Asciminib receives FDA priority review for newly diagnosed Ph+ CP-CML – Novartis

The FDA has granted priority review to asciminib (Scemblix) for the treatment of patients with newly diagnosed Philadelphia chromosome (Ph)–positive chronic myeloid leukemia (CML) in chronic phase (CP-CML).

The regulatory decision was supported by findings from the phase III ASC4FIRST study (NCT04971226), in which patients with Ph-positive CP-CML experienced superior major molecular response (MMR) rates with asciminib compared with the standard-of-care (SOC) TKIs nilotinib (Tasigna), imatinib (Gleevec), bosutinib (Bosulif), and dasatinib (Sprycel). Asciminib is the first treatment to demonstrate an efficacy advantage over SOC TKIs alongside a favorable safety and tolerability profile in CML.

Data presented at the 2024 ASCO Annual Meeting showed that, at a data cutoff of November 28, 2023, the 48-week MMR rate was 67.7% among patients who received asciminib (n = 201) vs 49.0% among patients treated with an investigator-selected TKI (n = 204), translating to a difference of 18.9% (95% CI, 9.6%-28.2%; P < .001). Notably, in the imatinib stratum of the investigational arm (n = 101) and comparator arm (n = 102), the 48-week MMR rates were 69.3% vs 40.2%, respectively, for a difference of 29.6% (95% CI, 16.9%-42.2%; P < .001).

In May 2024, asciminib was granted FDA breakthrough therapy designation for the treatment of adult patients with newly diagnosed Ph-positive CP-CML. The agent is currently under evaluation by the regulatory agency’s Real-Time Oncology Review program.

In October 2021,the FDA granted accelerated approval to asciminibfor this indication and concurrently approved asciminib for adult patients with Ph+ CP-CML harboring a BCR::ABL1T315I mutation. The agent is also approved by the European Medicines Agency and several other regulatory agencies for this patient population.