Novartis today announced that the US Food and Drug Administration (FDA) accepted and granted Priority Review to the company’s New Drug Application (NDA) for asciminib (ABL001) in chronic myeloid leukemia (CML), following its submission under the FDA's Real-Time Oncology Review (RTOR) program. Priority Review is granted to therapies that have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions, as determined by the FDA1. This designation could shorten the FDA review period to eight months compared to the 12 months under Standard Review1.
- Priority Review granted based on positive data from the pivotal, Phase III ASCEMBL trial, where asciminib was compared to Bosulif® (bosutinib)* in patients with Philadelphia chromosome-positive CML in chronic phase (Ph+ CML-CP) previously treated with two or more tyrosine-kinase inhibitors (TKIs), and data from a Phase I trial that included patients with Ph+ CML-CP harboring the T315I mutation2,3
- Despite available treatments, many patients with CML remain at risk of disease progression, and sequential therapy with currently available TKIs may be associated with increased resistance and/or intolerance4-10
- Asciminib, a novel investigational therapy specifically targeting the ABL myristoyl pocket – also known as a STAMP inhibitor –, is in development across multiple treatment lines for CML11-17
Novartis has previously received Orphan Drug, Fast Track and two Breakthrough Therapy designations for asciminib. Breakthrough Therapy designations were granted for asciminib for the treatment of adult patients with Ph+ CML-CP previously treated with two or more TKIs, as well as adult patients with Ph+ CML-CP harboring the T315I mutation.