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The approval of umbralisib was primarily based on data from the marginal zone lymphoma and follicular lymphoma patient cohorts of the phase 2b UNITY-NHL trial.
The FDA has granted accelerated approval to the once-daily, oral, dual inhibitor of PI3K-d and CK1-epsilon, umbralisib (Ukoniq), as a treatment for patients with previously treated marginal zone lymphoma (MZL) who have received at least 1 prior anti-CD20 based regimen or follicular lymphoma (FL) after at least 3 prior systemic therapies, announced the drug’s developer, TG Therapeutics.1
The FDA recommended dose of umbralisib is 800 mg taken orally once daily with food until disease progression or unacceptable toxicity.
The approval was primarily based on data from the MZL and FL patient cohorts of the global, multicenter, open-label phase 2b UNITY-NHL trial (NCT02793583).2 The MZL cohort was designed to evaluate the safety and efficacy of single-agent umbralisib in patients with MZL who have received at least 1 prior anti-CD20 regimen. The FL cohort was designed to evaluate the safety and efficacy of single-agent umbralisib in patients with relapsed or refractory FL who had received at least 2 prior lines of therapy, including an anti-CD20 regimen and an alkylating agent.
TG Therapeutics previously announced that each cohort met its primary end point of overall response rate (ORR), as confirmed by an independent review committee (IRC), meeting the company’s response target guidance of 40% to 50%.
The trial included a total 208 patients with indolent non-Hodgkin lymphoma who had received at least 1 dose of umbralisib, including 69 patients with MZL, 117 with FL, and 22 with small lymphocytic lymphoma.
The ORR for patients with MZL was 49% (95% CI, 37.0-61.6), with a 16% complete response (CR) rate. Additionally, median PFS was not reached for this patient population (95% CI, 9.3-NE).
Among patients with FL, the ORR was 43% (95% CI, 33.6-52.2) with 3% achieving a CR. Median PFS was 10.6 months. The median duration of response for patients with FL was 11.1 months (8.3, 16.4).
The most common adverse events (AEs) observed in at least 15% of patients, including laboratory abnormalities, 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, most often from diarrhea-colitis and infection. The most common reasons for dose modifications were diarrhea-colitis and transaminase elevation.
On August 12, 2020, the FDA accepted a new drug application for umbralisib as a treatment for patients with previously treated MZL and FL. The FDA also previously granted umbralisib breakthrough therapy designation for MZL and orphan drug designation for MZL and FL.
1. FDA. FDA grants accelerated approval to umbralisib for marginal zone lymphoma and follicular lymphoma. FDA website. Published February 5, 2021. Accessed February 5, 2021. https://www.fda.gov/drugs/drug-approvals-and-databases/fda-grants-accelerated-approval-umbralisib-marginal-zone-lymphoma-and-follicular-lymphoma?utm_medium=email&utm_source=govdelivery
2. TG Therapeutics Announces FDA Acceptance of New Drug Application for Umbralisib as a Treatment for Patients with Previously Treated Marginal Zone Lymphoma and Follicular Lymphoma. News release. TG Therapeutics. Published August 13, 2020. Accessed February 5, 2021. https://ir.tgtherapeutics.com/news-releases/news-release-details/tg-therapeutics-announces-fda-acceptance-new-drug-application