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  5. Drug Trial Snapshot: TEPEZZA
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Drug Trial Snapshot: TEPEZZA

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  TEPEZZA Package Insert for complete information.

TEPEZZA (teprotumumab-trbw)
(tep-ez-zə)
Horizon
Approval date: January 21, 2020


DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

TEPEZZA is a drug for the treatment of thyroid eye disease.

Thyroid eye disease is a rare, autoimmune disease characterized by proptosis (a condition where the eyes are pushed forward and bulge outward) leading to eye pain, double vision and difficulty closing the eyelid.

How is this drug used?

TEPEZZA is an injection. It is given by a healthcare provider directly into the vein (an intravenous infusion) once every three weeks for a total of eight infusions. It takes about 60-90 minutes to receive the infusion.

What are the benefits of this drug?

After 6 months of treatment, a higher proportion of patients treated with TEPEZZA (71-83%) demonstrated reduction in proptosis in comparison to patients treated with placebo (10-20%).

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

  • Sex: TEPEZZA worked similarly in men and women.
  • Race: The majority of patients were White. The number of patients in other races were limited; therefore, differences in how TEPEZZA worked among races could not be determined.
  • Age: TEPEZZA worked similarly in patients above and below 65 years of age.

What are the possible side effects?

TEPEZZA may cause serious side effects including infusion reactions, worsening of inflammatory bowel disease, and high blood sugar.

The most common side effects of TEPEZZA are muscle spasm, nausea, hair loss, diarrhea, fatigue, high blood sugar, dry skin, change in taste, and headache.

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

  • Sex: Occurrence of side effects was similar in men and women.
  • Race: The majority of patients were White. The number of patients in other races were limited; therefore, differences in side effects among races could not be determined.
  • Age: The occurrence of side effects was similar in patients above and below 65 years of age.

WHO WAS IN THE CLINICAL TRIALS?

Who participated in the clinical trials?

The FDA approved TEPEZZA based on evidence from two clinical trials (Trial 1/ NCT01868997 and Trial 2/ NCT03298867) of 170 patients with active thyroid eye disease. The trials were conducted at 28 sites in Europe and United States.

Figure 1 summarizes by sex how many patients were in the combined clinical trials.

Figure 1. Baseline Demographics by Sex

Pie chart summarizing how many men and women were in the clinical trial. In total, 124 women (73%) and 46 men (27%) participated in the clinical trial.

Adapted from FDA Review

Figure 2 summarizes patients by race in the clinical trials.

Figure 2. Baseline Demographics by Race

Pie chart summarizing the percentage of patients by race enrolled in the clinical trial. In total, 147 White (86%), 6 Asian (4%) and Black or African American 14 (8%) and 3 Other (2%).

*includes Native Hawaiian or other Pacific Islander and Other

Adapted from FDA Review

Figure 3 summarizes patients by age in the clinical trials.

Figure 3. Baseline Demographics by Age

Pie charts summarizing how many individuals of certain age groups were enrolled in the clinical trial. In total,  144 (85%) were less than 65 years, and 26 (15%) of patients were 65 years and older.

Adapted from FDA Review

Figure 4 summarizes patients by ethnicity in the clinical trials.

Figure 4. Baseline Demographics by Ethnicity

Pie charts summarizing how many individuals of certain ethnicity were enrolled in the clinical trial. In total,  9 patients were Hispanic or Latino (5%), and 161patients were not Hispanic or Latino (95%).

Adapted from FDA Review

How was the trial designed?

There were two trials that provided data for TEPEZZA approval. Both trials enrolled patients with active thyroid eye disease.

Patients received TEPEZZA or placebo by intravenous infusion every three weeks for a total of 8 infusions.

After 24 weeks, the trials compared the percentage of patients who achieved greater than 2 mm reduction in proptosis between TEPEZZA and placebo.

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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