FDA Approves Novartis’ Scemblix for Treatment of Chronic Myeloid Leukemia

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Novartis’ scemblix (asciminib) is designed to provide a single treatment option to patients struggling with chronic myeloid leukemia.

Novartis announced on Oct. 29, 2021, that FDA has approved Scemblix (asciminib) for the treatment of chronic myeloid leukemia (CML) in two key indications. FDA granted asciminib accelerated approval for adult patients with Philadelphia chromosome-positive CML in chronic phase (Ph+ CML-CP) previously treated with two or more tyrosine kinase inhibitors (TKIs) and full approval for adult patients with Ph+ CML-CP with the T315I mutation.

According to a company press release, many patients with CML experience resistance and/or intolerance to currently available TKI therapies, especially those who’ve undergone multiple therapies or who have the t3151 mutation. Asciminib is the first FDA approved CML treatment that binds to the ABL myristoyl pocket, a novel mechanism of action intended to help the set of patients who resist traditional TKI therapies.

Approval was made following the Phase III ASCEMBL trial, which looked at major molecular response (MMR) rates in patients treated with asciminib versus Bosulif (bosutinib). At week 24, asciminib (25%) nearly doubled the MMR rate of bosutinib (13%). Additionally, the proportion of patients who discontinued treatment due to adverse reactions was more than three times lower in the asciminib v (7%) patients than those given bosutinib (25%).


“CML can be difficult to treat when currently available treatments fail patients, when treatment side effects cannot be tolerated, or sometimes both,” expressed Michael J. Mauro, hematologist and myeloproliferative neoplasms program leader at Memorial Sloan Kettering Cancer Center, 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.”

Source: Novartis