FDA Approves Umbralisib for Relapsed/Refractory MZL and FL

FDA Approves Umbralisib for Relapsed/Refractory MZL and FL

The FDA approved umbralisib (Ukoniq) for the treatment of adults with relapsed/refractory marginal zone lymphoma (MZL) who had at least 1 prior anti-CD20—based regimen; and adults with relapsed/refractory follicular lymphoma (FL) who had at least 3 prior lines of systemic therapy.

“Today’s approval of UKONIQ marks a historic day for our Company with this being our first approval and we are extremely pleased to be able to bring our novel inhibitor of PI3K-delta and CK1-epsilon to patients with relapsed/refractory MZL and FL,” said Michael S. Weiss, executive chairman and CEO of TG Therapeutics, the manufacturer of the PI3K-delta and casein kinase CK1-epsilon inhibitor.

The accelerated approval was based on the open-label, multi-center, multi-cohort UTX-TGR-205 trial, which involved 69 previously treated patients with MZL and 117 patients with follicular lymphoma who had at least 2 prior systemic therapies.

Overall response rate (ORR) was 49% in patients with MZL; 16% had a complete response. Average duration of response (DOR) was not reached for this patient group.

In patients with FL, there as a 43% ORR and 3% complete response rate. Median DOR was 11.1 months.

“Despite treatment advances, MZL and FL remain incurable diseases with limited treatment options for patients who relapse after prior therapy and no defined standard of care. With the approval of umbralisib we now have a targeted, oral, once-daily option, offering a needed treatment alternative for patients,” stated Nathan Fowler, MD, Professor of Medicine at The University of Texas MD Anderson Cancer Center and the Study Chair of the UNITY-NHL MZL &FL cohorts.

The FDA recommends that umbralisib be given at 800 mg taken orally once a day with food until the patient experiences disease progression or unacceptable toxicity.

Common adverse events (AEs), which occurred in 15% or more of patients, were: creatinine, diarrhea-colitis, fatigue, nausea, neutropenia, transaminase elevation, musculoskeletal pain, anemia, thrombocytopenia, upper respiratory tract infection, vomiting, abdominal pain, decreased appetite, and rash.

Eighteen percent of patients had severe AEs, with diarrhea-colitis and infection being the most common. The most common reasons for dose modifications were diarrhea-colitis and transaminase elevation.

The prescribing information provides warnings and precautions for adverse reactions including infections, neutropenia, diarrhea and non-infectious colitis, hepatotoxicity, and severe cutaneous reactions.

“We want to thank the patients, physicians, nurses and clinical coordinators for their support and participation in our clinical trials, and the FDA for their collaboration throughout this process. We remain dedicated to patients with B-cell diseases and our mission of developing treatment options for those in need,” Weiss said.