FDA prioritizes review for Novartis’ leukaemia treatment

The US Food and Drug Administration (FDA) has granted priority review status to Novartis' drug Scemblix (asciminib) for the treatment of newly diagnosed adult patients with Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase (Ph+ CML-CP). This designation is given to treatments that have the potential to significantly enhance the safety or effectiveness of therapies for serious or life-threatening conditions.

The priority review for Scemblix is founded on data from the Phase III ASC4FIRST clinical trial. This trial evaluated the efficacy, safety, and tolerability of Scemblix, administered once daily, against investigator-chosen tyrosine kinase inhibitors (TKIs). The TKIs used for comparison included imatinib, nilotinib, dasatinib, and bosutinib, which are considered standard treatments for newly diagnosed Ph+ CML-CP patients.

The ASC4FIRST study enrolled 405 adult patients. Scemblix exhibited superior major molecular response (MMR) rates at week 48 when compared to standard TKIs and imatinib alone. Additionally, Scemblix demonstrated enhanced efficacy and a more favorable safety and tolerability profile compared to imatinib and second-generation TKIs. The treatment was associated with fewer grade ≥3 adverse events, fewer dose adjustments, and had half the rate of adverse events leading to treatment discontinuation. Importantly, the safety profile of Scemblix in newly diagnosed patients was consistent with previous registration studies, with no new safety issues observed.

Scemblix has already received approval from the FDA, the European Medicines Agency, and other regulatory bodies for use in adults with Ph+ CML-CP who have previously been treated with two or more TKIs. Rodney Gillespie, Novartis US oncology therapeutic area head and senior vice-president, emphasized the FDA's decision to grant priority review and breakthrough therapy designations to Scemblix for newly diagnosed CML patients. He highlighted the need for additional effective, safe, and tolerable treatment options. According to Gillespie, the ASC4FIRST data suggest that, if approved, Scemblix could meet a critical need in CML treatment by offering a highly effective therapy with a favorable safety and tolerability profile.

In related news, Novartis reported a substantial 43% increase in net income from continuing operations, reaching $3.24 billion in the second quarter of 2024, up from $2.27 billion during the same period in 2023.