FDA Approves Novel Therapy for Chronic Myeloid Leukemia

Novartis’ Scemblix was approved for previously treated patients and for those who are resistant to TKI inhibitors.

The FDA has approved Novartis’ Scemblix (asciminib) to treat patients with chronic myeloid leukemia (CML). The FDA issued accelerated approval for adults with Philadelphia chromosome-positive CML in the chronic phase and full approval for adults with Philadelphia chromosome-positive CML with the T315I mutation.

Scemblix represents an important development for patients who experience resistance and/or intolerance to currently available tyrosine kinase inhibitor (TKI) therapies. This is a first-in-class therapy of a STAMP (specifically targeting ABL myristoyl pocket) inhibitor. The BCR-ABL1 gene is associated with the overproduction of leukemia cells.

Resistance to TKI therapies have been seen, driven by the T315I mutation.

“The introduction of TKIs 20 years ago revolutionized treatment for CML; however, there remain many patients who do not respond adequately to at least two available treatments and often experience challenging side effects that add a burden to their daily lives,” Lee Greenberger, Ph.D., chief scientific officer at The Leukemia & Lymphoma Society, said in a statement.

The approval of Scemblix is based on the results from the phase 3 ASCEMBLE trial, as well as a phase 1 trial that included patients with the T315I mutation. In patients with the Philadelphia chromosome-positive CML in the chronic phase, Scemblix nearly doubled the major molecular response (MMR) rate at 24 weeks. The most common adverse reactions and laboratory abnormalities in the Scemblix arm were: upper respiratory tract infections and musculoskeletal pain; decrease in platelet and neutrophil counts, decrease in hemoglobin; increase in triglycerides, creatine kinase and alanine aminotransferase.