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 approved to asciminib (Scemblix) for the treatment of adult patients with Philadelphia chromosome-positive CML 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 accelerate approval program dictates that continued approval is contingent upon verification and confirmatory evidence of clinical evidence.

“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,” said Lee Greenberger, chief scientific officer, Leukemia & Lymphoma Society, 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, which showed that acimibinib 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 acimibimib needed to discontinue treatment because of adverse event (AE), compared with 25% in the bosutinib cohort.

The novel agent, also known as a STAMP inhibitor, functions by binding to the ABL myristoyl pocket..

Notable AEs from the trial included laboratory abnormalities, upper respiratory tract infections and musculoskeletal pain; decrease in platelet and neutrophil counts, decrease in hemoglobin; increase in triglycerides, creatine kinase and alanine aminotransferase (ALT).

“CML can be difficult to treat when currently available treatments fail patients, when treatment side effects cannot be tolerated, or sometimes both,” said Michael J. Mauro, MD, Hematologist and Myeloproliferative Neoplasms Program Leader at Memorial Sloan Kettering Cancer Center (MSK). “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. Novartis. News release. October 29, 201. Accessed October 29, 2021. https://www.globenewswire.com/news-release/2021/10/29/2323914/0/en/FDA-approves-Novartis-Scemblix-asciminib-with-novel-mechanism-of-action-for-the-treatment-of-chronic-myeloid-leukemia.html