis Pharmaceuticals Canada Inc. employs approximately 600 people to serve the evolving needs of patients and the healthcare system and invests over $30 million in R&D yearly in the country. For more information visit SCEMBLIX ® is a registered trademark. SOURCE Novartis Pharmaceuticals Canada Inc.
Medscape
04-06-2025
- Health
- Medscape
Fast Five Quiz: B-Cell Acute Lymphoblastic Leukemia
Though more common in pediatric populations, B-cell acute lymphoblastic leukemia (B-ALL) affects both children and adults, and it has several genetic subtypes that significantly affect prognosis and treatment decisions. Despite advances in treatment protocols, resistance mechanisms remain a significant challenge. However, some genetic subtypes are associated with more favorable prognosis, and the nuances of this disease are evident in the cascading treatment algorithm recommended by the National Comprehensive Cancer Network (NCCN) for pediatric and adult cases. What do you know about B-ALL? Check your knowledge with this quick quiz. The treatment of B-ALL involves a complex and intensive series of steps. In the induction phase, glucocorticoids are typically used to reduce tumor burden by clearing leukemic cells from the bone marrow. However, point mutations in the NR3C1 gene generally cause resistance to glucocorticoid therapy, which can significantly affect prognosis. Mutations in the CREBBP gene also cause resistance to glucocorticoids DHFR , FPGS , and TYMS genes have been shown to generally cause resistance to methotrexate, not glucocorticoids. Learn more about corticosteroids in B-ALL. In risk stratification for B-ALL, the NCCN notes that having a white blood cell count of > 30 x 109/L is considered a high-risk feature. This is consistent with European guidelines. The NCCN also states that age over 35 years is another high-risk feature for B-ALL. ETP phenotype is a high-risk feature for T-cell acute lymphoblastic leukemia rather than the B-ALL subtype. Hyperdiploidy (a molecular subtype of B-ALL with 51-65 chromosomes) is considered standard risk by the NCCN; data have shown better prognosis for pediatric patients with this subtype. Learn more about risk stratification for B-ALL. TKIs are an important component of induction, consolidation, and maintenance treatment for Ph+ B-ALL. They are recommended by the NCCN for use in conjunction with other drugs, such as corticosteroids, blinatumomab, and inotuzumab ozogamicin (depending on treatment phase and disease severity) and as a treatment post-HCT. They recommend continuing TKI therapy for at least 2 years after HCT, although they note that the 'optimal duration' is unknown for this population. A recent review, however, notes that despite their success in patients with Ph+ B-ALL, approximately 25% of cases will develop resistance to TKIs. Given this possibility, the NCCN notes that clinicians should consider prior TKI intolerance, dose used, BCR::ABL1 mutations, and disease-related features when choosing a specific TKI; they specifically recommend bosutinib, dasatinib, imatinib, nilotinib, or ponatinib as options for TKI therapy. Learn more about Ph+ B-ALL treatments. The DUX4-rearranged subtype of B-ALL is considered to have a favorable prognosis in adolescents and young adults, compared with MEF2D-rearranged, CDX2/UBTF, and IDH1/2; these subtypes generally have inferior prognosis in the same populations (although MEF2D-rearranged has intermediate prognosis in adults). Specifically, DUX4-rearranged B-ALL has been associated with 93% event-free survival and overall survival in pediatric patients, and adolescent and young-adult patients also see longer disease-free survival after complete remission is achieved. Learn more about B-ALL genomics. Several genetic subtypes of B-ALL are associated with varying prognoses, and the outcomes differences seen between pediatric and adult patients can be partly explained by the different subtypes expressed by these populations. Further, new therapies have enabled high survival rates among pediatric patients with B-ALL, with long-term survival of up to 90% in this population. Prognosis for B-ALL in adolescents and adults is comparatively poor. For example, a population-based study reported the following declining survival rates with age: 74% for patients 15-19 years old, 59% for patients 20-39 years old, and 43% for those 40-59 years old. However, the researchers explained that these percentages still demonstrate improvements, as survival rates were overall lower in the 1980s and 1990s. Learn more about B-ALL prognosis.
Malaysian Reserve
06-05-2025
- Business
- Malaysian Reserve
Fangzhou Launches Otsuka's Third-Generation Leukemia Drug Ponatinib on its Platform
GUANGZHOU, China, May 5, 2025 /PRNewswire/ — Fangzhou Inc. ('Fangzhou' or the 'Company') ( a leader in Internet healthcare solutions, announced the availability of Otsuka Pharmaceutical's third-generation tyrosine kinase inhibitor (TKI) Iclusig® (ponatinib) through its online platform, providing expanded treatment options for patients with chronic myeloid leukemia (CML) and Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). Iclusig®, approved in China in September 2024, has demonstrated particular efficacy against T315I-mutated BCR::ABL1 disease that is resistant to first and second generation TKIs, with the OPTIC trial showing a four-year survival rate of 88% among certain patient cohorts. Dr. Xie Fangmin, founder, chairman, and CEO of Fangzhou, commented, 'As a platform focused on patient-centered healthcare, we are committed to bringing innovative treatments such as Iclusig® to Chinese patients through our digital healthcare ecosystem.' Deep Collaboration with Pharmaceutical Companies Fangzhou has developed strong partnerships with leading domestic and multinational pharmaceutical companies, along with a comprehensive drug supply chain spanning multiple therapeutic areas including oncology, liver disease, and cardiovascular, and now offering over 210,000 drug SKUs to meet the diverse needs patient chronic disease patients. By harnessing AI, big data, and cloud computing technologies, Fangzhou is focused on continually enhancing its health management services. In addition, the company's proprietary 'AI Personal Health Assistant' and other innovative tools help to improve medication safety and enhance treatment adherence. AI-enabled Full-cycle Healthcare Fangzhou delivers comprehensive health services enabled by AI technology. Through the company's 'AI + H2H Smart Healthcare Platform,' patients can access online medical consultations and personalized health recommendations designed to enhance their quality of life. By combining access to innovative drugs with precision health services, Fangzhou is reshaping full-cycle health management through its 'technology + care' approach. Looking ahead, the company plans to deepen AI integration within its Internet-based medical services while expanding partnerships with leading global pharmaceutical companies in order to bring more breakthrough treatments and cutting-edge innovations to patients and their families. About Fangzhou Inc. Fangzhou Inc. ( is China's leading online chronic disease management platform, serving 49.2 million registered users and 223,000 physicians (as of December 31, 2024). The Company specializes in delivering tailored medical care and precision medicine solutions. For more information, visit Media Contact For further inquiries or interviews, please reach out to: Xingwei Zhao Associate Director of Public Relations Email: pr@ Disclaimer: This press release contains forward-looking statements. Actual results may differ materially from those anticipated due to various factors. Readers are cautioned not to place undue reliance on these statements
