Skip to content
Medical Health Aged Care, Science


Novartis Australia 5 mins read
  • Approval based on results from open-label randomised Phase III ASCEMBL trial, 4,5


  • Scemblix is a first-in-class BCR-ABL1 inhibitor specifically targeting the ABL Myristoyl Pocket (STAMP) 4,5


  • Scemblix offers a different therapeutic option to patients with chronic myeloid leukaemia (CML) who struggle with intolerance or inadequate response after at least two prior tyrosine kinase inhibitor treatments4,5


  • Chronic myeloid leukaemia is a type of cancer that affects the blood and bone marrow, that is estimated to impact around 330 Australians annually,with numbers forecast to rise to 2,130 by 20353


  • Novartis is proud of its work in transforming the standard of care in CML and delivers on its 20-year innovation in the space of CML by bringing Scemblix to patients across Australia



30 MAY 2023, Sydney: Novartis Australia welcomes the listing of Scemblix® (asciminib) on the Pharmaceutical Benefits Scheme (PBS) for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukaemia in chronic phase (Ph+ CML-CP), previously treated with two or more tyrosine kinase inhibitors (TKIs). We also welcome the listing of Scemblix for the treatment of Ph+ CML in CP with the T315I mutation.1,4

Scemblix is a novel treatment option that works by specifically targeting the ABL myristoyl pocket (also known as a STAMP inhibitor in scientific literature), offering a reimagined treatment approach for patients, who experience intolerance and/or resistance to currently available TKI therapies.4,5

“Despite major advances in CML treatments over the past two decades, many patients still struggle with treatment side effects and resistance, which can impact their quality of life and increase the risk of disease progression,” said Professor Timothy Hughes, an international expert in the biology and treatment of leukaemia, Chair of the International Chronic Myeloid Leukaemia Foundation (iCMLf) and Co-Theme Leader in Precision Cancer Medicine at SAHMRI in Adelaide.

"Scemblix’s unique mechanism of action provides an alternative treatment approach for patients who have experienced intolerance or inadequate response to currently available therapies, with the potential to improve outcomes and quality of life," Professor Hughes said.

Chronic myeloid leukaemia (CML) is a type of cancer that affects the blood and bone marrow, resulting in the overproduction of white blood (granulocytes), which can impede normal blood cell production and the body’s ability to fight infections.2

With the incidence of blood cancer continuing to rise, our priority is to ensure that every Australian diagnosed with blood cancer, no matter where they live, or community they identify with, is able to access the best treatments and services that will not only help them to survive their blood cancer, but to also live well,” said Chris Tanti, Leukaemia Foundation Chief Executive Officer.

It is estimated that each year in Australia, around 330 people will be diagnosed with CML, accounting for around 0.03% of all cancers diagnosed in the country.2 The number of people affected by CML in Australia is forecast to increase fivefold to 2,130 by 2035, according to a recent report published by the Leukaemia Foundation, and the number of Australians who will lose their lives to the disease is predicted to triple.3

“Building on over twenty years of innovation in haematology, Novartis, is proud to once again contribute, with the listing of Scemblix, to the potential transformation in the care of patients living with chronic myeloid leukaemia,” said Richard Tew, Country President Novartis Australia, and New Zealand.

The approval is based on results from the pivotal Phase III ASCEMBL trial.5

Please review the full Consumer Medicines Information for Scemblix® here -




About Chronic Myeloid Leukaemia

Chronic myeloid leukemia (CML) is a type of cancer that affects the blood and bone marrow, resulting in the overproduction of white blood (granulocytes), which can impede normal blood cell production and the body’s ability to fight infections.It is estimated that each year in Australia, around 330 people will be diagnosed with CML, accounting for around 0.03% of all cancers diagnosed in the country.2


About Novartis Commitment to CML

Novartis has a long-standing scientific commitment to patients living with CML. For more than 20 years, our bold science has helped transform CML into a chronic disease for many patients. Despite these advancements, we’re not standing still. We continue to research ways to target the disease, seeking to address the challenges with treatment resistance and/or intolerance that many patients face. Novartis also continues to reimagine CML care through its commitment to sustainable access for patients and collaboration with the global CML community.


