FDA Approval Summary: Asciminib for Ph+ CML in Chronic Phase Treated with Two or More Tyrosine Kinase Inhibitors and for the T315I Mutation.

Autor:	Pamuk GE; Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland., Chow ECY; Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland., Ionan AC; Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland., Chen H; Office of Biostatistics, Office of Translational Science, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland., Lee SL; Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland., Hsu V; Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland., Grimstein M; Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland., Zheng N; Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland., Sun J; Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland., Charlab R; Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland., Gehrke BJ; Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland., Vallejo J; Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland., Ehrlich LA; Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland., de Claro RA; Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland., Theoret MR; Oncology Center of Excellence, U.S. Food and Drug Administration, Silver Spring, Maryland.
Jazyk:	angličtina
Zdroj:	Clinical cancer research : an official journal of the American Association for Cancer Research [Clin Cancer Res] 2024 Oct 01; Vol. 30 (19), pp. 4266-4271.
DOI:	10.1158/1078-0432.CCR-24-1086
Abstrakt:	On October 29, 2021, FDA granted accelerated approval to asciminib (SCEMBLIX; Novartis), a tyrosine kinase inhibitor (TKI), for the treatment of adult patients with Philadelphia chromosome positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP), previously treated with two or more TKIs, and granted traditional approval to asciminib for adult patients with Ph+ CML in CP with the T315I mutation. The first indication was approved based on major molecular response (MMR) at 24 weeks in the ASCEMBL study, a randomized trial comparing asciminib with bosutinib in patients who had failed two or more TKIs. This indication was ultimately granted traditional approval on October 12, 2022, based on safety data and MMR rate at 96 weeks of 38% [95% confidence interval (CI), 30-46] in the asciminib arm versus 16% (95% CI, 8-26) in the bosutinib arm (P value: 0.001). The second indication was approved based on MMR rate by 96 weeks of 49% (95% CI, 34-64) in the single-arm CABL001X2101 study. The most common (≥20%) adverse reactions included upper respiratory tract infections, musculoskeletal pain, headache, fatigue, nausea, rash, and diarrhea. The most common (≥20%) laboratory abnormalities were thrombocytopenia, neutropenia, anemia, lymphopenia, hypertriglyceridemia, hyperuricemia, and increases in creatine kinase, alanine aminotransferase, aspartate aminotransferase, lipase, and amylase. This manuscript describes the basis for approval of these indications. (©2024 American Association for Cancer Research.)
Databáze:	MEDLINE
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