Last week, the U.S. Food and Drug Administration (FDA) has announced the approval of Scemblix (asciminib) for two indications in the treatment of chronic myeloid leukemia (CML), according to the drug’s manufacturer Novartis.
The FDA granted accelerated approval of Scemblix for adult patients with Philadelphia chromosome-positive CML in chronic phase (Ph+ CML-CP) previously treated with two or more tyrosine kinase inhibitors (TKIs). The agency also granted full approval of the drug for adult patients with Ph+ CML-CP with the T315I mutation.
Also called STAMP inhibitor, Scemblix works by binding to the ABL myristoyl pocket, offering a ray of hope for patients who have been intolerant of current TKI therapies and/or have experienced resistance, according to the company.
Dr. Lee Greenberger, Chief Scientific Officer at The Leukemia & Lymphoma Society, said, “The introduction of TKIs twenty 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.”
“The approval of Scemblix may offer hope to patients by addressing gaps in CML care,” he added.
The FDA approved the drug considering positive results from the Phase III trial and a Phase I trial on patients with Ph+ CML-CP with T31RI mutation.
Dr. Michael Mauro of Memorial Sloan Kettering Cancer Center said, “CML can be difficult to treat when currently available treatments fail patients, when treatment side effects cannot be tolerated, or sometimes both.”
“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,” he noted.
In the United States, physicians can now prescribe Scemblix to qualified adult patients with CML.
The drug, which is available in a tablet form, is not recommended for pregnant women and nursing mothers. It is unclear whether Scemblix is safe and effective in children.
Novartis has begun regulatory filings for Scemblix in multiple nations and regions across the world.