About Scemblix® (asciminib)

Scemblix is the first CML treatment that acts as a STAMP inhibitor, specifically targeting the ABL myristoyl pocket.4,5 This novel mechanism of action may help address resistance in patients with CML previously treated with two or

more TKIs and overcome mutations at the defective BCR:ABL1 gene, which is associated with the over-production of leukaemic cells.4,5,12-18 While many patients will benefit from available TKI therapies, a significant proportion may experience intolerance or resistance to these treatments.6-11 It is estimated that over 25% of CML patients will switch TKIs at least once during their lifetime due to TKI intolerance or resistance.27 Scemblix represents an important development for patients who experience resistance and/or intolerance to currently available TKI therapies, and it is being studied across multiple treatment lines for CML-CP, both as a monotherapy and in combination.4,12-26 Specifically, the ASC4FIRST Phase III study (NCT04971226) evaluates Scemblix in newly diagnosed adult patients with Ph+ CML-CP vs. an investigator-selected TKI.20 In October 2021, the USFDA granted accelerated approval of Scemblix for adult patients with Ph+ CML-CP, previously treated with two or more TKIs based on MMR rate at 24 weeks, and full approval for adult patients with Ph+ CML-CP with the T315I mutation. In accordance with the Accelerated Approval Program, continued approval for the first indication may be contingent upon verification and description of clinical benefit from confirmatory evidence. The longer-term, 96- week efficacy and safety data have been shared with the FDA and are currently under evaluation through a priority review. Scemblix has received approval in several countries outside the US, including Japan, Switzerland, and the United Kingdom, for adult patients with Ph+ CML-CP with resistance or intolerance to at least two or more previous therapies.28


About Novartis

Novartis is improving the lives of more than 2.8 million patients across Australia and New Zealand through our medicines. By partnering with the healthcare system, we are working for patients to address their needs, and we are committed to accelerating patient access to life saving treatments and associated healthcare. We are reimagining medicine by using innovative science and technology to address challenging healthcare issues and our rich research pipeline has 200+ projects in development. We are transforming how clinical trials are run to increase patients’ access to our industry-leading clinical trials footprint in Australia. We continue to work on improving our environment for diversity, equity and inclusion and being a family friendly employer. Every day our inspired, curious, unbossed, and integrity-driven culture unites our people and encourages leadership at every level.




    1. Scemblix
    2. Leukaemia Foundation. (n.d.). Chronic myeloid leukaemia. Leukaemia Foundation.
    3. Leukaemia Foundation. (2020). State of the nation: Blood cancer in Australia. Australia_Leukaemia-Foundation.pdf
    4. Scemblix Summary of Product Characteristics, 2022.
    5. Rea D, et al. A Phase 3, Open-Label, Randomized Study of Asciminib, a STAMP Inhibitor, vs Bosutinib in CML After≥ 2 Prior TKIs. Blood. 2021. DOI: 10.1182/blood.2020009984. PMID: 34407542.
    6. Flis S, et al. Chronic myelogenous leukemia, a still unsolved problem: pitfalls and new therapeutic possibilities. Drug Des Devel Ther. 2019;13:825-843.
    7. Akard LP, et al. The “Hit Hard and Hit Early” Approach to the Treatment of Chronic Myeloid Leukemia: Implications of the Updated National Comprehensive Cancer Network Clinical Practice Guidelines for Routine Practice. Clin Adv Hematol Oncol. 2013;11(7):421-432.
    8. Cortes JE, et al. Long-term bosutinib for chronic phase chronic myeloid leukemia after failure of imatinib plus dasatinib and/or nilotinib. Am J Hematol. 2016;91(12):1206-1214.
    9. Cortes JE, et al. Ponatinib efficacy and safety in Philadelphia chromosome–positive leukemia: Final 5-year results of the phase 2 PACE trial. Blood. 2018;132(4):393-404.
    10. Garg RJ, et al. The use of nilotinib or dasatinib after failure to 2 prior tyrosine kinase inhibitors: long-term follow-up. Blood. 2009;114(20):4361-4368
    11. Steegmann JL., et al. European LeukemiaNet recommendations for the management and avoidance of adverse events of treatment in chronic myeloid leukaemia. Leukemia. 2016;30:1648-1671.
    12. Wylie AA, et al. The allosteric inhibitor ABL001 enables dual targeting of BCR–ABL1. Nature. 2017;543(7647):733-737.
    13. Schoepfer J, 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.
    14. Hughes TP, et al. Asciminib in Chronic Myeloid Leukemia after ABL Kinase Inhibitor Failure. N Engl J Med. 2019; 381(24):2315-2326.
    15. Hughes TP, et al. Expanded Phase 1 Study of ABL001, a Potent, Allosteric Inhibitor of BCR-ABL, Reveals Significant and DurableResponses in Patients with CML-Chronic Phase with Failure of Prior TKI Therapy. Poster presented at: ASH Annual Meeting & Exposition; Dec. 5, 2016.
    16. Ottmann OG, et al. ABL001, a Potent, Allosteric Inhibitor of BCR-ABL, Exhibits Safety and Promising Single- Agent Activity in a Phase I Study of Patients with CML with Failure of Prior TKI Therapy. Blood. 2015;126(23):138.
    17. Mauro MJ, et al. Combination of Asciminib Plus Nilotinib (NIL) or Dasatinib (DAS) in Patients (PTS) with Chronic Myeloid Leukemia(CML): Results from a Phase 1 Study. Poster presented at: EHA Annual Meeting; June 15, 2019.
    18. Cortes JE, et al. Combination Therapy Using Asciminib Plus Imatinib (IMA) in Patients (PTS) with Chronic Myeloid Leukemia (CML):Results from a Phase 1 Study. Poster presented at: EHA Annual Meeting; June 15, 2019.
    19. 2017. Study of Efficacy of CML-CP Patients Treated with ABL001 Versus Bosutinib, Previously Treated With 2 or More TKIs. [online] Available at:
    20. 2021. A Study of Oral Asciminib Versus Other TKIs in Adult Patients With Newly Diagnosed Ph+ CML-CP. [online] Available at:
    21. 2014. A Phase I Study of Oral ABL001 in Patients With CML or Ph+ ALL. [online] Available at:
    22. 2018. Study of Efficacy And Safety Of Asciminib In Combination With Imatinib In Patients With Chronic Myeloid Leukemia In Chronic Phase (CML-CP). [online] Available at:
    23. 2021. Study of Efficacy and Safety of CML-CP Patients Treated With Asciminib Versus Best Available Therapy,Previously Treated With 2 or More Tyrosine Kinase Inhibitors. [online] Available at:
    24. 2021 Asciminib Treatment Optimization in ≥ 3rd Line CML-CP. [online] Available at:
    25. 2021. Study to Determine the Dose and Safety of Asciminib in Pediatric Patients With Chronic Myeloid Leukemia [online] Available at:
    26. 2020. Asciminib in Monotherapy for Chronic Myeloid Leukemia in Chronic Phase (CML- CP) With and WithoutT315I Mutation (AIM4CML). [online] Available at:
    27. Steegmann JL, Baccarani M, Breccia M, et al. European LeukemiaNet recommendations for the management and avoidance of adverse events of treatment in chronic myeloid leukaemia. Leukemia. 2016
    28. Novartis. (2022, January 20). Novartis' Scemblix, a novel mechanism of action, approved by European Commission for adult patients with chronic myeloid leukemia [Press release]. european-commission-adult-patients-chronic-myeloid-leukemia






