CHMP Recommends Scemblix for Newly Diagnosed CML in Europe, Offering Superior Efficacy and Tolerability
Novartis has announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending the approval of Scemblix® (asciminib) for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP) across all lines of therapy. This recommendation marks a significant step forward in expanding treatment options for patients living with CML in Europe.
Expanding Options for Newly Diagnosed Patients
For individuals diagnosed with CML, long-term therapy often comes with substantial physical and emotional challenges. David FitzGerald, Member of the CML Advocates Network, emphasized the impact of additional therapeutic choices:
The availability of more treatment options earlier in the care pathway is a welcome development that brings forward additional possibilities for patients and their healthcare teams to choose approaches that best support both clinical goals and patient well-being.
Scemblix’s potential for use in newly diagnosed patients could allow clinicians to intervene earlier with a therapy that combines high efficacy with favorable tolerability, potentially improving long-term patient outcomes while maintaining quality of life.
Clinical Evidence Supporting CHMP Recommendation
The CHMP’s positive opinion is based on data from the Phase III ASC4FIRST trial, which evaluated Scemblix against investigators’ choice of standard tyrosine kinase inhibitors (TKIs) in adults with newly diagnosed Ph+ CML-CP. TKIs included in the comparator group were imatinib, nilotinib, dasatinib, and bosutinib.
Results from ASC4FIRST demonstrated that Scemblix achieved superior major molecular response (MMR) rates compared with all TKIs collectively and specifically against imatinib. Importantly, patients treated with Scemblix experienced fewer dose reductions and approximately half the rate of adverse events leading to treatment discontinuation, underscoring its favorable tolerability profile.
Expert Perspectives
Professor Andreas Hochhaus, Head of the Department of Hematology and Medical Oncology at Jena University Hospital, Germany, highlighted the clinical importance of early intervention:
To give patients newly diagnosed with CML the best chance to reach key efficacy milestones while maintaining quality of life, it is critical to intervene early with a more selective treatment that combines superior efficacy with tolerability. If approved, Scemblix could provide patients with a well-tolerated option that may deliver faster, deeper and longer-lasting molecular response with fewer treatment discontinuations due to adverse events — potentially paving the way for more patients to reach treatment-free remission.
Such outcomes are particularly relevant given the growing emphasis on treatment-free remission (TFR) as a long-term goal in CML management. Early, deep molecular responses are strongly associated with the ability to safely discontinue therapy while maintaining disease control.
Scemblix: Global Adoption and Guideline Recognition
Scemblix is already approved in earlier lines of therapy in over 20 countries, including the United States, Japan, and China. It is recommended for newly diagnosed Ph+ CML-CP in the 2025 European LeukemiaNet (ELN) recommendations and the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology (NCCN Guidelines®).
Since its initial approval in 2021, Scemblix has become a standard of care in more than 80 countries for patients previously treated with two or more TKIs, offering a well-tolerated alternative for those who have experienced suboptimal responses or intolerance to existing therapies.
Patrick Horber, M.D., President, International, Novartis, emphasized the significance of this development:
People newly diagnosed with CML need treatments that deliver both better efficacy and excellent tolerability to achieve early, deep molecular response, which is critical to achieve long-term outcomes like treatment-free remission. Scemblix is the only treatment that has demonstrated superior efficacy and tolerability compared to current first-line treatments. Today’s positive CHMP opinion marks a major milestone in our 25-year journey to improve CML care and could help establish a new standard of care in Europe.
Mechanism of Action
Scemblix is a first-in-class allosteric inhibitor of BCR-ABL1, which differentiates it from conventional ATP-competitive TKIs. By specifically targeting the myristoyl pocket of BCR-ABL1, Scemblix inhibits the kinase in a highly selective manner, potentially contributing to its superior efficacy and favorable safety profile. This novel mechanism of action allows for effective suppression of leukemic activity while reducing off-target effects that can lead to adverse events commonly seen with traditional TKIs.
Next Steps in Europe
Following CHMP’s positive recommendation, the European Commission (EC) is expected to issue a final decision within two months, which would formally authorize Scemblix for use in adults with Ph+ CML-CP in all lines of therapy across the EU. Once approved, the treatment could be rapidly integrated into European clinical practice, providing physicians and patients with a new, well-tolerated option for early intervention in newly diagnosed CML.
Implications for Patients
The CHMP recommendation for Scemblix represents a significant advancement in the CML treatment landscape, particularly for patients seeking therapies that balance efficacy and tolerability. With fewer dose reductions, lower discontinuation rates due to adverse events, and superior molecular responses, Scemblix has the potential to improve long-term outcomes and quality of life, offering hope for achieving treatment-free remission — a milestone increasingly recognized as a key goal in modern CML management.
Scemblix’s novel mechanism of action, coupled with robust clinical evidence from the ASC4FIRST trial, positions it as a potential new standard of care for adults with newly diagnosed Ph+ CML-CP in Europe. The CHMP’s positive opinion is a major step forward in expanding access to this innovative therapy, offering patients a highly effective, well-tolerated treatment option early in their disease journey, and supporting Novartis’ ongoing commitment to transforming care in chronic myeloid leukemia.
