On March 5, 2020 TG Therapeutics, Inc. (NASDAQ: TGTX) reported that the U.S. Food and Drug Administration (FDA) granted orphan drug designation to umbralisib, the Company’s investigational dual inhibitor of PI3K-delta and CK1-epsilon, for the treatment of patients with follicular lymphoma (FL) (Press release, TG Therapeutics, MAR 5, 2020, View Source [SID1234555214]).
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Umbralisib is being evaluated across several types of lymphoma in the UNITY-NHL Phase 2b registration directed clinical trial. The FL cohort of the UNITY-NHL trial is designed to evaluate the safety and efficacy of umbralisib in patients with FL who have received at least two prior lines of therapy including an anti-CD20 monoclonal antibody and an alkylating agent. In October 2019, the Company announced that the FL cohort met the primary endpoint of overall response rate (ORR), and in January the Company initiated a rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for umbralisib as a treatment for patients with previously treated marginal zone lymphoma (MZL) and FL.
The FDA has previously granted orphan drug designation to umbralisib, for the treatment of patients with all three types of MZL: nodal, extranodal, and splenic MZL.
Michael S. Weiss, Executive Chairman and Chief Executive Officer of TG Therapeutics, stated, "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." Mr. Weiss continued, "We were pleased to announce last year that both the MZL and FL cohorts of the UNITY-NHL trial met their primary endpoints and have commenced our first rolling submission for these indications. We are excited by the progress so far and look forward to completion of this submission targeted in the first half of this year."
ABOUT ORPHAN DRUG DESIGNATION
Orphan drug designation is granted by the U.S. FDA to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. Orphan drug designation provides certain incentives which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers. If a product that has orphan drug designation subsequently receives the first FDA approval for the disease for which it has such designation, the product is entitled to orphan drug exclusivity.
ABOUT FOLLICULAR LYMPHOMA
Follicular lymphoma (FL) is typically a slow-growing or indolent form of non-Hodgkin lymphoma (NHL) that arises from B-lymphocytes, making it a B-cell lymphoma. Follicular lymphoma is generally not curable and is a chronic disease. Patients can live for many years with this form of lymphoma. With an annual incidence in the United States of approximately 15,000 newly diagnosed patients1, FL is the most common indolent lymphoma accounting for approximately 20 percent of all NHL cases2.
ABOUT THE UNITY-NHL PHASE 2b STUDY—FOLLICULAR LYMPHOMA COHORT
The multicenter, open-label, UNITY-NHL Phase 2b study follicular lymphoma (FL) cohort was designed to evaluate the safety and efficacy of single agent umbralisib in patients with FL who have received at least two prior lines of therapy, including an anti-CD20 regimen and an alkylating agent. The primary endpoint is overall response rate (ORR) as determined by Independent Review Committee (IRC) assessment. Secondary endpoints include safety, duration of response, and progression-free survival (PFS).
In October of 2019, the Company announced that the primary endpoint of ORR as determined by IRC was met for all treated FL patients (n=118). The results met the Company’s prespecified response target of 40-50% ORR. In January of 2020, the Company announced the initiation of a rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) requesting accelerated approval of umbralisib as a treatment for patients with previously treated marginal zone lymphoma (MZL) and follicular lymphoma (FL).