Novartis’ Scemblix Gains Positive CHMP Opinion For Broad Use In Ph+ Chronic Myeloid Leukemia

Novartis announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending marketing authorization for 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 major milestone for Scemblix as it expands its potential use beyond later-line settings.

“For people living with CML, long-term therapy can be physically and emotionally demanding, and many face challenges in reaching treatment milestones without compromising quality of life,” said David FitzGerald, Member of the CML Advocates Network. “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.”

The Phase III ASC4FIRST trial served as the basis for the CHMP’s decision, comparing Scemblix with several tyrosine kinase inhibitors (TKIs) including imatinib, nilotinib, dasatinib, and bosutinib in patients newly diagnosed with Ph+ CML-CP. Scemblix showed superior major molecular response (MMR) rates over all TKIs tested, including imatinib alone. Patients receiving Scemblix also had fewer dose reductions and half the discontinuation rate due to side effects, highlighting its improved tolerability and effectiveness profile.

"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," said Professor Andreas Hochhaus, Head of the Department of Hematology and Medical Oncology at Jena University Hospital, Germany. "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, compared with available first-line treatments — potentially paving the way for more patients to reach treatment-free remission."

Scemblix is already approved in earlier treatment lines in more than 20 countries, including the U.S., Japan, and China, and is recognized as a recommended therapy in both the European LeukemiaNet 2025 guidelines and the NCCN Clinical Practice Guidelines for CML. Since 2021, it has been a standard of care in over 80 countries for patients previously treated with two or more TKIs. Following the CHMP’s positive recommendation, the European Commission (EC) is expected to make a final approval decision within two months, potentially broadening access to Scemblix for more CML patients across Europe.

“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,” said Patrick Horber, M.D., President, International, Novartis. “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."