FDA approves Scemblix for certain patients with chronic myeloid leukemia
The FDA approved asciminib for treatment of certain patients with chronic myeloid leukemia, according to a press release from the agent’s manufacturer.
The agency granted accelerated approval to asciminib (Scemblix, Novartis) for adults with Philadelphia chromosome-positive CML in chronic phase who received two on more tyrosine kinase inhibitors. The agent also received full approval for adults with Ph+CML in chronic phase that harbors T315I mutations.
Asciminib is known as a STAMP inhibitor because it functions by binding to the ABL myristoyl pocket — a new mechanism of action.
The FDA based the approval on results of two trials: a phase 1 trial that included patients with Ph+CML, some of whom harbored T315I mutations, and the randomized phase 3 ASCEMBL trial, which included patients with Ph+CML in chronic phase who had been treated with two or more TKIs.
Results of the ASCEMBL trial, previously reported by Healio, showed a higher percentage of patients assigned asciminib vs. bosutinib (Bosulif, Pfizer) achieved major molecular response at 24 weeks (25% vs. 13%; P = .029). In addition, a higher proportion of patients in the bosutinib group discontinued treatment due to adverse events (25% vs. 7%).
The most common adverse events in the asciminib group were upper respiratory tract infections and musculoskeletal pain; decreased platelet and neutrophil counts, decreased hemoglobin; and increased triglycerides, creatine kinase and alanine aminotransferase.
“CML can be difficult to treat when currently available treatments fail patients, when treatment side effects cannot be tolerated, or sometimes both,” Michael J. Mauro, MD, hematologist and leader of the myeloproliferative neoplasms program at Memorial Sloan Kettering Cancer Center, said in the press release. “The addition of Scemblix into the CML treatment landscape gives us a novel approach to combat this blood cancer, helping address clinical challenges in patients struggling after switching to a second treatment, as well as in patients who develop the T315I mutation and face significantly worse outcomes.”
Asciminib is being investigated across multiple treatment lines for CML in chronic phase, including as first-line treatment in the phase III ASC4FIRST study, according to the press release.