The FDA has granted accelerated approval to Scemblix (asciminib) as a first-line treatment for adults newly diagnosed with Philadelphia chromosome-positive chronic myeloid leukemia (CML), a rare blood cancer. Previously approved in 2021 for CML patients with prior treatment, Scemblix can now be prescribed to newly diagnosed patients, expanding its reach to four times as many people, according to Novartis, the drug’s manufacturer. This oral tablet, taken once or twice daily, targets tyrosine kinase—a protein that stimulates leukemia cell production—in a unique way compared to other treatments.
CML often results from an overproduction of tyrosine kinase, which drives the production of abnormal white blood cells. Asciminib works by blocking this protein at a different site than other tyrosine kinase inhibitors (TKIs), offering a potentially improved treatment pathway for CML patients. “Many newly diagnosed CML patients struggle to manage this chronic condition and may switch or even stop treatment due to side effects,” said Lee Greenberger, PhD, chief scientific officer at The Leukemia & Lymphoma Society. He emphasized the importance of finding a suitable treatment early in a patient’s journey, as this can improve long-term disease control and reduce side effects.
Approval for Scemblix was based on a phase III trial involving 405 newly diagnosed CML patients, who were treated with either asciminib or one of four other TKIs over 48 weeks. Results indicated that asciminib was more effective, safer, and better tolerated than standard treatments, though some side effects were noted, such as muscle pain, fatigue, rash, respiratory infections, headaches, and gastrointestinal issues. An additional study supported approval, showing benefits for long-term leukemia patients who stopped standard TKIs due to treatment failure or side effects.
As reported by webmd, in a statement, Novartis highlighted Scemblix as the first drug of its kind. Dr. Jorge Cortes, director of the Georgia Cancer Center, added that Scemblix’s data across efficacy, safety, and tolerability has the potential to change clinical practice. Trials will continue through 96 weeks for further insights.