Asciminib Granted Accelerated Approval by FDA for Ph+ Chronic Myeloid Leukemia in 2 Indications

The FDA granted accelerated approval to asciminib for the treatment of patients with Philadelphia chromosome–positive chronic myeloid leukemia for 2 indications.

The FDA granted accelerated approval to asciminib (Scemblix) for the treatment of patients with Philadelphia chromosome (Ph)–positive chronic myeloid leukemia (CML) in chronic phase who previously received treatment with 2 or more tyrosine kinase inhibitors (TKIs), according to a press release from Novartis; additionally, a full approval was granted to the drug for patients with Ph-positive CML in chronic phase with a T315I mutation.1

The approval was based on results from the phase 3 ASCEMBL trial (NCT03106779), with results demonstrating a major molecular response (MMR) at 24 weeks of 25.5% in the asciminib arm compared with 13.2% in the bosutinib arm. The difference in MMR rate between arms after adjusting for major cytogenetic response status at baseline was 12.2% (95% CI, 2.19%-22.30%; P = .029).2

“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 that add a burden to their daily lives,” Lee Greenberger, chief scientific officer at The Leukemia & Lymphoma Society, said in a press release. “The approval of Scemblix may offer hope to patients by addressing gaps in CML care.”

The trial enrolled a total of 233 patients who were randomized 2:1 to either asciminib (n = 157) twice daily at 40 mg or bosutinib (n=76) once daily at 500 mg. The median follow-up was 14.9 months.

Further, the proportion of patients who discontinued treatment due to adverse effects (AEs) was significantly lower with asciminib (7%) compared with bosutinib (25%). Common AEs for patients receiving asciminib included upper respiratory tract infections and musculoskeletal pain, platelet and neutrophil count decrease, hemoglobin decrease, increase in triglycerides, and creatine kinase and alanine aminotransferase.


1. 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.

2. Rea D, Mauro MJ, Boquimpani C, et al. A Phase 3, Open-Label, Randomized Study of Asciminib, a STAMP Inhibitor, vs Bosutinib in CML After ≥2 Prior TKIs. Published online August 18, 2021. Blood. doi:10.1182/blood.2020009984