Asciminib Gets Accelerated Approval for Ph+ CML

The FDA granted an accelerated approval to asciminib (Scemblix) for adults with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) that is in chronic phase, according to an announcement from the agency.¹

The approval is based off findings from the multicenter, randomized, active-controlled, open-label ASC4FIRST trial (NCT04971226), in which 405 patients were randomized 1:1 to receive either asciminib or investigator’s choice of a selected tyrosine kinase inhibitor (TKI). Investigators could choose from the following TKIs: imatinib (Gleevec), nilotinib (Tasigna), dasatinib (Sprycel), or bosutinib (Bosulif).

READ MORE: Asciminib Has Superior Response Rates in CML

Major molecular response (MMR) at 48 weeks—the trial’s main efficacy outcome—was 68% (95% CI, 61-74) in the asciminib arm, compared with 49% (95% CI, 42-56) in the TKI arm (p value < 0.001). MMR rates were 69% (95% CI, 59-78) and 40% (95% CI, 31-50) in the asciminib and TKI groups, respectively (p-value <0.001).²

In a pooled safety population that included patients with newly diagnosed and previously treated Ph+ CML in chronic phase, the most common adverse effects that occurred in 20% or more of patients were musculoskeletal pain, rash, fatigue, upper respiratory tract infection, headache, abdominal pain, and diarrhea.

Meanwhile, common laboratory abnormalities that occurred in 40% or more of patients were: lymphocyte count, decreased leukocyte count, decreased platelet count, decreased neutrophil count, and decreased calcium corrected.

According to the FDA, recommended asciminib dosing is 80 mg once per day or 40 mg twice per day at approximately 12-hour intervals.

References

1. FDA. FDA grants accelerated approval to asciminib for newly diagnosed chronic myeloid leukemia. October 29, 2024. Accessed October 29, 2024. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-asciminib-newly-diagnosed-chronic-myeloid-leukemia

2. Hochhaus A, Wang J, Kim DW, et al; ASC4FIRST Investigators. Asciminib in newly diagnosed chronic myeloid leukemia. N Engl J Med. 2024;391(10):885-898. doi:10.1056/NEJMoa2400858