Health Canada expands approval of Scemblix®, making it an option for newly diagnosed and previously treated chronic myeloid leukemia (CML) patients

• 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 treatments²
• 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 www.novartis.ca.

SCEMBLIX^® is a registered trademark.

References

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¹ Scemblix^® (asciminib) 2025 Product Monograph. https://www.novartis.com/ca-en/content/scemblix.

² Hochhaus A, Wang J, Dong-Wook K, et al. Asciminib in Newly Diagnosed Chronic Myeloid Leukemia. N Engl J Med. 2024. doi:10.1056/NEJMoa2400858

³ Canadian Cancer Society. Prognosis and survival for chronic myeloid leukemia. Available at: https://cancer.ca/en/cancer-information/cancer-types/chronic-myeloid-leukemia-cml/prognosis-and-survival. Accessed on June 16, 2025.

⁴ Hochhaus, A., & Ernst, T. 2021. TKI discontinuation in CML: How do we make more patients eligible? How do we increase the chances of a successful treatment-free remission? Hematology Am Soc Hematol Educ Program, 2021(1), 106–112. 

⁵ Hehlmann R, Cortes JE, Zyczynski T, et al. Tyrosine kinase inhibitor interruptions, discontinuations and switching in patients with chronic-phase chronic myeloid leukemia in routine clinical practice: SIMPLICITY. Am J Hematol. 2019;94(1):46-54. doi:10.1002/ajh.25306  

⁶ Claudiani S, Chughtai F, Khan A. et al. Long-term outcomes after upfront second-generation tyrosine kinase inhibitors for chronic myeloid leukemia: managing intolerance and resistance. Leukemia. 2024;38:796 - 802. doi:10.1038/s41375-024-02187-w  

⁷ Mayo Clinic. Chronic myelogenous leukemia. Available at: www.mayoclinic.org/diseases-conditions/chronic-myelogenous-leukemia/symptoms-causes/syc-20352417. Accessed on May 16, 2025.

⁸ Canadian Cancer Society. Chronic myeloid leukemia statistics. Available at: https://cancer.ca/en/cancer-information/cancer-types/chronic-myeloid-leukemia-cml/statistics. Accessed on May 16, 2025.

⁹ Cortes JE, Hughes TP, Mauro MJ, et al. Asciminib, a First-in-Class STAMP Inhibitor, Provides Durable Molecular Response in Patients (pts) with Chronic Myeloid Leukemia (CML) Harboring the T315I Mutation: Primary Efficacy and Safety Results from a Phase 1 Trial. Oral presentation at: ASH Annual Meeting; Dec. 7, 2020. 

¹⁰ Schoepfer J, Jahnke W, Berellini G, et al. Discovery of Asciminib (ABL001), an Allosteric Inhibitor of the Tyrosine Kinase Activity of BCR-ABL1. J Med Chem. 2018;61(18):8120-8135. doi:10.1021/acs.jmedchem.8b01040 

SOURCE Novartis Pharmaceuticals Canada Inc.

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