USFDA awards Breakthrough Therapy designation to Novartis’ asciminib

Novartis has announced that the novel investigational treatment asciminib, specifically targeting the ABL myristoyl pocket (STAMP), has been awarded Breakthrough Therapy designation (BTD) by the US Food and Drug Administration (FDA).

Novartis has announced that the novel investigational treatment asciminib, specifically targeting the ABL myristoyl pocket (STAMP), has been awarded Breakthrough Therapy designation (BTD) by the US Food and Drug Administration (FDA).

Medicines that are being assessed for critical conditions are granted BTD if early clinical evidence indicates significant improvement potential over available therapy.

The STAMP inhibitor Asciminib received the designation for the treatment of Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP), previously treated with two or more tyrosine kinase inhibitors (TKIs), in adult patients.

The Novartis designed therapy was also awarded BTD for the treatment of adult patients suffering from Ph+ CML in CP and also having the T315I mutation.

Asciminib or ABL001 is an investigational treatment specifically targeting the ABL myristoyl pocket, otherwise called STAMP.  It is also being developed across multiple treatment lines of CML.

Although considerable advancement has been happening in CML treatment, quite a few patients risk progression of the disease due to resistance and intolerance.

The FDA designations, which could permit quick development and review of asciminib, were based on the key Phase III ASCEMBL trial and a Phase I trial that included patients with Ph+ CML (with some having the T315I mutation).

ASCEMBL is a multicenter, Phase III, randomised, open-label study and is the first head-to-head clinical trial in chronic myeloid leukemia using a second-generation tyrosine-kinase inhibitor (TKI) as a comparator.

Earlier, the USFDA had awarded asciminib Fast Track designation, which Novartis plans to follow up under the FDA Oncology Center of Excellence Real-Time Oncology Review program, with a submission for review in the first half of 2021.