Novartis has secured accelerated approval from the US Food and Drug Administration (FDA) for Scemblix (asciminib) to treat adults with newly diagnosed chronic myeloid leukaemia (CML).
This approval applies to adults with Philadelphia chromosome-positive CML in the chronic phase (Ph+ CML-CP).
Asciminib received breakthrough therapy designation for newly diagnosed adult patients and was reviewed under the FDA’s Real-Time Oncology Review (RTOR) programme.
It is approved in over 75 countries for patients previously treated with two or more TKIs for Ph+ CML-CP. In the US, Scemblix is also approved for patients with the T315I mutation.
The latest expanded indication approval is based on the major molecular response rate (MMR) at week 48 from the Phase 3 ASC4FIRST trial. This study compared once-daily Scemblix to investigator-selected standard of care tyrosine kinase inhibitors (TKIs) like imatinib, nilotinib, dasatinib, and bosutinib.
According to the results, Scemblix showed superior MMR rates at week 48 compared to both the standard TKIs and imatinib alone. It demonstrated superior efficacy compared to all standard therapies, along with a favourable safety and tolerability profile.
Continued approval for this indication may depend on further confirmation of clinical benefits.
The expanded indication for asciminib in Ph+ CML-CP increases eligible patients by about four times, covering newly diagnosed and previously treated adults.
The ASC4FIRST trial is ongoing, with the next analysis scheduled for week 96 to assess the secondary endpoint of MMR at that time, along with additional secondary endpoints.
The approval is also supported by preliminary data from the Phase 2 ASC2ESCALATE study, which involves Ph+ CML-CP patients previously treated with one TKI who discontinued treatment.
Novartis US president Victor Bulto said: “With this approval, we can offer newly diagnosed adult Ph+ CML-CP patients a new treatment option that combines both, with the potential to change the trajectory of many more people living with CML.”
Currently, Scemblix is being studied across multiple treatment lines for Ph+ CML-CP, both as a monotherapy and in combination therapies.
Separately, Novartis reported that its net sales in Q3 2024 grew 10% year-over-year (YoY) to reach $12.8bn, driven by strong performance from several products. Core operating income of the Swiss firm increased 20% YoY to $5.1bn.
