FDA D.I.S.C.O. Burst Edition: FDA approval of Scemblix (asciminib) for patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase, previously treated with tyrosine kinase inhibitors, and for adult patients in chronic phase with the T315I mutation
Welcome back to the D.I.S.C.O., FDA’s Drug Information Soundcast in Clinical Oncology, Burst Edition, brought to you by FDA’s Division of Drug Information in partnership with FDA’s Oncology Center of Excellence. Today we’ll provide a quick update on a recent FDA cancer drug approval.
On October 29, 2021, the FDA granted accelerated approval to asciminib (brand name Scemblix) for patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase, previously treated with two or more tyrosine kinase inhibitors, and approved asciminib for adult patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase with the T315I mutation.
The accelerated approval was based on results of a multi-center, randomized, active-controlled, open-label clinical trial, evaluating asciminib in patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase, previously treated with two or more tyrosine kinase inhibitors. A total of 233 patients were randomized 2:1 and stratified according to major cytogenetic response status to receive either asciminib 40 mg twice daily or bosutinib 500 mg once daily. Patients continued treatment until unacceptable toxicity or treatment failure occurred. The main efficacy outcome measure was major molecular response at 24 weeks. The major molecular response rate was 25% in patients treated with asciminib compared with 13% in those receiving bosutinib. With a median duration of follow-up of 20 months, the median duration of major molecular response has not yet been reached.
The regular approval for asciminib for patients with Philadelphia chromosome positive chronic myeloid leukemia in chronic phase with the T315I mutation was based on a multicenter, open-label clinical trial enrolling 45 patients who received asciminib 200 mg twice daily. Patients continued treatment until unacceptable toxicity or treatment failure occurred. The main efficacy outcome measure was major molecular response. Major molecular response was achieved by 24 weeks in 42% of the patients. Major molecular response was achieved by 96 weeks in 49% of the patients. The median duration of treatment was 108 weeks.
The most common adverse reactions reported in more than 20% of patients were upper respiratory tract infections, musculoskeletal pain, fatigue, nausea, rash, and diarrhea. The most common laboratory abnormalities were decreased platelet counts, increased triglycerides, decreased neutrophil counts and hemoglobin, and increased creatine kinase, alanine aminotransferase, lipase, and amylase.
This review used the Real-Time Oncology Review pilot program, which streamlined data submission prior to the filing of the entire clinical application, and the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment. The FDA approved this application 4 months ahead of the FDA goal date.
Full prescribing information for these approvals can be found on the web at www.fda.gov/drugsatFDA.
Health care professionals should report serious adverse events to FDA’s MedWatch Reporting Program at www.fda.gov/medwatch.
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