Scemblix receives FDA approval for Ph+ CML

FDA granted accelerated approval to Scemblix (asciminib) for patients with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase, who were previously treated with two or more tyrosine kinase inhibitors. FDA also approved Scemblix for adult patients with Ph+ CML in CP with the T315I mutation.

Scemblix is sponsored by Novartis.

ASCEMBL (NCT03106779), a multi-center, randomized, active-controlled, open-label clinical trial, is evaluating Scemblix in patients with Ph+ CML in CP, previously treated with two or more TKIs. A total of 233 patients were randomized (2:1) and stratified according to major cytogenetic response status to receive either Scemblix 40 mg twice daily or Bosulif (bosutinib) 500 mg once daily. 

Patients continued treatment until unacceptable toxicity or treatment failure occurred. The main efficacy outcome measure was major molecular response at 24 weeks. The MMR rate was 25% (95% CI: 19, 33) in patients treated with Scemblix compared with 13% (95% CI: 6.5, 23; p=0.029) in those receiving Bosulif. With a median duration of follow-up of 20 months, the median duration of MMR has not yet been reached.

CABL001X2101 (NCT02081378), a multi-center, open-label clinical trial, is evaluating Scemblix in patients with Ph+ CML in CP with the T315I mutation. Efficacy was based on 45 patients with the T315I mutation who received Scemblix 200 mg twice daily. 

Patients continued treatment until unacceptable toxicity or treatment failure occurred. The main efficacy outcome measure was MMR, which was achieved by 24 weeks in 42% (19/45, 95% CI: 28% to 58%) of the patients and by 96 weeks in 49% (22/45, 95% CI: 34% to 64%) of the patients. The median duration of treatment was 108 weeks (range, 2 to 215 weeks).

FDA approved this application four months ahead of the FDA goal date. This application was granted priority review, breakthrough designations, fast track designation, and orphan drug designation.