Novartis’ Scemblix (asciminib) has been granted accelerated approval by the US Food and Drug Administration (FDA) to treat a new subset of chronic myeloid leukaemia (CML) patients.
The STAMP inhibitor has been authorised to treat adults with newly diagnosed Philadelphia chromosome-positive CML in the chronic phase (Ph-positive CML-CP), increasing the population eligible for the drug by approximately four times.
The FDA’s decision was supported by positive results from the late-stage ASC4FIRST trial, in which Scemblix demonstrated superior major molecular response rates in both primary endpoints at week 48 versus investigators’ choice of tyrosine kinase inhibitor (TKI) treatment and imatinib alone.
Approximately 9,280 new cases of CML, a type of cancer that starts in certain blood-forming cells of the bone marrow, are expected to be diagnosed in the US this year. The majority of patients have an abnormality known as the Philadelphia chromosome, which causes malignant white blood cells to proliferate.
While TKIs have transformed CML into a chronic disease, Novartis outlined that efficacy and safety challenges associated with the drugs continue to impact long-term treatment success, with many of those with newly diagnosed disease not meeting molecular response goals and many discontinuing or changing treatment due to intolerance.
Novartis’ Scemblix, which is already approved in more than 75 countries to treat Ph-positive CML-CP in patients who have previously been treated with two or more TKIs, is designed to be highly specific and minimise off-target kinase mediated effects.
Victor Bulto, president US, Novartis, said: “Despite many advances in the field, patients still need treatment options that are highly effective with a favourable tolerability profile to help enable them to achieve meaningful outcomes as they manage chronic conditions.
“With this approval, we can offer newly diagnosed adult Ph-positive CML-CP patients a new treatment option that combines both, with the potential to change the trajectory of many more people living with CML.”
In line with the FDA’s accelerated approvals pathway, continued approval for Scemblix in the newly diagnosed indication may be contingent upon verification and description of clinical benefit from confirmatory evidence. The ASC4FIRST trial remains ongoing, with the next analysis scheduled for week 96.