Novartis Receives FDA Approval for Scemblix as First-Line Treatment for CML

Novartis today announced that the FDA has approved Scemblix (asciminib) for the treatment of adults with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). The approval was based on results from the Phase 3 ASC4FIRST trial, which demonstrated superior major molecular response rates compared to standard-of-care TKIs. The MMR rate at 48 weeks was 67.7% for Scemblix versus 49.0% for standard-of-care TKIs. Scemblix is a STAMP inhibitor that specifically targets the ABL myristoyl pocket, offering a differentiated mechanism of action from ATP-competitive TKIs. This approval establishes Scemblix as a new first-line treatment option for CML.