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  5. Drugs Trials Snapshot: Oxlumo
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Drugs Trials Snapshot: Oxlumo

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 OXLUMO Prescribing Information for complete information.

OXLUMO (lumasiran)
(ox loo’ moe )
Alnylam Pharmaceuticals
Approval date: November 23, 2020


DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

OXLUMO is a drug used to lower the level of urine oxalate in children and adults with primary hyperoxaluria type 1 (PH1).

PH1 is a rare, inherited disorder in which the liver makes too much oxalate. Excess oxalate can lead to kidney stones and kidney damage. Over time, oxalate can also build up in the body and damage other organs, including the heart, bones and eyes.

How is this drug used?

OXLUMO is an injection given under the skin (subcutaneously) once a month for the first three months. Some patients can then switch to an injection once every three months depending on their weight.

What are the benefits of this drug?

In one trial, patients treated with OXLUMO had a 65% decrease in urinary oxalate after 6 months of treatment compared with a 12% decrease in patients who received placebo. In a second trial, patients treated with OXLUMO had a decrease in urinary oxalate of 71%.

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

The trials that looked at the benefit of OXLUMO were too small to determine if there were differences among sex, race, and age subgroups.

What are the possible side effects?

The most common side effects of OXLUMO are injection site reactions.

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

The trials that looked at the side effects of OXLUMO were too small to determine if there were differences among sex, race, and age subgroups.

WHO WAS IN THE CLINICAL TRIALS?

Who participated in the trials?

The FDA approved OXLUMO based on two clinical trials (Trial 1/ NCT03681184 and Trial 2/ NCT03905694) of 55 patients with PH1. The trials were conducted at 25 centers in the United States, Europe, and the Middle East.

Figure 1 summarizes how many male and female patients were in the clinical trials.

Figure 1. Demographics by Sex (efficacy population)

Pie chart summarizing how many men and women were in the clinical trials. In total, 33 men (60%) and 22 women (40%) participated in the clinical trial.

Adapted from FDA Review

Figure 2 summarizes by race how many patients were in the clinical trials.

Figure 2. Demographics by Race (efficacy population)

Pie chart summarizing how many patients of different races were in the clinical trial. In total, 44 patients were White (80%), 6 patients were Asian (11%), and 5 patients were Other (9%).

Adapted from FDA Review

The figure below summarizes the age groups of patients in the clinical trials.

Figure 3. Demographics by Age (efficacy population)

Pie chart summarizing how many individuals of certain age groups were in the clinical trial.  In total, 16 patients were less than 6 years old (29%), 16 patients were 6-11 years old (29%), 6 patients were 12-17 years old (11%), and 17 patients were 18 years and older (31%).

Adapted from FDA Review

The figure below summarizes the patients by ethnicity in the clinical trials.

Figure 4. Demographics by Ethnicity (efficacy population)

Pie chart summarizing how many individuals of certain ethnicity groups were in the clinical trial.  In total, 2 patients were Hispanic or Latino (4%) and 53 patients were not Hispanic or Latino (96%).

Adapted from FDA Review

How were the trials designed?

Trial 1 enrolled patients with PH1 who were 6 years of age or older. Patients randomly received either OXLUMO or placebo injections once a month for the first three months and then once every three months. Neither the patients nor the healthcare providers knew which treatment was being given until after the trial was completed. The benefit of OXLUMO was assessed by measuring the amount of oxalate in the urine over 24 hours and comparing it to placebo.

Trial 2 enrolled pediatric patients with PH1 who were younger than 6 years. All patients received OXLUMO for 6 months. The benefit of OXLUMO was assessed by measuring the amount of oxalate in a urine sample.

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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