FDA Approves Novartis Scemblix® for Newly Diagnosed CML with Superior Efficacy and Safety

Novartis has announced that Scemblix® (asciminib) has received accelerated approval from the US Food and Drug Administration (FDA) for treating adults with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). This approval is based on a Phase III clinical trial (ASC4FIRST) showing that Scemblix outperformed all other standard of care (SoC) tyrosine kinase inhibitors (TKIs), including imatinib, nilotinib, dasatinib, and bosutinib, in achieving major molecular response rates (MMR) at week 48.

Scemblix demonstrated superior efficacy and a favorable safety profile compared to existing treatments. The trial results indicated that nearly 20% more patients treated with Scemblix achieved MMR compared to those on other SoC TKIs. Additionally, Scemblix was associated with fewer adverse reactions leading to treatment discontinuation, fewer dose reductions, and lower rates of treatment-related grade 3 or higher adverse reactions.

Dr. Lee Greenberger, Chief Scientific Officer at The Leukemia & Lymphoma Society, highlighted the significance of this approval, stressing the importance of having effective first-line treatment options for patients to achieve better long-term disease control with fewer side effects. Despite the transformation of CML into a manageable condition thanks to TKIs, a significant number of patients still fail to meet efficacy milestones or discontinue treatment within the first year due to intolerance.

Dr. Jorge Cortes, Director at Georgia Cancer Center, emphasized the practice-changing potential of Scemblix, given that it addresses both efficacy and tolerability challenges. The ASC4FIRST trial's first-of-its-kind design demonstrated Scemblix's impressive outcomes across these crucial parameters, making it a promising new option for clinicians and patients.

The expanded indication for Scemblix now includes both newly diagnosed and previously treated adults, significantly increasing the eligible patient population. This broader approval enables access to a treatment that combines superior efficacy with a favorable safety profile.

Victor Bulto, President of Novartis US, expressed pride in the company’s ongoing commitment to innovation in CML treatment. He noted that while there have been many advances in the field, there remains a need for treatments that are both highly effective and well-tolerated. Scemblix represents a significant step toward meeting this need, offering newly diagnosed patients a new treatment option that may positively impact their disease trajectory.

The ASC4FIRST trial will continue to monitor patients, with the next analysis scheduled at week 96 to evaluate further endpoints, including MMR at week 96 and additional secondary outcomes. Preliminary data from the Phase II ASC2ESCALATE study, which includes previously treated patients, also supported the FDA approval of Scemblix.

Scemblix is a first-in-class treatment that targets the ABL Myristoyl Pocket, distinguishing it from other TKIs that target the ATP-binding site. This novel mechanism of action has proven effective in both newly diagnosed and previously treated patients with Ph+ CML-CP. Scemblix has received Breakthrough Therapy designation and was reviewed under the FDA's Real-Time Oncology Review program. It is approved in over 75 countries, including the EU, for patients previously treated with two or more TKIs, and in some countries, for those with the T315I mutation.

Novartis continues to study Scemblix in various treatment lines for Ph+ CML-CP, both as a monotherapy and in combination with other treatments. The company remains dedicated to addressing unmet patient needs and reducing barriers to access and affordability, ensuring that more patients can benefit from innovative treatments. Novartis' long-standing collaboration with the Max Foundation has significantly impacted patients in low- and middle-income countries, providing access to life-changing medications.

This development marks another milestone in Novartis' two-decade commitment to transforming CML treatment and improving patient outcomes.