Targeting Two Types of Lymphoma
The FDA granted accelerated approval to the molecularly targeted therapeutic umbralisib for certain patients with marginal zone lymphoma or follicular lymphoma.
The U.S. Food and Drug Administration (FDA) granted accelerated approval to umbralisib (Ukoniq), to treat certain adult patients with marginal zone lymphoma or follicular lymphoma, two types of non-Hodgkin lymphoma.
Umbralisib is a kinase inhibitor. These therapeutics block the action of one or more enzymes involved in cell signaling. Blocking those pathways may help keep the cancer cells from growing. Umbralisib inhibits both the PI3K-delta and casein kinase CK1-epsilon pathways.
The 1988 discovery of the phosphoinositide 3-kinase (PI3K) signaling pathway by Lewis C. Cantley, PhD, a Fellow of the AACR Academy, laid fundamental groundwork for the development of kinase inhibitors such as umbralisib.
The FDA approval is for certain patients with relapsed or refractory marginal zone lymphoma (MZL) who had received at least one prior anti-CD20-based treatment, and for certain patients with relapsed or refractory follicular lymphoma who had received prior therapies. Both are forms of B-cell lymphoma, the most common form of non-Hodgkin lymphoma.
The approval was based on two single-arm cohorts of an open-label, multi-center, multi-cohort trial. The trial involved 69 patients with marginal zone lymphoma who had undergone at least one prior therapy, including an anti-CD20-based regimen, and 117 patients with follicular lymphoma who had undergone at least two systemic therapies.
The overall response rate among the patients with marginal zone lymphoma was 49 percent, with 16 percent achieving complete responses. The median duration of response was not reached in these patients. For patients with follicular lymphoma, the overall response rate was 43 percent, with 3 percent of the study participants achieving complete responses. The median duration of response for these patients was 11.1 months.
Learn more about marginal zone and follicular lymphomas here.
The FDA granted accelerated approval to umbralisib on February 5, 2021. Accelerated approval means continued approval may be contingent upon a confirmatory trial.