FDA Approves Umbralisib for Adults With FL, MZL
On February 5, 2021, the FDA granted accelerated approval to umbralisib (Ukoniq; TG Therapeutics), a kinase inhibitor including PI3K-delta and casein kinase CK1-epsilon, for the treatment of adults with relapsed/refractory follicular lymphoma (FL) or marginal zone lymphoma (MZL).
This approval was based on data from 2 single-arm trials involving 69 patients with MZL who received at least 1 prior therapy and 117 patients with FL after at least 3 prior systemic therapies.
For patients with MZL, the overall response rate (ORR) was 49% (95% CI, 37.0-61.6), with 16% achieving compete responses. The median duration of response (DOR) was not reached (95% CI, 9.3, NE).
For patients with FL, the ORR was 43% (95% CI, 33.6-52.2), with 3% achieving complete responses. The median DOR was 11.1 months (95% CI, 8.3-16.4).
The most common side effects reported include increased creatinine, diarrhea-colitis, fatigue, nausea, neutropenia, transaminase elevation, musculoskeletal pain, anemia, thrombocytopenia, upper respiratory tract infection, vomiting, abdominal pain, decreased appetite, and rash.—Emily Bader
Source: US Food and Drug Administration. FDA grants accelerated approval to umbralisib for marginal zone lymphoma and follicular lymphoma. February 5, 2021. www.fda.gov/drugs/drug-approvals-and-databases/fda-grants-accelerated-approval-umbralisib-marginal-zone-lymphoma-and-follicular-lymphoma. Accessed February 8, 2021.