Novartis gets USFDA Breakthrough Therapy designations for STAMP inhibitor asciminib to treat chronic myeloid leukemia
Asciminib (ABL001) is an investigational treatment specifically targeting the ABL myristoyl pocket (STAMP).
Basel: Novartis has recently announced that asciminib – a novel investigational treatment specifically targeting the ABL myristoyl pocket (STAMP) – has been granted Breakthrough Therapy designation (BTD) by the US Food and Drug Administration (USFDA) for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP), previously treated with two or more tyrosine kinase inhibitors (TKIs).
Asciminib was also granted BTD for the treatment of adult patients with Ph+ CML in CP harboring the T315I mutation.
These FDA designations, which may allow for an expedited development and review of asciminib, were based on:
- The pivotal, Phase III ASCEMBL trial, where asciminib was compared to Bosulif (bosutinib)* in patients with Ph+ CML in CP previously treated with two or more TKIs
- A Phase I trial that included patients with Ph+ CML, some of them harboring the T315I mutation
The FDA previously granted Fast Track designation to asciminib, and Novartis plans for a submission in the first half of 2021 for review under the FDA Oncology Center of Excellence Real-Time Oncology Review program.