FDA Approves Asciminib for Treatment of 2 CML Subtypes


The FDA have granted an accelerated approval to acimibinib, which treats CML by binding to the ABL myristoyl pocket.

The FDA granted accelerated approval to asciminib (Scemblix) for the treatment of adult patients with Philadelphia chromosome–positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP) who have received prior treatment with 2 or more tyrosine kinase inhibitors (TKIs), as well as for the treatment of adult patients with Ph+ CML-CP and a T315I mutation. The novel agent, also known as a STAMP inhibitor, functions by binding to the ABL myristoyl pocket. The accelerated approval program dictates that continued approval is contingent upon verification and confirmatory evidence of clinical evidence.

“The introduction of TKIs 20 years ago revolutionized treatment for CML. However, there remain many patients who do not respond adequately to at least 2 available treatments and often experience challenging [adverse] effects [AEs] that add a burden to their daily lives,” Lee Greenberger, PhD, chief scientific officer of the Leukemia and Lymphoma Society, said in a press release. “The approval of Scemblix may offer hope to patients by addressing gaps in CML care.”

The approval was based on findings from the phase 2 ASCEMBL trial (NCT03106779), which showed that asciminib resulted in a major molecular response (MMR; 25%), double that of bosutinib (Bosulif; 13%) at 24 weeks (P = .029) in previously pretreated patients with Ph+ CML-CP. In addition, 7% of the 156 patients receiving asciminib needed to discontinue treatment because of AEs, compared with 25% in the bosutinib cohort.

Notable AEs from the trial included laboratory abnormalities, upper respiratory tract infections, musculoskeletal pain, a decrease in platelet and neutrophil counts, a decrease in hemoglobin level, and an increase in triglycerides, creatine kinase, and alanine aminotransferase levels.

“CML can be difficult to treat when currently available treatments fail patients, when treatment [adverse] effects cannot be tolerated, or sometimes both,” said Michael J. Mauro, MD, hematologist and leader of the Myeloproliferative Neoplasms Program at Memorial Sloan Kettering Cancer Center. “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.”


FDA approves Novartis Scemblix (asciminib), with novel mechanism of action for the treatment of chronic myeloid leukemia. News release. Novartis; October 29, 2021. Accessed October 29, 2021. https://bit.ly/3GoBu4k

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