European Commission Approves Asciminib in Philadelphia Chromosome–Positive CML

Results from the phase 3 ASCEMBL trial led to the approval of asciminib by the European Commission for patients with Philadelphia chromosome–positive chronic myeloid leukemia in chronic phase.

The European Commission has approved asciminib (Scemblix) for patients with Philadelphia chromosome–positive chronic myeloid leukemia (CML) in chronic phase who have been treated with 2 or more Tyrosine kinase inhibitors (TKI), according to a press release from Novartis.1

The approval is based on data the phase 3 ASCEMBL trial (NCT03106779) which compared asciminib vs bosutinib (Bosulif) in the aforementioned patient population. An improved molecular response rate of 25.5% vs 13.2% was reported in the asciminib and bosutinib arms, respectively (P = .029). The findings were further confirmed at 96 weeks, with investigators reporting a major molecular response rate of 37.6% (95% CI, 29.99%-45.65%) in the asciminib arm and 15.8% (95% CI, 8.43%-25.96%) in the bosutinib arm.

“Until now, patients with CML in Europe had oral TKI therapies with the same mechanism of action to turn to, and those experiencing significant [adverse] effects [AEs] or resistance to these treatment options would often cycle between these very similar therapies, with little success in controlling their disease or improving their quality of life,” Andreas Hochhaus, MD, head of the Department of Hematology and Medical Oncology at Jena University Hospital in Germany, said in the press release. “The approval of [asciminib] in Europe is a timely milestone that will help many patients find hope for the management of their CML.”

Rates of discontinuation due to AEs were lower in the asciminib arm at 5.8% vs 21.1% in the bosutinib arm at 24 weeks. The most common AEs among those treated with asciminib included musculoskeletal pain (37.1%), upper respiratory tract infections (28.1%), thrombocytopenia (27.5%), fatigue (27.2%), headache (24.2%), arthralgia (21.6%), increased pancreatic enzymes (21.3%), abdominal pain (21.3%), diarrhea (20.5%) and nausea (20.2%).

In October 2021, asciminib was granted accelerated approval by the FDA for patients with Philadelphia chromosome–positive CML in chronic phase.2 Full approval was granted for those with Philadelphia chromosome–positive CML in chronic phase for those with a T35I mutation.

References

  1. Novartis Scemblix®, with novel mechanism of action, approved by the European Commission for adult patients with chronic myeloid leukemia. News Release. Novartis. August 29, 2022. Accessed August 29, 2022. https://bit.ly/3RgfqOx
  2. FDA approves Novartis Scemblix® (asciminib), with novel mechanism of action for the treatment of chronic myeloid leukemia. News release. Novartis. October 29, 2021. Accessed August 29, 2022. https://bit.ly/3AxnkfW