The US Food and Drug Administration (FDA) has granted priority review designation for Novartis’ Scemblix (asciminib) to treat newly diagnosed adult patients with Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase (Ph+ CML-CP).
This status is awarded to potential treatments that could significantly improve the safety or efficacy of therapies for serious or life-threatening conditions.
The priority review for Scemblix is based on data from the Phase III ASC4FIRST clinical trial.
This trial assessed the efficacy, tolerability and safety of Scemblix, administered once daily, compared to investigator-selected tyrosine kinase inhibitors (TKIs), which include imatinib, nilotinib, dasatinib and bosutinib and are considered the standard of care for newly diagnosed Ph+ CML-CP patients.
In the ASC4FIRST study, which enrolled 405 adult patients, Scemblix demonstrated superior major molecular response (MMR) rates at week 48 versus the standard of care TKIs and imatinib alone.
Scemblix also showed superior efficacy along with a more favourable safety and tolerability profile versus imatinib and second-generation TKIs.
The treatment also exhibited fewer grade ≥3 adverse events and dose adjustments and half the rate of adverse events leading to treatment discontinuation.
The safety profile of Scemblix in newly diagnosed patients aligns with that observed in previous registration studies, with no new safety concerns reported.
Scemblix is already approved by the FDA, the European Medicines Agency and other regulatory authorities for use in adults with Ph+ CML-CP who have been treated previously with two or more TKIs.
Novartis US oncology therapeutic area head and senior vice-president Rodney Gillespie stated: “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.
“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 favourable safety and tolerability profile.”
The company posted a significant 43% increase in net income from continuing operations, reaching $3.24bn in the second quarter (Q2) of 2024, up from $2.27bn in the same period of 2023.
