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FDA Approves Scemblix for Pretreated CML in the Chronic Phase


The Food and Drug Administration approved Scemblix for patients with Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase whose disease progressed on prior TKI treatment.

The Food and Drug Administration (FDA) granted an accelerated approval to Scemblix (asciminib) for the treatment of patients with Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase (Ph+ CML-CP) who are not tolerant or have had an inadequate response to two or more tyrosine kinase inhibitors (TKIs). The drug was also granted a full approval for adults with Ph+ CML-CP with a T3151 mutation, according to Novartis, the manufacturer of the agent.

Scemblix is unlike any other CML drug because it binds to the ABL myristoyl pocket, which is promising for disease that has become resistant to TKIs, a common occurrence for patients with T3151 mutations.

“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,” said Lee Greenberger, Chief Scientific Officer at The Leukemia & Lymphoma Society, in a statement. “The approval of Scemblix may offer hope to patients by addressing gaps in CML care.”

The approval is based on findings from the phase 3 ASCEMBL trial, where Scemblix led to improvements in major molecular response (MMR) compared to Bosulif (bosutinib) at 24 weeks, at 25% and 13% MMR for Scemblix and Bosulif, respectively. The drug also had far lower discontinuation rates from side effects (7% versus 25%).

The most common side effect from Scemblix occurring in 20% or more of patients were upper respiratory tract infections and musculoskeletal pain. As far as laboratory abnormalities, common side effects were a decrease in platelets and neutrophil counts, decreased hemoglobin and increases in 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,” expressed Dr. Michael J. Mauro, hematologist and Myeloproliferative Neoplasms Program Leader at Memorial Sloan Kettering Cancer Center, in the statement.

The continued approval of Scemblix for Ph+ CML-CP is dependent on data from further investigations.

“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,” Mauro said.

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