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The EMA’s CHMP has recommended the approval of asciminib for Philadelphia chromosome–positive chronic myeloid leukemia in chronic phase.
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending the approval of asciminib (Scemblix) for the treatment of adult patients with Philadelphia chromosome (Ph)–positive chronic myeloid leukemia in chronic phase (CP-CML) in all lines of treatment.1
The recommendation was supported by data from the phase 3 ASC4FIRST trial (NCT04971226), which showed that adult patients with newly diagnosed Ph-positive CP-CML treated with asciminib (n = 201) experienced a major molecular response (MMR) rate of 67.7% at week 48 compared with 49.0% for those treated with investigator-selected TKIs (n = 204; difference, 18.9%; 95% CI, 9.6%-28.2%; adjusted 2-sided P < .001).2 Within the imatinib (Gleevec) stratum, the 48-week MMR rate was 69.3% for asciminib vs 40.2% for imatinib (difference, 29.6%; 95% CI, 16.9%-42.2%; adjusted 2-sided P < .001). In the second-generation TKI stratum, the 48-week MMR rate was 66.0% and 57.8% for asciminib and second-generation TKIs, respectively (difference, 8.2%; 95% CI, –5.1% to 21.5%).
The 96-week MMR rate was 74.1% with asciminib vs 52% for all other TKIs, and 76.2% for asciminib vs 47.1% imatinib in the imatinib stratum.1
“For [individuals] 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,” David FitzGerald, member of the CML Advocates Network, stated in a news release. “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.”
In October 2024, the FDA granted accelerated approval to asciminib for the treatment of adult patients with newly diagnosed, Ph-positive CP-CML, based on data from ASC4FIRST.3
The multicenter, open-label, randomized ASC4FIRST trial enrolled patients at least 18 years of age with CP-CML within 3 months of diagnosis who had confirmed Ph-positive disease.4 Patients could not have extramedullary leukemic involvement, other than hepatosplenomegaly. Other key inclusion criteria comprised an ECOG performance status of 0 or 1 and adequate end organ function.
Investigators excluded patients who received prior anticancer therapy for CML, other than hydroxyurea and/or anagrelide. Notably, patients were allowed to receive imatinib, nilotinib (Tasigna), dasatinib (Sprycel), or bosutinib (Bosulif) prior to enrollment if they received treatment for no more than two weeks; however, no other prior TKI exposure was permitted.
Patients were randomly assigned 1:1 to receive asciminib at 80 mg once daily or investigator’s choice of TKI, including imatinib, nilotinib, dasatinib, or bosutinib.
The study’s primary end point was MMR rate at week 48 for asciminib vs other TKIs in the overall population, as well as MMR rate at week 48 for asciminib vs imatinib within the imatinib stratum. Secondary end points included MMR rate at week 96, rates of treatment discontinuation due to adverse effects (AEs), complete hematological response rate, complete cytogenic response rate, duration of MMR, failure-free survival, event-free survival, progression-free survival, and overall survival.
"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," Andreas Hochhaus, MD, head of the Department of Hematology and Medical Oncology at Jena University Hospital in Germany, added in a news release.1 "If approved, [asciminib] could provide patients with a well-tolerated option that may deliver faster, deeper and longer-lasting molecular response with fewer treatment discontinuations due to AEs, compared with available first-line treatments—potentially paving the way for more patients to reach treatment-free remission."
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