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Drug Trials Snapshots: RIVFLOZA

HOW TO USE THIS SNAPSHOT
The information provided in Snapshots highlights who participated in the key clinical trials that supported the original FDA approval of this drug, and whether there were differences among sex, race, age, and ethnic 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 healthcare provider about the benefits and risks of a drug.

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 RIVFLOZA Prescribing Information for all 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).

RIVFLOZA (nedosiran)
(Riv-flo-za)
Novo Nordisk, Inc.
Approval date: September 29, 2023


DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

RIVFLOZA reduces urine oxalate in children 9 years of age and older and adults with primary hyperoxaluria type 1 (PH1) and relatively preserved kidney function.

PH1 is a rare disease in which urine oxalate is too high, which over time can harm the kidneys.

How is this drug used?

RIVFLOZA is an injection given under the skin (subcutaneously) once a month.

Who participated in the clinical trials?

FDA approved RIVFLOZA based on evidence from a clinical trial which included 29 patients with PH1. The trial was conducted at 19 sites in 11 countries in North America, Europe, Asia, and Australia.

How were the trials designed?

RIVFLOZA was evaluated in one clinical trial of 29 patients with PH1 who were 9 years of age and older. Patients randomly received either RIVFLOZA or placebo injections once a month for six months. Neither the patients nor the healthcare providers knew which treatment was being given. The benefit of RIVFLOZA was assessed by measuring the amount of oxalate in the urine and comparing it to placebo.


DEMOGRAPHICS SNAPSHOT

Figure 1 summarizes how many males and females were enrolled in the clinical trial used to evaluate the efficacy of RIVFLOZA.

Figure 1. Baseline Demographics by Sex

Pie chart summarizing how many male and female patients were in the clinical trial. In total, 13 (45%) male patients and 16 (55%) female patients participated in the clinical trial.

Source: Adapted from FDA Review

Figure 2 summarizes how many patients by race enrolled in the clinical trial used to evaluate the efficacy of RIVFLOZA.

Figure 2. Baseline Demographics by Race

Pie chart summarizing how many White, Asian, and other patients were in the clinical trial. In total, 20 (69%) White patients, 5 (17%) Asian patients, and 4 (14%) other patients participated in the clinical trial.

Source: Adapted from FDA Review

Figure 3 summarizes how many patients by age were enrolled in the clinical trial used to evaluate the efficacy of RIVFLOZA.

Figure 3. Baseline Demographics by Age

Pie chart summarizing how many patients by age were in the clinical trial. In total, 3 (10%) patients between 9 and 11 years of age, 8 (28%) patients between 12 and 17 years of age, and 18 (62%) patients 18 years of age or older participated in the clinical trial.

Source: Adapted from FDA Review

Figure 4 summarizes how many patients by ethnicity enrolled in the clinical trial used to evaluate the efficacy of RIVFLOZA.

Figure 4. Baseline Demographics by Ethnicity

Pie chart summarizing how many Hispanic and not Hispanic patients were in the clinical trial. In total, 2 (8%) Hispanic or Latino patients and 24 (92%) not Hispanic or Latino patients participated in the clinical trial.

Source: Adapted from FDA Review

What are the benefits of this drug?

One trial was conducted in participants 9 years of age and older with PH1. In this trial, patients treated with RIVFLOZA had a 50% decrease in urinary oxalate after six months of treatment compared with a 6% increase in patients who received placebo.

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

  • Sex: The observed effect of RIVFLOZA was similar for females and males.
  • Race: The number of patients of races other than White was small; therefore, differences in how RIVFLOZA worked among races could not be determined.
  • Age: The observed effect of RIVFLOZA was similar in patients 9 to 11 years of age, 12 to 17 years of age, and adults.
  • Ethnicity: The number of Hispanic or Latino patients was small; therefore, differences in how RIVFLOZA worked according to ethnicity could not be determined.

What are the possible side effects?

The most common side effects of RIVFLOZA are injection site reactions, which were reported in seven patients with PH1 (39%) on RIVFLOZA as compared to no patients on placebo.

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

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

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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