FDA

Asciminib granted accelerated approval for newly diagnosed CML

November 1, 2024

Brand name: Scemblix

Generic name: asciminib

Manufacturer: Novartis

Approval date: October 29, 2024

Scemblix (asciminib), a kinase inhibitor, is now approved for adults with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP). This indication is approved under accelerated approval based on major molecular response rate. Continued approval for this indication may be contingent upon verification of clinical benefit in a confirmatory trial(s).

Efficacy

Efficacy of asciminib for newly diagnosed Ph+ CML in CP was evaluated in ASC4FIRST (NCT04971226), a multicenter, randomized, active-controlled, open-label trial in which 405 patients were randomized (1:1) to receive either asciminib or investigator-selected tyrosine kinase inhibitors (IS-TKIs) (imatinib, nilotinib, dasatinib, or bosutinib). The main efficacy outcome measure was major molecular response (MMR) rate at 48 weeks. MMR rate at 48 weeks was 68% (95% CI, 61-74) in the asciminib arm vs. 49% (95% CI, 42-56) in the IS-TKIs arm (difference 19%; 95% CI, 10-28], p-value <0.001). Within the imatinib stratum, 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-value <0.001).

Safety

In the pooled safety population in patients with newly diagnosed and previously treated Ph+ CML in CP, the most common adverse reactions (≥20%) were:

• musculoskeletal pain
• rash
• fatigue
• upper respiratory tract infection
• headache
• abdominal pain
• diarrhea

The most common lab abnormalities (≥40%) in patients with newly diagnosed Ph+ CML in CP were decreases in:

• lymphocyte count
• leukocyte count
• platelet count
• neutrophil count
• calcium corrected

Recommended dose

The dosage of asciminib is 80 mg PO daily at approximately the same time of day or 40 mg PO twice daily at approximately 12-hour intervals.

Source:

FDA. (2024, October 29). FDA grants accelerated approval to asciminib for newly diagnosed chronic myeloid leukemia. [Press release]. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-asciminib-newly-diagnosed-chronic-myeloid-leukemia