FDA Grants Breakthrough Therapy Designation to Asciminib for CML

The FDA has granted breakthrough therapy designation to asciminib, a first-in-class allosteric inhibitor specifically targeting the ABL myristoyl pocket (STAMP), for the treatment of adults with Philadelphia chromosome-positive chronic-phase chronic myeloid leukemia (CML) that is resistant to two or more tyrosine kinase inhibitors. This indication includes patients who harbor the T315I mutation.

The decision, which may allow for expedited development and review of the treatment, is based on data from the phase III ASCEMBL trial, in which asciminib was more effective at achieving major molecular response than bosutinib in patients with resistant/
intolerant CML.

Previously, the FDA granted fast track designation to asciminib.

Source: Novartis press release, February 8, 2021.