Contact details:

Val Staikou

Communications and Patient Engagement Lead


0438 404 878


Joni Thomes

Associate Director

Palin Communications

0421 967 709

More from this category

  • Medical Health Aged Care
  • 07/12/2023
  • 13:30
Royal Australian College of GPs and Australian College of Rural and Remote Medicine

Public consultation on Rural Generalist Medicine recognition closes next Tuesday

With less than one week to go until the public consultation closes on recognition of Rural Generalist Medicine (RGM) as a specialist field, Australia’s two GP colleges urge doctors and community members to have their say. The Australian College of Rural and Remote Medicine (ACRRM) and the Royal Australian College of General Practitioners (RACGP) are buoyed by the conversation that has been generated on the recognition of RGM throughout the process. ACRRM President Dr Dan Halliday says it has been wonderful to see the engagement through the recent webinars hosted by the Rural Generalist Taskforce, through College channels and at…

  • Contains:
  • Medical Health Aged Care, Science
  • 07/12/2023
  • 13:26
The Florey

Researchers identify neurons in the brain that control nasofacial muscles during breathing

Whilst most of us consider breathing a relatively simple process, it actually requires complex coordination of many muscles to enable airflow into and out of the lungs and to control airflow to allow us to talk, eat, and drink. Breathing also influences other related brain functions, such as emotional state, sense of smell, blood pressure, and heart rate. Breathing is generated in a brain region called Pre-Bötzinger complex (preBötC), which is composed of many subgroups of neurons that are breathing and non-breathing related. Up to now, due to technical limitations, it was almost impossible to specifically silence a subgroup of…

  • Medical Health Aged Care
  • 07/12/2023
  • 13:19
Royal Australian College of GPs

GPs vital to improving National Disability Insurance Scheme

The Royal Australian College of GPs (RACGP) has welcomed the final National Disability Insurance Scheme report and reiterated calls for GPs to be better utilised. It comes following the release today of the final report into the scheme. RACGP President Dr Nicole Higgins said that GPs must be front and centre. “It is concerning that the report doesn’t deeply delve into healthcare for people with a disability, including general practice care,” she said. “The Government must recognise that GPs play a vital role in disability care, and barriers do exist. For example, there is no Medicare patient rebate for NDIS…

Media Outreach made fast, easy, simple.

Feature your press release on Medianet's News Hub every time your distribute with Medianet. Pay per release or save with a subscription.