FDA grants Breakthrough Therapy Designation to umbralisib (TGR-1202) for adult patients with previously treated MZL

On 22 January 2019, umbralisib (TGR-1202), a novel phosphoinositide 3-kinase (PI3K)-delta inhibitor, was granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with marginal zone lymphoma (MZL) who have received one prior anti-CD20 regimen.

The Breakthrough Therapy Designation granted by the FDA was based on the interim data from one cohort of the ongoing, registration-directed, phase IIb UNITY-NHL trial (NCT02793583) that is evaluating umbralisib as monotherapy or as part of a doublet or triplet, in patients across a number of relapsed/refractory non-Hodgkin lymphoma subtypes, including the single agent umbralisib therapy in the MZL cohort.

In the published data from the relapsed/refractory chronic lymphocytic leukemia (CLL) cohort who received single-agent umbralisib, the median progression-free survival and overall survival were not reached, and a median follow-up of 9.5 months, suggesting durable responses in high-risk patients.

According to the drug manufacturers, the Breakthrough Therapy Designation granted for TGR-1202 provides a critical treatment option for MZL patients who fail first-line chemoimmunotherapy treatment which leaves patients who relapse with limited options, and “can play an important role in fulfilling this unmet clinical need.”