The FDA has granted accelerated approval to asciminib as a first-line treatment for adults newly diagnosed with Philadelphia chromosome-positive chronic myeloid leukemia, a rare type of blood cancer.
Known by the brand name Scemblix, the drug was first approved in 2021 for the same type of cancer, but not as a first-line treatment. According to Novartis, Scemblix’s maker, this once- or twice-daily oral tablet is now approved for both newly diagnosed and previously treated chronic myeloid leukemia, giving access to this potential new treatment to four times as many patients.
This cancer causes the bone marrow to make tyrosine kinase, a special protein that increases the production of leukemia cells, which are abnormal white blood cells. Aciminib blocks this protein at a unique site, different from other drugs in its class.
"Many patients who are newly diagnosed with CML struggle to navigate this chronic condition and may switch or even stop treatment because of side effects that interrupt their daily lives," said Lee Greenberger, PhD, the chief scientific officer at The Leukemia & Lymphoma Society. He also stressed the need for finding a medicine that’s right for patients at the beginning of treatment as it may lead to better long-term disease control with fewer side effects.
The approval was based on a phase III trial with 405 patients newly diagnosed with Philadelphia chromosome-positive chronic myeloid leukemia. Patients received either a daily dose of asciminib or one of four other tyrosine kinase inhibitors (TKIs) for 48 weeks. Asciminib proved more effective, safer, and better tolerated than standard treatments, though common side effects included muscle pain, rash, fatigue, respiratory infections, headaches, stomach pain, and diarrhea. Approval was also supported by early results from another study on long-term leukemia patients who stopped standard TKI therapy due to treatment failure or side effects.
In their press release, Novartis called Scemblix the first drug of its kind. Jorge Cortes, MD, director of the Georgia Cancer Center, noted that clinicians often face challenges with available treatments.
“Scemblix achieved impressive results across all three parameters of efficacy, safety, and tolerability versus all standard-of-care TKIs. This Scemblix data has the potential to be practice-changing,” he said.
The trials will continue till 96 weeks for additional results, the company said.
