Novartis announced that the US Food and Drug Administration (FDA) has granted Priority Review status to Scemblix® (asciminib) for the treatment of newly diagnosed adult patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP).
The FDA’s Priority Review is given to medicines that address serious or life-threatening conditions and could significantly improve treatment safety or efficacy. Scemblix also received Breakthrough Therapy designation for this use and is being reviewed under the FDA’s Real-Time Oncology Review (RTOR) program. Previously, Scemblix had received Priority Review and Breakthrough Therapy designations for treating adult patients with Ph+ CML-CP who had been treated with two or more tyrosine kinase inhibitors (TKIs).
Rodney Gillespie, Senior Vice President, Therapeutic Area Head, US Oncology, Novartis, stated, “We are pleased with the FDA’s Priority Review and Breakthrough Therapy designations for Scemblix in newly diagnosed CML patients, highlighting the need for more effective, safe, and tolerable treatments. The ASC4FIRST study data suggest that Scemblix, if approved, could fill a critical gap in CML treatment with its strong efficacy and favorable safety profile.”
The Priority Review is based on results from the ASC4FIRST Phase III study, which compared the efficacy, safety, and tolerability of once-daily Scemblix against investigator-selected TKIs (imatinib, nilotinib, dasatinib, and bosutinib) in newly diagnosed adult patients with Ph+ CML-CP. Scemblix demonstrated superior major molecular response (MMR) rates at week 48 compared to these standard-of-care TKIs (68% vs. 49.0%) and imatinib alone (69% vs. 40%). Scemblix is the first CML treatment to show superior efficacy with a favorable safety and tolerability profile compared to both imatinib and second-generation TKIs, with fewer grade ≥3 adverse events, fewer dose adjustments, and a lower rate of treatment discontinuation.
Earlier this year, the ASC4FIRST data were presented as a late-breaking abstract at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting and as a plenary at the European Hematology Association (EHA) 2024 Congress, and were published in The New England Journal of Medicine.
Scemblix is already approved by the FDA, European Medicines Agency, and other regulatory authorities for treating adult patients with Ph+ CML-CP who have been treated previously with two or more TKIs.
About the ASC4FIRST Phase III Clinical Trial
ASC4FIRST (NCT04971226) is a Phase III, multi-center, open-label, randomized study comparing oral Scemblix® 80 mg QD with investigator-selected first- or second-generation TKIs (imatinib, nilotinib, dasatinib, or bosutinib) in 405 adult patients with newly diagnosed Ph+ CML-CP. The primary endpoints are to compare the efficacy of asciminib with investigator-selected TKIs and to compare efficacy against imatinib, based on the proportion of patients achieving MMR at week 48.
The study, which is ongoing, includes secondary endpoints such as MMR at week 96 and safety, measured by treatment discontinuation due to adverse events (AEs) by week 96. Additional secondary endpoints include MMR, MR4, MR4.5, complete hematological response (CHR), BCR::ABL1 ≤1%, time to first MMR, MR4 and MR4.5, time to treatment failure, and various survival measures.
About Scemblix® (asciminib)
Scemblix® is the first CML treatment targeting the ABL Myristoyl Pocket (STAMP inhibitor). Current CML treatments are ATP-competitive TKIs. Scemblix is approved in over 70 countries, including the US, EU, and Japan, for adult patients with Ph+ CML-CP previously treated with two or more TKIs. In the US and some other countries, it is also approved for patients with Ph+ CML-CP with the T315I mutation.
Scemblix is an important treatment option for patients who develop resistance or intolerance after two prior TKI therapies and is being studied across multiple treatment lines for Ph+ CML-CP, both as a monotherapy and in combination.
About Novartis’ Commitment to CML
Novartis has a long-standing commitment to advancing treatment for CML. Over the past two decades, their research has helped transform CML from a life-threatening disease to a manageable chronic condition. Despite these advancements, challenges remain, and Novartis continues to explore innovative treatments to address unmet needs in CML. Their efforts include a long-term partnership with the Max Foundation, providing access to medications like Gleevec (imatinib), Tasigna (nilotinib), and now Scemblix, supporting over 100,000 patients in low- and middle-income countries.
