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Drug Trials Snapshots: UKONIQ

HOW TO USE THIS SNAPSHOT
The information provided in Snapshots highlights who participated in the clinical trials that supported the FDA approval of this drug, and whether there were differences among sex, race and age groups. The “MORE INFO” bar shows more detailed, technical content for each section. The Snapshot is intended as one tool for consumers to use when discussing the risks and benefits of the drugs.

LIMITATIONS OF THIS SNAPSHOT
Do not rely on Snapshots to make decisions regarding medical care. Always speak to your health provider about the risks and benefits of a drug. Refer to the UKONIQ Prescribing Information for complete information.

Some of the information in this Snapshot is for presentation purposes and does not represent the approved conditions of use of this drug. Refer to the UKONIQ Prescribing Information for all of the approved conditions of use of this drug (e.g., indication(s), population(s), dosing regimen(s), safety information).

Snapshots are limited to the information available at the time of the original approval of the drug and do not provide information on who participated in clinical trials that supported later approvals for additional uses of the drug (if applicable).

UKONIQ (umbralisib)
You-KON-ik
TG Therapeutics, Inc.
Approval date: February 5, 2021


DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

UKONIQ is a drug used to treat adult patients with follicular lymphoma (FL) who have received at least three prior treatments that did not work or are no longer working.

Follicular lymphoma is a form of blood cancer.

How is this drug used?

UKONIQ is taken as a 200 mg tablet. Patients take 4 tablets (800 mg) once a day with food.

What are the benefits of this drug?

In a clinical trial with 117 patients with follicular lymphoma, 43% had a complete or partial shrinkage of their tumors (response).

UKONIQ was approved under FDA’s accelerated approval program, which provides earlier patient access to a promising new drug while the company continues to conduct clinical trials to confirm that the drug works well.

Were there any differences in how well the drug worked in clinical trials among sex, race and age?

  • Sex: UKONIQ worked similarly in women and men.
  • Race: The number of patients of races other than White was small; therefore, differences in how UKONIQ worked among races could not be determined.
  • Age: UKONIQ worked similarly among patients younger and older than 65 years of age.

What are the possible side effects?

UKONIQ may cause serious side effects including infections, diarrhea or inflammation of the intestines, decreased level of infection-fighting white blood cells (neutropenia), abnormal liver blood tests and severe skin reactions. UKONIQ may also cause allergic reactions due to a certain inactive ingredient (FD&C Yellow #5) included in the tablets. In addition, taking UKONIQ can cause fetal harm and breastfeeding is not advised.

The most common side effects of UKONIQ are decreased kidney function (increased creatinine), diarrhea or inflammation of the intestines, fatigue, nausea, decreased level of infection-fighting white blood cells (neutropenia), abnormal liver blood tests, musculoskeletal pain, low levels of red blood cells (anemia), low levels of blood platelets (thrombocytopenia), upper respiratory tract infection, vomiting, abdominal pain, decreased appetite, and rash.

Were there any differences in side effects among sex, race and age?

  • Sex: The occurrence of side effects were generally similar, in men and women. However, diarrhea, nausea, and vomiting were reported more frequently in women compared to men.
  • Race: The number of patients of races other than White was small; therefore, differences in the occurrence of side effects among races could not be determined.
  • Age: The occurrence of side effects was generally similar in patients younger and older than 65 years of age. However, in patients 65 years of age and older, 23% experienced serious adverse reactions compared to 12% in patients younger than 65 years of age. There was a higher incidence rate of infectious serious adverse reactions in patients 65 years of age or older (13%) compared to patients younger than 65 years of age (4%).

WHO WAS IN THE CLINICAL TRIALS?

Who participated in the trials?

The FDA approved UKONIQ based on evidence from four clinical trials (Study TGR-1202-101, TGR 1202-202, UTX-TGR-205, and UTX-TGR-501) that included 221 adults with marginal zone lymphoma (37%) and follicular lymphoma (63%) who received at least one prior treatment for marginal zone lymphoma and two prior treatments for follicular lymphoma that did not work or was no longer working.

