Umbralisib gains FDA breakthrough designation for MZL


The Food and Drug Administration (FDA) has granted breakthrough therapy designation to umbralisib for the treatment of adults with marginal zone lymphoma (MZL) who have received at least one prior anti-CD20 regimen.

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Umbralisib (formerly TGR-1202) is a PI3K-delta inhibitor being developed by TG Therapeutics.

Breakthrough designation entitles the company developing a therapy to more intensive FDA guidance and other actions that may expedite FDA review. For a treatment to earn breakthrough designation, early clinical results must show that it provides improvement over available therapies or fulfills an unmet need.

The breakthrough designation for umbralisib was based on interim data from the MZL cohort in the ongoing, phase 2b UNITY-NHL trial (NCT02793583).

In this trial, researchers are testing umbralisib alone or in combination with ublituximab, with or without bendamustine, in patients with previously treated non-Hodgkin lymphoma.

“The MZL single-agent umbralisib cohort of the UNITY-NHL study is fully enrolled, and we look forward to reporting topline results from this cohort by mid-year and presenting the data at a major medical meeting in 2019,” Michael S. Weiss, chief executive officer of TG Therapeutics, said in a statement.

Umbralisib monotherapy was previously evaluated in a phase 1 trial (NCT01767766) of patients with relapsed or refractory B-cell malignancies (Lancet Oncol. 2018 Apr;19[4]:486-96).

The trial enrolled 90 patients, and five of them had MZL. A total of 33 patients achieved a response to umbralisib. This includes one MZL patient who achieved a partial response.

The most common treatment-emergent adverse events (AEs) in this trial were diarrhea, nausea, and fatigue. The most common grade 3/4 AEs were neutropenia, anemia, and thrombocytopenia.

Serious AEs considered at least possibly related to umbralisib were pneumonia, lung infection, febrile neutropenia, and colitis. There were no treatment-related deaths.

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