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FDA Grants Accelerated Approval to Novartis' Scemblix for Newly Diagnosed CML

• The FDA has granted accelerated approval to Scemblix (asciminib) for adults with newly diagnosed Philadelphia chromosome-positive CML in the chronic phase (Ph+ CML-CP). • The approval expands Scemblix's eligible patient population by approximately four times, offering a new treatment option for those with CML. • Scemblix demonstrated superior major molecular response rates compared to tyrosine kinase inhibitors (TKIs) in the ASC4FIRST trial, supporting the FDA's decision. • Continued approval may be contingent upon verification of clinical benefit from confirmatory evidence, as the ASC4FIRST trial remains ongoing.

Novartis' Scemblix (asciminib) has received accelerated approval from the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase (Ph+ CML-CP). This approval significantly broadens the drug's availability, potentially impacting a larger segment of the CML patient population.
The FDA's decision was primarily based on the positive outcomes of the Phase 3 ASC4FIRST trial. The trial demonstrated that Scemblix achieved superior major molecular response rates at week 48 compared to investigator-chosen tyrosine kinase inhibitors (TKIs), including imatinib. These results highlight Scemblix's potential as a first-line treatment option.

Clinical Efficacy and Safety

The ASC4FIRST trial evaluated Scemblix against standard TKI therapies. The primary endpoint, major molecular response at week 48, was significantly in favor of Scemblix. While detailed safety data is forthcoming, Novartis emphasizes that Scemblix is designed to be highly specific, minimizing off-target kinase-mediated effects, potentially leading to a more favorable tolerability profile compared to traditional TKIs.
Victor Bulto, president US, Novartis, stated, "With this approval, we can offer newly diagnosed adult Ph-positive CML-CP patients a new treatment option that combines both [high effectiveness and favorable tolerability], with the potential to change the trajectory of many more people living with CML."

CML Disease Burden and Current Treatment Landscape

Approximately 9,280 new cases of CML are expected to be diagnosed in the U.S. this year. The Philadelphia chromosome abnormality is present in the majority of these patients, driving the proliferation of malignant white blood cells. While TKIs have revolutionized CML treatment, transforming it into a chronic condition, challenges related to efficacy and safety persist. Many newly diagnosed patients do not achieve optimal molecular responses, and intolerance to TKIs often leads to treatment discontinuation or changes.

Mechanism of Action and Prior Approvals

Scemblix functions as a STAMP inhibitor. It is already approved in over 75 countries for Ph+ CML-CP patients who have been previously treated with two or more TKIs. This new approval expands its use to the first-line setting.

Regulatory Considerations

As an accelerated approval, continued approval for Scemblix in this newly diagnosed indication is contingent upon verification and description of clinical benefit from confirmatory evidence. The ASC4FIRST trial is ongoing, with the next analysis scheduled for week 96.
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Reference News

[1]
Novartis' Scemblix granted FDA accelerated approval in newly diagnosed CML - PMLiVE
pmlive.com · Nov 1, 2024

Novartis' Scemblix (asciminib) granted accelerated FDA approval for newly diagnosed Philadelphia chromosome-positive CML...

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