Trials were conducted at 85 sites in United States, Poland, United Kingdom, Spain, South Korea, Israel, Italy, Australia, and Slovakia.

The population that provided data for side effects of UKONIQ (safety population) is presented below. Demographics of the patients who provided data for evaluation of benefits (efficacy population) are presented in Table 8, under the MORE INFO section.

Figure 1 summarizes how many men and women were enrolled in the clinical trials used to evaluate the safety of UKONIQ (safety population).

Figure 1. Baseline Demographics by Sex (Safety population)

Figure 1. Baseline Demographics by Sex (Safety population)

Source: FDA Review

Figure 2 summarizes the percentage of patients by race enrolled in the clinical trials used to evaluate the safety of UKONIQ (safety population).

Figure 2. Baseline Demographics by Race (Safety population)

Figure 2 is a pie chart summarizing how many participants by race in the population were evaluated for safety in the TGR-1202-101, TGR-1202-202, UTX-TGR-205, and UTXTGR-501 clinical trials.  Of the 221 participants assessed for safety, 180 (89%) were White, 12 (6%) Black or African American, and 6 (3%) Asian; Other races accounted for 5 (2%) of volunteers.

*Race was reported in 92% of patients. The pie chart and Table 6 reflect information on race in this group of patients.
*Other includes American Indian or Alaska Native, Native Hawaiian or Pacific Islander and Missing.

Adapted from FDA review and Prescribing Information

Figure 3. Baseline Demographics by Age (Safety Population)

Figure 3 is a pie chart summarizing how many participants by age in the population were evaluated for safety in the TGR-1202-101, TGR-1202-202, UTX-TGR-205, and UTXTGR-501 clinical trials.  Of the 221 participants assessed for safety, 98 (44%) were < 65 years of age and 123 (56%) were ≥ 65 years of age.

Adapted from FDA review and Prescribing Information

How were the trials designed?

The benefit and side effects of UKONIQ were evaluated in four clinical trials that included adult patients 29-88 years of age with follicular lymphoma and marginal zone lymphoma.

Trial 1 enrolled adult patients with follicular lymphoma after at least two prior treatments and marginal zone lymphoma after one prior treatment, that did not work or was no longer working. Patients received UKONIQ 800 mg orally once daily. Treatment continued until either disease worsened or patients experienced unacceptable toxicity. The benefit of UKONIQ was evaluated by measuring how many patients had complete or partial tumor shrinkage (response) and by how long that response lasted.

Trial 2 included patients with follicular lymphoma and marginal zone lymphoma whose disease has come back after at least two previous treatments. All patients received UKONIQ 800 mg oral daily until disease progression or unacceptable side effects. The benefit of UKONIQ was evaluated by measuring how many patients experienced complete or partial shrinkage of their tumors after treatment (overall response rate). Patients in Trial 2 were primarily evaluated for side effects for the purpose of this drug application.

Trial 3 enrolled patients with marginal zone lymphoma who had received one prior treatment. Patients received UKONIQ 800 mg orally once daily. The benefit of UKONIQ was evaluated by measuring how many patients had complete or partial tumor shrinkage (response) and how long that response lasted. Patients in Trial 3 were primarily evaluated for side effects for the purpose of this drug application.

Trial 4 was a long-term safety and efficacy extension study included patients with follicular lymphoma and marginal zone lymphoma who had completed treatment in Trial 2. Patients in Trial 4 were primarily evaluated for side effects.

GLOSSARY

CLINICAL TRIAL: Voluntary research studies conducted in people and designed to answer specific questions about the safety or effectiveness of drugs, vaccines, other therapies, or new ways of using existing treatments.
COMPARATOR: A previously available treatment or placebo used in clinical trials that is compared to the actual drug being tested.
EFFICACY: How well the drug achieves the desired response when it is taken as described in a controlled clinical setting, such as during a clinical trial.
PLACEBO: An inactive substance or “sugar pill” that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo.
SUBGROUP: A subset of the population studied in a clinical trial. Demographic subsets include sex, race, and age groups.

PRESCRIBING INFORMATION

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