WASHINGTON — The FDA granted accelerated approval to the PI3K inhibitor umbralisib (Ukoniq) for two types of relapsed/refractory lymphoma, said manufacturer TG Therapeutics.
The approval encompasses patients with marginal zone lymphoma (MZL) treated with at least one prior anti-CD20 agent and patients with follicular lymphoma (FL) treated with at least three prior lines of systemic therapy. Umbralisib is an oral medication that targets PI3K-delta and casein kinase 1-epsilon.
“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,” said principal investigator Nathan Fowler, MD, of the University of Texas MD Anderson Cancer Center in Houston, in a TG therapeutics statement. “With the approval of umbralisib we now have a targeted, oral, once-daily option, offering a needed treatment alternative for patients.”
Primary support for the approval came from two single-arm cohorts enrolled in the open-label, multicenter UNITY-NHL trial. One cohort comprised 69 patients with MZL and prior exposure to anti-CD20 therapy and the other included 117 patients with FL treated with at least two prior systemic regimens. Patients received umbralisib once daily until disease progression or unacceptable toxicity. The primary outcomes were overall response rate (ORR) and duration of response (DOR).
The MZL cohort had an ORR of 49%, including complete responses (CRs) in 16% of the patients. Median DOR was not reached. The FL cohort had an ORR of 43%, including CRs in 3%. Median DOR was 11.1 months.
Adverse events (AEs) occurring in ≥15% of patients were increased creatinine, diarrhea-colitis, fatigue, nausea, neutropenia, transaminase elevation, musculoskeletal pain, anemia, thrombocytopenia, upper respiratory tract infection, vomiting, abdominal pain, decreased appetite, and rash. Serious AEs occurred in 18% of patients, primarily diarrhea-colitis and infection.
Accelerated approval is a conditional status, and the sponsor might be required to conduct additional studies to accumulate supporting data before the FDA makes a decision about full approval.