The Food and Drug Administration (FDA) has granted accelerated approval to Scemblix® (asciminib) for the treatment of adults with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP), previously treated with 2 or more tyrosine kinase inhibitors. The FDA has also granted full approval to Scemblix for the treatment of adults with Ph+ CML in CP with T315I mutation.
The accelerated approval was based on data from the multicenter, open-label, randomized, active-controlled phase 3 ASCEMBL study (ClinicalTrials.gov Identifier: NCT03106779), which evaluated the efficacy and safety of asciminib, an ABL/BCR-ABL1 tyrosine kinase inhibitor, in 233 patients with Ph+ CML in CP previously treated with 2 or more tyrosine kinase inhibitors. Patients were randomly assigned 2:1 to receive either asciminib 40mg orally twice daily or bosutinib 500mg once daily until unacceptable toxicity or treatment failure occurred.
At 24 weeks, findings showed a major molecular response (MMR) rate of 25% (95% CI, 19-33) in patients receiving asciminib compared with 13% (95% CI, 6.5-23) in those treated with bosutinib (P =.029). At 48 weeks, the MMR rate was 29% (95% CI, 22-37) in patients receiving asciminib and 13% (95% CI, 6.5-23) in patients receiving bosutinib. With a median duration of follow-up of 20 months (range, 1 day to 36 months), the median duration of response had not yet been reached for patients with MMR at any time.
The most common adverse reactions reported with asciminib were upper respiratory tract infections, musculoskeletal pain, fatigue, nausea, rash, and diarrhea. The most common laboratory abnormalities were decreased platelet count, increased triglycerides, decreased neutrophil count, decreased hemoglobin, increased creatine kinase, increased alanine aminotransferase, increased lipase, and increased amylase.
Full approval of Scemblix in patients with Ph+ CML in CP with T315I mutation was based on data from a multicenter, open-label phase 1 study (N=45; ClinicalTrials.gov Identifier: NCT02081378). Findings showed that MMR was achieved by 24 weeks in 42% (95% CI, 28-58) of patients, and by 96 weeks in 49% (95% CI, 34-64) of patients. The median duration of treatment was 108 weeks (range, 2 to 215 weeks).
Scemblix is supplied as 20mg and 40mg tablets in 60-count bottles.
- FDA approves Novartis Scemblix® (asciminib), with novel mechanism of action for the treatment of chronic myeloid leukemia. News release. Novartis Pharma AG. October 29, 2021. Accessed November 1, 2021. https://www.globenewswire.com/news-release/2021/10/29/2323914/0/en/FDA-approves-Novartis-Scemblix-asciminib-with-novel-mechanism-of-action-for-the-treatment-of-chronic-myeloid-leukemia.html.
- FDA approves asciminib for Philadelphia chromosome-positive chronic myeloid leukemia. News release. US Food and Drug Administration. October 29, 2021. Accessed November 1, 2021. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-asciminib-philadelphia-chromosome-positive-chronic-myeloid-leukemia?utm_medium=email&utm_source=govdelivery.
- Scemblix. Package. Novartis Pharmaceuticals Corporation; 2021. Accessed November 1, 2021. https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/215358s000Orig1lbl.pdf.