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FDA Approves New Treatment Option for Philadelphia Chromosome-Positive Chronic Myeloid Leukemia

Rye Brook, N.Y., November 11, 2021 – The U.S. Food and Drug Administration (FDA) recently granted accelerated approval to asciminib (Scemblix®) for patients with Philadelphia chromosome-positive chronic myeloid leukemia chronic phase (Ph+ CML-CP) who did not tolerate or had inadequate response to treatment with tyrosine kinase inhibitors (TKIs). FDA also approved asciminib to treat adult patients with Ph+ CML-CP with the T315I mutation.


Asciminib works through a different mechanism than other drugs used to treat CML. This is promising for patients whose disease has become resistant to treatment with TKIs, which is a common occurrence in patients with T3151 mutations. It also provides a new option for patients who cannot tolerate treatment with TKIs.


According to Lee Greenberger, Chief Scientific Officer at The Leukemia & Lymphoma Society, “The introduction of TKIs 20 years ago revolutionized treatment for CML; however, there remain many patients who do not respond adequately to at least two available treatments and often experience challenging side effects that add a burden to their daily lives.”


Approval for Ph+ CML-CP without the T3151 mutation was based on findings from the ASCEMBL trial. The major molecular response (MMR) rate, a common measure used to determine how well a CML treatment is working, was 25% in asciminib-treated patients compared with 13% in patients receiving the TKI bosutinib (Bosulif®) after 24 weeks. The median duration of response to asciminib still had not been reached at a follow up on 20 months.


Approval for Ph+ CML-CP with the T3151 mutation was based on findings from the CABL001X2101 trial. MMR was achieved by 24 weeks in 42% of asciminib-treated patients and by 96 weeks in 49% of patients. The median duration of treatment was 108 weeks.


The most common side effect from asciminib, occurring in 20% or more of patients, were upper respiratory tract infections, musculoskeletal pain, fatigue, nausea, rash and diarrhea. The drug can also cause lab abnormalities in measures such as blood counts, cholesterol and liver enzymes.


The addition of asciminib to the CML treatment arsenal provides a novel approach to help treat this form of blood cancer, addressing gaps in current CML care.