FDA Grants Investigational Therapy Breakthrough Designation for Chronic Myeloid Leukemia

The Food and Drug Administration has granted the investigational therapy asciminib a breakthrough therapy designation for the treatment of certain adults with Philadelphia chromosome-positive chronic myeloid leukemia. The designation may allow for expedited review of the drug.

The Food and Drug Administration (FDA) recently granted breakthrough therapy designation for asciminib for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase, who have already received treatment with two or more tyrosine kinase inhibitors, according to the agent’s manufacturer, Novartis.

The drug also received the designation for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase whose disease harbors the T315I genetic mutation.

Although there have been strides over the past few decades in treating chronic myeloid leukemia, the manufacturer noted that patients who have received previous treatment tend to struggle to reach treatment goals.

The breakthrough therapy designations were based on data from two clinical trials. The phase 3 ASCEMBL trial demonstrated that, at 24 weeks, treatment with asciminib in previously treated patients with Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase resulted in an almost doubled major molecular response rate, compared to Bosulif (bosutinib).

The data, which were presented at a major scientific meeting in 2020, demonstrated that more patients who received study drug (157 patients) achieved a complete cytogenetic response than those who received Bosulif (76 patients) (40.8% vs. 24.2%).

“These important comparative data are impressive, and they reinforce the critical role asciminib may play, if approved, in overcoming the treatment challenges we face in later treatment lines of chronic-phase (chronic myeloid leukemia),” Dr. Michael J. Mauro, Myeloproliferative Neoplasms Program Leader at Memorial Sloan Kettering Cancer Center and professor at Weill Cornell Medicine in New York, said in a press release at the time the data were presented in December. “While the advent and expansion of TKI therapies has resulted in tremendous progress for patients living with (chronic myeloid leukemia) over the last decades, many of our patients in later treatment lines still face inadequate response, disease progression and intolerable side effects.”

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