(Leukemia) FDA Grants Accelerated Approval to Scemblix

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(Leukemia) FDA Grants Accelerated Approval to Scemblix

• October 29, 2021
• 2 min read

On October 29, 2021, the U.S. Food and Drug Administration (FDA) granted accelerated approval to Scemblix (asciminib) for patients with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase who have been previously treated with two or more tyrosine kinase inhibitors (TKIs). It was also approved for patients with Ph+ CML in chronic phase with the T315I mutation.

The efficacy was evaluated in the ASCEMBL (NCT03106779) trial, a multicenter, randomized, open-label trial. A total of 233 patients with Ph+ CML-CP who had previously received two or more TKIs were randomized (2:1) to receive Scemblix (40 mg twice daily) or bosutinib (500 mg once daily). The primary endpoint was the major molecular response (MMR) rate at 24 weeks. The MMR rate was 25% for the Scemblix group compared to 13% for the bosutinib group.

In the CABL001X2101 (NCT02081378) trial, an open-label multicenter study, 45 patients with Ph+ CML-CP with the T315I mutation received Scemblix (200 mg twice daily). The results showed that 42% of patients achieved an MMR by 24 weeks and 49% achieved an MMR by 96 weeks. The median duration of treatment was 108 weeks.

The most common side effects (≥20%) included upper respiratory tract infection, musculoskeletal pain, fatigue, nausea, rash, and diarrhea. Common laboratory abnormalities included decreased platelets, increased triglycerides, decreased neutrophils, decreased hemoglobin, and increased levels of creatine kinase, alanine aminotransferase, lipase, and amylase.

The recommended dose of Scemblix for patients previously treated with two or more TKIs is 80 mg once daily or 40 mg twice daily. For patients with the T315I mutation, the recommended dose is 200 mg twice daily.

Source: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-asciminib-philadelphia-chromosome-positive-chronic-myeloid-leukemia

#Leukemia #CML #FDA #Scemblix #Asciminib #CancerTreatment #T315I

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