On October 30, Novartis announced that the US FDA approved Scemblix (asciminib) for the treatment of two different indications for chronic myeloid leukemia (CML)
.
The FDA has accelerated the approval of Scemblix for the treatment of Philadelphia (Ph) chromosome-positive CML patients in the chronic phase who have previously received two or more tyrosine kinase inhibitors (TKI) treatments
.
At the same time, the FDA fully approved it for the treatment of the above-mentioned patients with the T315I mutation
.
The press release pointed out that Scemblix is the first CML therapy combined with the myristoyl pocket of ABL1, providing an important new treatment option for patients who are resistant or intolerant to current TKI therapies
.
CML is a malignant myeloproliferative tumor that occurs in pluripotent hematopoietic stem cells.
It is characterized by a significant increase in granulocytes in the peripheral blood.
In some CML patients, Ph chromosome and/or BCR-ABL fusion genes can be found
.
For many patients, current therapies are limited by intolerance and drug resistance, and patients with T315I mutations are resistant to most TKIs, so the risk of disease progression increases
.
Scemblix is an allosteric inhibitor against ABL1, which inhibits the activity of BCR-ABL1 by binding to the myristoyl pocket of ABL1
.
Because its binding site with BCR-ABL1 is different from common TKI, it may solve the problem of TKI resistance and intolerance in the later treatment of CML patients
.
▲Scemblix's mechanism of action (picture source: reference [2])
This approval is based on the results of a phase 3 clinical trial and a phase 1 clinical trial
.
The results of the phase 3 clinical trial showed that Scemblix almost doubled the patient's major molecular response rate (MMR) in 24 weeks compared with the active control (25.
5% vs.
13.
2%, p=0.
029)
.
Moreover, the proportion of patients who stopped receiving treatment due to adverse reactions was one third of the control group (7% vs.
25%)
.
Note: The original text has been deleted
Reference materials:
[1] FDA approves Novartis Scemblix® (asciminib), with novel mechanism of action for the treatment of chronic myeloid leukemia.
Retrieved October 29, 2021, from https:// 29/2323914/0/en/FDA-approves-Novartis-Scemblix-asciminib-with-novel-mechanism-of-action-for-the-treatment-of-chronic-myeloid-leukemia.
html
[2] Hughes, et al.
(2019).
Asciminib in Chronic Myeloid Leukemia after ABL Kinase Inhibitor Failure.
NEJM, DOI: 10.
1056/NEJMoa1902328
