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  5. Drug Trials Snapshot: Orladeyo
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Drug Trials Snapshot: Orladeyo

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  ORLADEYO Package Insert for complete information.

ORLADEYO (berotralstat)
(or-luh-DAY-oh)
BioCryst Pharmaceuticals, Inc.
Approval date: December 3, 2020


DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

ORLADEYO is a drug used to prevent attacks of hereditary angioedema (HAE) in patients 12 years and older.

Hereditary angioedema is a rare, inherited and sometimes life-threatening condition with repeat episodes (attacks) of severe swelling in various parts of the body, including stomach, limbs, face and throat.

How is this drug used?

ORLADEYO is a capsule taken by mouth, 1 time every day with food.

What are the benefits of this drug?

Patients who received ORLADEYO had fewer angioedema attacks compared to those receiving the placebo.

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

  • Sex: ORLADEYO worked similarly in males and females.
  • Race: The majority of patients were White. The number of patients of other races was limited; therefore, differences in response among races could not be determined.
  • Age: The majority of patients were adults 18 to 64 years of age. The number of adolescents and patients 65 years and older was limited; therefore, differences in the occurrence of side effects among younger and older age groups could not be determined.

What are the possible side effects?

In people who take more than one capsule a day, ORLADEYO may cause heart rhythm problem due to prolongation of heart electrical activity (QT prolongation).

The most common side effects of ORLADEYO are abdominal pain, vomiting, diarrhea, back pain, and heartburn.

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

  • Sex: The occurrence of side effects was similar in males and females.
  • Race: The majority of patients were White. The number of patients of other races was limited; therefore, differences in the occurrence of side effects among races could not be determined.
  • Age: The majority of patients were adults 18 to 64 years of age. The number of adolescents and patients 65 years and older was limited; therefore, differences in the occurrence of side effects among younger and older age groups could not be determined.

WHO WAS IN THE CLINICAL TRIALS?

Who participated in the clinical trials?

The FDA approved ORLADEYO based on evidence from one clinical trial (Trial 1 /NCT03485911) of 120 patients with hereditary angioedema. The trial was conducted at 40 sites in the United States, European Union and Canada.

Figure 1 summarizes how many males and females were in the clinical trial.

Figure 1. Baseline Demographics by Sex

Pie chart summarizing how many men and women were in the clinical trials. In total, 41 men (34%) and 79 women (66%) participated in the clinical trial.

FDA Review

Figure 2 summarizes the percentage of patients by race in the clinical trial.

Figure 2. Baseline Demographics by Race

Pie chart summarizing how many patients of different races were in the clinical trial. In total, 113 patients were White (94%), 4 patients were Black or African American (3%), 1 patient was Asian (1%), and 2 patients were Other (2%).

FDA Review

Figure 3 summarizes the percentage of patients by age in the clinical trial.

Figure 3. Baseline Demographics by Age

Pie chart summarizing how many individuals of certain age groups were in the clinical trial.  In total, 6 patients were between 12-17 years old (5%), 105 patients were between 18-64 years old (87%), and 9 patients were between 65-74 years old (8%).

FDA Review

Figure 4 summarizes the percentage of patients by ethnicity in the clinical trial.

Figure 4. Baseline Demographics by Ethnicity

Pie chart summarizing how many individuals of certain ethnicity groups were in the clinical trial.  In total, 3 patients were Hispanic or Latino (2%), 114 patients were not Hispanic or Latino (95%), and for 3 patients not reported (3%).

FDA Review

How were the trials designed?

The benefit and side effects of ORLADEYO were evaluated in one clinical trial. Trial investigators evaluated patients 12 years and older with hereditary angioedema for 8 weeks to determine the number of attacks for each patient. The trial enrolled only patients who had at least 2 attacks during the 8-week period. Patients were assigned to receive one of two doses of ORLADEYO or placebo once every day for 24 weeks. Neither the patients nor the investigators knew which treatment was being given until after the trial was completed. All patients could use other medications for treatment of attacks.

Investigators recorded the number of angioedema attacks in all treated groups over the 24 weeks. The benefit of ORLADEYO was assessed by comparing the reduction rate of HAE attacks relative to placebo over a 24-weeks treatment period.

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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