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).

Published On 2021-02-09 05:30 GMT | Update On 2021-02-09 10:36 GMT

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.

Despite tremendous advances in CML treatment over the past few decades, some of these pre-treated patients struggle to meet treatment goals due to resistance and intolerance.

With few remaining treatment options, patients in later lines of care may be at risk of progression.

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.

Asciminib (ABL001) is an investigational treatment specifically targeting the ABL myristoyl pocket (STAMP). As a STAMP inhibitor, asciminib is being studied in patients with chronic myeloid leukemia (CML) who experience resistance or intolerance to two or more tyrosine-kinase inhibitors (TKIs), and in several clinical trials in hopes of helping patients across multiple treatment lines of CML.

ASCEMBL is the first head-to-head clinical trial in chronic myeloid leukemia using a second-generation tyrosine-kinase inhibitor (TKI) as a comparator. As a Phase III, multicenter, open-label, randomized study, ASCEMBL was designed to evaluate superiority in major molecular response rate at 24 weeks of the oral investigational treatment asciminib (ABL001) versus bosutinib in patients with Philadelphia-chromosome positive CML in chronic phase previously treated with two or more TKIs. Patients with failure or intolerance to the most recently administered TKI therapy were included in the trial.

Read also: Novartis to provide manufacturing capacity for Pfizer-BioNTech COVID-19 vaccine production

Disclaimer: This website is primarily for healthcare professionals. The content here does not replace medical advice and should not be used as medical, diagnostic, endorsement, treatment, or prescription advice. Medical science evolves rapidly, and we strive to keep our information current. If you find any discrepancies, please contact us at corrections@medicaldialogues.in. Read our Correction Policy here. Nothing here should be used as a substitute for medical advice, diagnosis, or treatment. We do not endorse any healthcare advice that contradicts a physician's guidance. Use of this site is subject to our Terms of Use, Privacy Policy, and Advertisement Policy. For more details, read our Full Disclaimer here.

NOTE: Join us in combating medical misinformation. If you encounter a questionable health, medical, or medical education claim, email us at factcheck@medicaldialogues.in for evaluation.

Ruchika Sharma

Ruchika Sharma joined Medical Dialogue as an Correspondent for the Business Section in 2019. She covers all the updates in the Pharmaceutical field, Policy, Insurance, Business Healthcare, Medical News, Health News, Pharma News, Healthcare and Investment. She has completed her B.Com from Delhi University and then pursued postgraduation in M.Com. She can be contacted at editorial@medicaldialogues.in Contact no. 011-43720751

Comments Policy

Our comments section is governed by our Comments Policy . By posting comments at Medical Dialogues you automatically agree with our Comments Policy , Terms And Conditions and Privacy Policy .

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).

Similar News

48 projects approved to ensure uninterrupted supply of critical APIs

Serum Institute seeks Covovax inclusion in COVID vaccination program

Aurobindo Pharma inks pact with US biotech Covaxx to develop COVID-19 vaccine