On May 25, 2021 TG Therapeutics, Inc. (NASDAQ: TGTX), reported that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for ublituximab, the Company’s investigational glycoengineered anti-CD20 monoclonal antibody, in combination with UKONIQ (umbralisib), the Company’s once-daily, oral, inhibitor of PI3K-delta and CK1-epsilon, as a treatment for patients with chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) (Press release, TG Therapeutics, MAY 25, 2021, View Source [SID1234580524]). The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of March 25, 2022. The FDA also notified the Company that it is not currently planning to hold an advisory committee meeting to discuss this application.

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Michael S. Weiss, Executive Chairman and Chief Executive Officer of TG Therapeutics stated, "We are extremely pleased that the ublituximab BLA has been accepted by the FDA. This is an important milestone for us as it brings us one step closer to our goal of providing a novel combination treatment option to patients with both treatment naive and relapsed or refractory CLL and SLL. We look forward to collaborating with the FDA throughout this review process."

The BLA submission was based on the results of the UNITY-CLL trial, a global Phase 3 trial evaluating the combination of ublituximab plus UKONIQ (U2) compared to obinutuzumab plus chlorambucil in patients with treatment naive and relapsed or refractory CLL. The U.S. FDA previously granted Fast Track designation to the combination of ublituximab and UKONIQ for the treatment of adult patients with CLL and orphan drug designation for ublituximab in combination with UKONIQ for the treatment of CLL.

ABOUT UNITY-CLL PHASE 3 TRIAL
UNITY-CLL is a global Phase 3 randomized controlled clinical trial comparing the combination of ublituximab plus UKONIQ (umbralisib), or U2, to an active control arm of obinutuzumab plus chlorambucil in patients with both treatment-naïve and relapsed or refractory chronic lymphocytic leukemia (CLL). The trial randomized patients into four treatment arms: ublituximab single agent, UKONIQ single agent, ublituximab plus UKONIQ, and an active control arm of obinutuzumab plus chlorambucil. A prespecified interim analysis was conducted to assess the contribution of ublituximab and UKONIQ in the U2 combination arm and allowed for the termination of the single agent arms. Accordingly, the UNITY-CLL Phase 3 trial continued enrollment in a 1:1 ratio into the two combination arms: the investigational arm of U2 and the control arm of obinutuzumab plus chlorambucil. Approximately 420 subjects enrolled to the two combination arms and approximately 60% of patients were treatment-naïve and 40% were relapsed or refractory. The primary endpoint for this study was superior progression-free survival (PFS) for the U2 combination compared to the control arm. The trial met its primary endpoint and results were presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2020. The UNITY-CLL Phase 3 trial is being conducted under a Special Protocol Assessment (SPA) agreement with the U.S. Food and Drug Administration (FDA).

ABOUT CHRONIC LYMPHOCYTIC LEUKEMIA
Chronic lymphocytic leukemia (CLL) is the most common type of adult leukemia. It is estimated there will be more than 20,000 new cases of CLL diagnosed in the United States in 2020 and approximately 45,000 new cases globally in 2020.1,2 Although signs and symptoms of CLL may disappear for a period of time after initial treatment, the disease is considered incurable and many people will require additional treatment due to the return of malignant cells.

ABOUT FAST TRACK
Fast Track is a program designed to expedite the development and review of drugs that treat serious conditions and that demonstrate the potential to address an unmet medical need. Filling an unmet medical need is defined as providing a therapy where none exists or providing a therapy that may be potentially better than available therapy.

A drug that receives Fast Track designation is eligible for more frequent interactions with the FDA, priority review if relevant criteria are met, and rolling submission of the Biologics License Application or New Drug Application.

ABOUT ORPHAN DRUG DESIGNATION
Orphan drug designation is granted by the 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 product exclusivity.