Swiss pharmaceutical giant Novartis (NYSE: NVS) announced that its drug Scemblix (asciminib) has received accelerated approval from the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP).
This accelerated approval is supported by the major molecular response rate (MMR) at week 48 from the ASC4FIRST Phase III trial, which compared once-daily Scemblix to other investigator-selected standard of care (SoC) tyrosine kinase inhibitors (TKIs), including imatinib, nilotinib, dasatinib, and bosutinib. The results demonstrated that Scemblix achieved superior MMR rates at week 48 for both primary endpoints compared to SoC TKIs, including imatinib alone.
Distinctively, Scemblix is the first CML treatment specifically designed to target the ABL Myristoyl Pocket, classified as a STAMP inhibitor in scientific literature. Unlike existing TKIs that target the ATP-binding site, Scemblix represents the world’s only STAMP inhibitor. It has been granted accelerated approval for newly diagnosed adult patients and also holds approval for previously treated adults with Ph+ CML-CP. The expanded indication significantly increases the eligible patient population for Scemblix by approximately fourfold, encompassing both newly diagnosed and previously treated adults.- Flcube.com
