FDA Grants Accelerated Approval to Asciminib in Ph+ CML in Chronic Phase

Supporting data for the approval came from the phase 3 ASC4FIRST trial (NCT04971226) assessing treatment with asciminib vs investigator-selected tyrosine kinase inhibitors (TKIs) among 405 patients with Ph-positive CML in CP.

The FDA has granted accelerated approval to asciminib (Scemblix) as a therapy for adults with newly diagnosed Philadelphia chromosome–positive chronic myeloid leukemia (CML) in chronic phase (CP), according to a news release from the agency.¹

Supporting data for the approval came from the phase 3 ASC4FIRST trial (NCT04971226) assessing treatment with asciminib vs investigator-selected (IS) tyrosine kinase inhibitors (TKIs) among 405 patients with Ph-positive CML in CP. Topline data showed a 48-week major molecular response (MMR) rate of 68% (95% CI, 61%-74%) in the asciminib arm vs 49% (95% CI, 42%-56%) with IS-TKIs. Within the imatinib (Gleevec) stratum of the trial, the MMR rates were 69% (95% CI, 59%-78%) vs 40% (95% CI, 31%-50%) in each respective treatment arm.

Pooled safety population data showed that frequent adverse effects included musculoskeletal pain, fatigue, rash, upper respiratory tract infection, headache, abdominal pain, and diarrhea.

The FDA approved asciminib at a recommended dose of 80 mg orally once daily or 40 mg orally twice daily in 12-hour intervals.

The FDA previously granted priority review to asciminib as a treatment for patients with newly diagnosed Ph-positive CML in CP in July 2024.²

References

1. FDA grants accelerated approval to asciminib for newly diagnosed chronic myeloid leukemia. News release. FDA. October 29, 2024. Accessed October 29, 2024. https://tinyurl.com/2sjs7dp6
2. Novartis Scemblix® granted FDA priority review for the treatment of adults with newly diagnosed CML. News release. Novartis. July 29, 2024. Accessed October 29, 2024. https://tinyurl.com/2ap664kx