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FDA Grants Accelerated Approval to Frontline Asciminib for Patients With Ph+ Chronic Myeloid Leukemia
On October 29, 2024, the US Food and Drug Administration (FDA) granted accelerated approval to asciminib for adult patients with newly diagnosed Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) in the chronic phase (CP).
This approval was based on efficacy results from ASC4FIRST, a multicenter, randomized, active-controlled, open-label trial. A total of 405 patients were randomized on a 1-to-1 basis to receive either asciminib or investigator-selected tyrosine kinase inhibitors (IS-TKIs) including imatinib, nilotinib, dasatinib, or bosutinib.
The main efficacy outcome measure was major molecular response (MMR) rate at 48 weeks. The MMR rate at 48 weeks was 68% (95% confidence interval [CI], 61 to 74) in the asciminib cohort and 49% (95% CI, 42 to 56) in the IS-TKIs arm (difference 19% [95% CI, 10 to 28], P < 0.001). Within the imatinib branch, the MMR rate was 69% (95% CI, 59 to 78) in the asciminib arm and 40% (95% CI, 31 to 50) in the IS-TKIs arm (difference 30% [95% CI, 17 to 42], P < 0.001).
A pooled safety population analysis among patients with newly diagnosed and previously treated Ph+ CML in CP showed the most common adverse reactions (≥ 20%) were musculoskeletal pain, rash, fatigue, upper respiratory tract infection, headache, abdominal pain, and diarrhea. The most common laboratory abnormalities (≥ 40%) among patients with newly diagnosed Ph+ CML in CP were decreased lymphocyte count, decreased leukocyte count, decreased platelet count, decreased neutrophil count, and decreased calcium corrected.
Source:
FDA grants accelerated approval to asciminib for newly diagnosed chronic myeloid leukemia. US Food and Drug Administration. Published online October 29, 2024. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-asciminib-newly-diagnosed-chronic-myeloid-leukemia
