FDA Grants Accelerated Approval for Asciminib as First-Line Treatment in Ph+CML-CP

Novartis received accelerated approval by the US Food and Drug Administration (FDA) for asciminib, a medication for adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+CML-CP).

The approval comes from the Phase III ASC4FIRST trial, which demonstrated asciminib’s superior major molecular response (MMR) rates at 48 weeks compared with standard-of-care (SoC) tyrosine kinase inhibitors (TKIs), including imatinib, nilotinib, dasatinib, and bosutinib. Asciminib is the first treatment targeting the ABL myristoyl pocket (STAMP inhibitor), differing from other TKIs that target the ATP-binding site.

Compared with SoC TKIs, asciminibs’ MMR achievement rates were 68% vs 49% and 69% vs 40% with imatinib alone. Patients treated with asciminib experienced fewer severe adverse reactions (grade ≥3): 25.5% vs 33% and 42%, respectively. Asciminib also showed lower rates of treatment discontinuation (4.5% vs 11% and 9.8%) and dose reductions (6% vs 14 and 24%). Additionally, asciminib demonstrated deeper rates of molecular responses, including MR4. These findings suggest that asciminib may alter treatment standards by providing a more balanced efficacy and tolerability profile.

The ASC4FIRST trial will continue to evaluate secondary endpoints at week 96, looking at long-term MMR and additional safety data. The expanded indication in PH+CML-CP increases the population eligible for asciminib by approximately 4 times, including both newly diagnosed patients and previously treated adults. The recommended dosage is 80 mg taken orally once daily at approximately the same time each day or 40 mg taken orally twice daily at approximately 12-hour intervals.

“Despite many advances in the field, patients still need treatment options that are highly effective with a favorable tolerability profile to help enable them to achieve meaningful outcomes as they manage chronic conditions,” said Victor Bulto, President US, Novartis. “With this approval, we can offer newly diagnosed adult Ph+CML-CP patients a new treatment option that combines both, with the potential to change the trajectory of many more people living with CML.”

Reference

Novartis Scemblix® FDA approved in newly diagnosed CML, offering superior efficacy, and favorable safety and tolerability profile. News release. Novartis (US). Published 2024. Accessed October 31, 2024. https://www.novartis.com/us-en/news/media-releases/novartis-scemblix-fda-approved-newly-diagnosed-cml-offering-superior-efficacy-and-favorable-safety-and-tolerability-profile