FDA approved Scemblix (asciminib) for Philadelphia Chromosome-Positive Chronic Myeloid LeukemiaFDA approved New Daily Pill for a Common Type of Adult Leukemia

Scemblix is a kinase inhibitor. The specific mechanism of action is not described in the provided label text.

Scemblix is indicated for the treatment of adult patients with: - Newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP). This indication is approved under accelerated approval based on major molecular response rate. Continued approval for this indication may be contingent upon verification of clinical benefit in a confirmatory trial(s). - Previously treated Ph+ CML in CP. - Ph+ CML in CP with the T315I mutation.

Recommended dosage in Ph+ CML in CP: 80 mg orally once daily or 40 mg orally twice daily. Recommended dosage in Ph+ CML in CP with the T315I mutation: 200 mg orally twice daily. Avoid food for at least 2 hours before and 1 hour after taking Scemblix. Swallow tablets whole. Do not break, crush, or chew the tablets. Missed dose: For once daily regimen, if a dose is missed by more than approximately 12 hours, skip the dose and take the next dose as scheduled. For twice daily regimens, if a dose is missed by more than approximately 6 hours, skip the dose and take the next dose as scheduled. Dosage modifications for adverse reactions: For newly diagnosed or previously treated Ph+ CML-CP, first reduction is 40 mg once daily or 20 mg twice daily; subsequent reduction: permanently discontinue if unable to tolerate 40 mg once daily or 20 mg twice daily. For Ph+ CML-CP with T315I mutation, first reduction is 160 mg twice daily; permanently discontinue if unable to tolerate 160 mg twice daily. Specific modifications: For thrombocytopenia and/or neutropenia (ANC less than 1 x 10^9/L and/or PLT less than 50 x 10^9/L), withhold until resolved to ANC greater than or equal to 1 x 10^9/L and/or PLT greater than or equal to 50 x 10^9/L. If resolved within 2 weeks, resume at starting dose; if after more than 2 weeks, resume at reduced dose. For recurrent severe thrombocytopenia and/or neutropenia, withhold until resolved, then resume at reduced dose. For asymptomatic amylase and/or lipase elevation greater than 2 x ULN, withhold until resolved to less than 1.5 x ULN; if resolved, resume at reduced dose; if events reoccur at reduced dose, permanently discontinue; if not resolved, permanently discontinue. Perform diagnostic tests to exclude pancreatitis. For non-hematologic adverse reactions Grade 3 or higher, withhold until recovery to Grade 1 or less; if resolved, resume at reduced dose; if not resolved, permanently discontinue.

The efficacy of Scemblix in newly diagnosed Ph+ CML-CP was evaluated in the multicenter, randomized, active-controlled, open-label study ASC4FIRST (NCT04971226). A total of 405 patients were randomized 1:1 to receive Scemblix or investigator-selected tyrosine kinase inhibitors (IS-TKIs). Prior to randomization, the investigator selected the TKI (imatinib, nilotinib, dasatinib, or bosutinib) for the comparator arm based on patient characteristics and comorbidities. Patients were stratified according t... [label text truncated]. Detailed trial results are not available in the provided label text.

Warnings and precautions include thrombocytopenia and neutropenia, asymptomatic amylase and/or lipase elevation, and other non-hematologic adverse reactions. Contraindications are not reported in the provided label text