Novartis Scemblix granted FDA Priority Review for the treatment of adults with newly diagnosed CML

Novartis announced that Scemblix (asciminib) has been granted Priority Review status by the US Food and Drug Administration (FDA) for treatment of newly diagnosed adult patients with Philadelphia chromosome-positive CML in chronic phase (Ph+ CML-CP).

The FDA grants Priority Review to medicines that address serious or life-threatening diseases or conditions and, if approved, would provide significant improvements in treatment safety or efficacy. Scemblix previously received Breakthrough Therapy designation for the treatment of newly diagnosed adult patients and is currently being reviewed under the FDA’s Real-Time Oncology Review (RTOR) program. Scemblix received Priority Review and Breakthrough Therapy designations at the time of the original new drug application for the treatment of adult patients with Ph+ CML-CP who have previously been treated with two or more TKIs.

“We welcome the FDA’s decision to grant Priority Review and Breakthrough Therapy designations to Scemblix for newly diagnosed CML patients, which underscores the substantial need for additional effective, safe and tolerable treatment options,” said Rodney Gillespie, Senior Vice President, Therapeutic Area Head, US Oncology, Novartis. “The ASC4FIRST data indicate that Scemblix, if approved, has the potential to address a critical gap in CML by offering a highly effective treatment along with a favorable safety and tolerability profile.”

Priority Review designation is based on results from ASC4FIRST, a Phase III study that evaluated the efficacy, tolerability and safety of once daily Scemblix against investigator-selected (IS) tyrosine kinase inhibitors (TKIs) (imatinib, nilotinib, dasatinib, and bosutinib) representing the current standard of care (SoC) in newly diagnosed adult patients with Ph+ CML-CP. Scemblix demonstrated superior major molecular response (MMR) rates in both primary endpoints at week 48 vs. IS SoC TKIs (68% vs. 49.0%) and imatinib alone (69% vs. 40%). Additionally, Scemblix is the first CML treatment to show superior efficacy along with a favorable safety and tolerability profile vs. imatinib and 2G TKIs, with fewer grade ≥3 AEs (38% vs. 44% and 55%), dose adjustments (30% vs. 39% and 53%) and half the rate of AEs leading to treatment discontinuation (5% vs. 11% and 10%). In newly diagnosed patients, the safety profile was consistent with previous registration studies, with no new safety concerns observed.

Earlier this year, ASC4FIRST data were presented as a late-breaking abstract at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting, as a plenary at the European Hematology Association (EHA) 2024 Congress and published in The New England Journal of Medicine.

Scemblix is currently approved by the FDA, European Medicines Agency and other regulatory authorities for adult patients with Ph+ CML-CP who have been treated previously with two or more TKIs.