Asciminib (Scemblix)

Key Messages

• Canada’s Drug Agency (CDA-AMC) recommends that Scemblix should not be reimbursed by public drug plans for the treatment of adult patients with Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) in chronic phase previously treated with 1 tyrosine kinase inhibitor (TKI).
• Evidence from a clinical trial (ASC2ESCALATE) of 101 patients who could not tolerate or did not respond to their first-line TKIs suggested that Scemblix was associated with a clinically meaningful proportion of patients achieving major molecular response (MMR) at 9 months. However, these results were highly uncertain because of the lack of a control group that did not compare Scemblix to other treatments and the short and incomplete study duration that relied on early data that had not reached the primary end point. Additionally, there was no indirect comparison with other available drugs, so it was not possible to determine the benefit or risks of Scemblix compared to other treatments.
• Patients and clinicians identified the need for additional effective treatments that improve symptom control, reduce side effects, and enhance quality of life. Patients also want multiple treatment options as resistance or intolerance to other TKIs are common. However, the committee was unable to conclude that Scemblix would meet the clinical needs of patients and provide an effective treatment with improved symptom control and quality of life, or superiority in efficacy relative to other treatments in Ph+ CML in second line.