Asciminib (Scemblix; Novartis AG) has been approved for use in adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP).
The FDA’s approval was based on efficacy data from the ASC4FIRST (NCT04971226) clinical trial, a multicenter, randomized, active-controlled, open-label trial in which 405 patients were randomly assigned in a 1:1 fashion to receive either asciminib or investigator-selected tyrosine kinase inhibitors (IS-TKIs) (imatinib, nilotinib, dasatinib, or bosutinib).1
The major molecular response rate at 48 weeks was 68% (95% confidence interval [CI]: 61-74) in the asciminib arm and 49% (95% CI: 42-56) in the IS-TKIs arm (difference 19% [95% CI, 10-28], P<0.001). Within the imatinib stratum, the MMR rate was 69% (95% CI: 59, 78) in the asciminib arm and 40% (95% CI: 31-50) in the IS-TKIs arm (difference 30% [95% CI: 17-42], P<0.001).
“While there are a range of effective TKIs currently available for newly diagnosed patients, clinicians frequently have had to weigh sacrificing either efficacy or tolerability,” Jorge Cortes, MD, director of Georgia Cancer Center said in a press release about the approval. “In the first-of-its-kind ASC4FIRST trial, Scemblix achieved impressive results across all 3 parameters of efficacy, safety and tolerability versus all standard-of-care TKIs. This data has the potential to be practice-changing.”2
The most commonly reported adverse reactions (≥20%), according to an FDA statement, were “musculoskeletal pain, rash, fatigue, upper respiratory tract infection, headache, abdominal pain, and diarrhea. The most common laboratory abnormalities (≥40%) in patients with newly diagnosed Ph+ CML in CP were decreased lymphocyte count, decreased leukocyte count, decreased platelet count, decreased neutrophil count, and decreased calcium corrected.”
FDA officials announced the approval on October 29, 2024.
References
- FDA. FDA grants accelerated approval to asciminib for newly diagnosed chronic myeloid leukemia. Published October 29, 2024. Accessed November 19, 2024. www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accel erated-approval-asciminib-newly-diagnosed-chronic-myeloid-leukemia
- Novartis. Novartis Scemblix FDA approved in newly diagnosed CML, offering superior efficacy, and favorable safety and tolerability profile [press release]. Published October 29, 2024. Accessed November 19, 2024. www.novartis.com/us-en/news/media-releases/novartis-scemblix-fda-approved-newly-diagnosed-cml-offering-superior-efficacy-and-favorable-safety-and-tolerability-profile