FDA to prioritize review of Novartis STAMP inhibitor asciminib for chronic myeloid leukemia

Novartis (NYSE:NVS) has announced that the FDA has granted priority review for its application for allosteric inhibitor asciminib (ABL001) in chronic myeloid leukemia (CML).

The drug targets the ABL myristoyl pocket (STAMP).

The priority review status could trim four months off the review time, dropping the review period to eight months from 12 months under a standard review.

Novartis won the status after sharing positive data from its Phase 3 ASCEMBL trial, which pitted asciminib against Pfizer’s Bosulif (bosutinib) in participants with Philadelphia chromosome-positive CML in chronic phase (Ph+ CML-CP). Patients in the trial had received prior treatment with two or more tyrosine-kinase inhibitors (TKIs).

The prognosis for chronic myeloid leukemia has improved considerably since 2001, when promising treatments for the blood cancer began to emerge. Nevertheless, many people with CML face a risk of disease progression.

Sequential therapy with tyrosine-kinase inhibitors could lead to increased resistance or significant side effects, as a 2019 NEJM article explains.

FDA has granted two breakthrough therapy designations to asciminib and has also granted it orphan drug status.