Asciminib is an investigational STAMP inhibitor, currently under development across multiple treatment lines of acute myeloid syndrome

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Novartis gets FDA BTD for asciminib. (Credit: Novartis AG.)

Novartis has secured the US Food and Drug Administration (FDA) Breakthrough Therapy designation (BTD) for its investigational treatment specifically targeting the ABL myristoyl pocket (STAMP), dubbed asciminib.

The US FDA granted BTD for asciminib in treating adult patients with Philadelphia chromosome-positive chronic myeloid leukaemia (Ph+ CML) in chronic phase (CP), previously treated with two or more tyrosine kinase inhibitors (TKIs).

The drug was also granted BTD for the treatment of adult patients with T315I mutation-positive Ph+ CML in CP.

Asciminib is currently being studied in patients with CML, who experience resistance or intolerance to two or more TKIs, to help patients across multiple treatment lines of CML.

The Swiss drugmaker said that few pre-treated patients struggle to meet treatment goals due to resistance and intolerance. They may be at risk of progression and are left with only a few remaining treatment options.

The FDA designations were based on Phase 3 ASCEMBL trial, in patients with Ph+ CML in CP previously treated with two or more TKIs, and a Phase 1 trial in patients with Ph+ CML, with few of them bearing T315I mutation.

ASCEMBL is a Phase 3 multicentre, open-label, randomised study, designed to evaluate the superiority of asciminib in providing major molecular response rate at 24 weeks, compared to Bosulif (bosutinib).

Novartis said that asciminib has been previously granted Fast Track designation, and is planning to apply for a review under the FDA Oncology Centre of Excellence Real-Time Oncology Review programme, in the first half of 2021.

Furthermore, the company has received the US FDA BTD for its investigational oral therapy iptacopan in treating paroxysmal nocturnal hemoglobinuria (PNH).