Novartis’ Scemblix® Gains FDA Approval for Newly Diagnosed Chronic Myeloid Leukemia (CML)
Wednesday, October 30, 2024
Novartis has announced that Scemblix® (asciminib) received accelerated approval from the US Food and Drug Administration (FDA) for use in adult patients newly diagnosed with Philadelphia chromosome-positive chronic myeloid leukaemia in chronic phase (Ph+ CML-CP).
CML treatment has advanced significantly with TKIs, yet many patients still struggle to achieve molecular response targets or may discontinue treatment due to adverse effects.
Approximately half of CML patients do not reach MMR milestones, and nearly a quarter stop or switch treatment within the first year. Scemblix addresses these challenges, providing enhanced efficacy and tolerability that may support better long-term treatment outcomes.
This accelerated approval stems from findings in the ASC4FIRST Phase III trial, which compared once-daily Scemblix with investigator-selected standard of care tyrosine kinase inhibitors (TKIs)—namely, imatinib, nilotinib, dasatinib, and bosutinib.
Results showed Scemblix achieved superior rates of major molecular response (MMR) at week 48, surpassing both the full set of standard TKIs and imatinib alone. Continued approval for this indication may rely on confirmation of clinical benefits from ongoing evidence.
With this expanded indication, the eligible patient population for Scemblix now includes newly diagnosed and previously treated adults, broadening treatment options.
Notably, newly diagnosed patients can access a treatment demonstrating improved efficacy and a favourable safety profile compared to other standard therapies.
Scemblix’s approval was primarily based on data from the ASC4FIRST Phase III trial, where almost 20% more patients on Scemblix achieved MMR compared to standard TKIs, with nearly 30% more achieving MMR compared to imatinib alone.
It also showed fewer high-grade adverse reactions, dose reductions, and treatment discontinuations compared to both imatinib and other second-generation TKIs. In addition, Scemblix achieved deeper molecular responses, with 41% of patients reaching MR4 compared to standard TKIs (22%) and imatinib alone (16%).
In line with previous registration studies, Scemblix’s safety profile in newly diagnosed patients remained consistent, showing no new safety concerns. Common adverse reactions included musculoskeletal pain, rash, fatigue, respiratory infections, headache, and gastrointestinal symptoms.
Source: novartis.com
