FDA Grants Orphan Drug Status to Umbralisib for Treatment of Follicular Lymphoma

TG Therapeutics announced that their investigational dual inhibitor drug umbralisib was granted orphan drug designation by the FDA for treatment of patients with follicular lymphoma (FL).

As part of TG Therapeutics’ UNITY-NHL phase 2b registration directed clinical trial, umbralisib is being evaluated across several types of lymphoma. Umbralib, a once-daily oral therapy, works as a dual-inhibitor of the P13K-delta and CK1-epsilon proteins, and was noted by the company as demonstrating anti-cancer activity in preclinical models of blood cancers and in clinical studies as well.

Findings reported in October 2019 on the FL cohort of the UNITY-NHL trial (n = 118), which was designed to evaluate the safety and efficacy of umbralisib in patients who had received at least 2 prior lines of therapy, indicated that patients achieved an overall response rate (ORR) greater than 40%, meeting its primary endpoint of a prespecified ORR of 40-50%. Additionally, results from the patient cohort with previously treated marginal zone lymphoma (MZL) met its primary endpoint as well.

Following the study findings, the company initiated a rolling submission of a New Drug Application (NDA) to the FDA in January 2020 for umbralisib as a treatment for patients with previously treated MZL and FL. “The receipt of orphan drug designation for umbralisib to treat patients with FL is another important milestone in the development and anticipated commercialization of umbralisib in MZL and FL,” said Michael Weiss, executive chairman and chief executive officer of TG Therapeutics.

FL is a typically slow-growing or indolent form of non-Hodgkin lymphoma (NHL) that serves as the most common indolent lymphoma, accounting for approximately 20% of all NHL cases. While patients with FL can live for many years, the condition is generally not curable and is a chronic disease. When in advanced stages, 12-month cumulative costs associated with FL were approximately 3.5 times higher for patients with progressive disease than for individuals without progressive disease ($30,890 vs $8704), indicating the importance of novel therapies to reduce disease burden.

The orphan drug designation for umbralisib adds to the previous orphan drug designations granted to the drug by the FDA for treatment of patients with all 3 types of MZL, which are nodal, extranodal, and splenic MZL.

“We are excited by the progress so far and look forward to completion of this submission targeted in the first half of this year,” said Weiss.