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FDA Approves Novartis' Scemblix (asciminib) for First-Line CML Treatment

• The FDA granted accelerated approval to Novartis' Scemblix (asciminib) for newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML-CP). • The approval was based on the ASC4FIRST Phase III trial, demonstrating Scemblix's superiority over standard-of-care tyrosine kinase inhibitors (TKIs) in achieving major molecular response (MMR) at week 48. • Scemblix showed a favorable safety and tolerability profile compared to existing TKIs, potentially improving long-term disease control for CML patients. • The ASC4FIRST trial results indicated a statistically significant improvement in MMR rates with Scemblix compared to both standard TKIs and imatinib alone.

The FDA has granted accelerated approval to Novartis' Scemblix (asciminib) for adult patients newly diagnosed with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). This approval marks a significant advancement in first-line treatment options for CML patients, potentially offering improved efficacy and tolerability compared to existing therapies.
The decision was based on data from the Phase III ASC4FIRST trial, a head-to-head, multi-center, open-label, randomized study comparing oral Scemblix to first- or second-generation TKIs. The trial enrolled 405 patients and assessed the efficacy of Scemblix versus TKIs based on the proportion of patients achieving major molecular response (MMR) at week 48.

Superior Efficacy of Scemblix

Results from the ASC4FIRST trial demonstrated that 68% of patients treated with Scemblix achieved MMR at week 48, compared to 49% of patients receiving standard-of-care TKIs. Furthermore, when compared to imatinib alone, Scemblix achieved a 69% MMR rate versus 40% for imatinib. Scemblix also demonstrated a 41% rate of molecular responses by week 48, while the TKI group and imatinib alone group achieved 22% and 16%, respectively.

Improved Tolerability and Safety Profile

The safety profile of Scemblix was consistent with previous studies, with no new safety concerns identified. Common adverse events included musculoskeletal pain, rash, fatigue, upper respiratory tract infection, headache, abdominal pain, and diarrhea. This favorable tolerability profile is a crucial factor, as many CML patients may switch or discontinue treatment due to side effects.

Clinical Significance and Impact

"In the first-of-its-kind ASC4FIRST trial, Scemblix achieved impressive results across all three parameters of efficacy, safety and tolerability versus all standard of care TKIs. This Scemblix data has the potential to be practice-changing," said Jorge Cortes, MD, director, Georgia Cancer Center.
CML accounts for approximately 15% of all new leukemia cases in the United States. The American Cancer Society estimates that 9,280 new cases of CML will be diagnosed in 2024, with around 1,280 deaths. This approval offers a new treatment option that combines both efficacy and a favorable tolerability profile, potentially changing the trajectory for many individuals living with CML.

Novartis' Commitment to CML Treatment

"We are proud to help redefine CML treatment once again with Scemblix, as we continue to deliver on our 20+ year commitment to innovation and support in CML," said Victor Bulto, president, US, Novartis. "Despite many advances in the field, patients still need treatment options that are highly effective with a favorable tolerability profile to help enable them to achieve meaningful outcomes as they manage chronic conditions."
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Reference News

[1]
FDA Grants Accelerated Approval to Novartis' Scemblix for Newly Diagnosed Chromosome ...
pharmexec.com · Oct 30, 2024

The FDA granted accelerated approval to Novartis’ Scemblix for newly diagnosed Ph+ CML-CP, based on superior major molec...

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