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 REDEMPLO 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).

REDEMPLO (plozasiran) injection 
(ree-DEM-plo) 
Arrowhead Pharmaceuticals, Inc. 
Approval date: November 18, 2025

DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

REDEMPLO is a prescription medicine (an apolipoprotein C-III [APOC-III]–directed small interfering ribonucleic acid) that is used along with a low-fat diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS).

FCS is a rare genetic disorder that prevents the body from breaking down fats.

How is this drug used?

REDEMPLO is a subcutaneous injection that is taken once every three months.

Who participated in the clinical trials?

The FDA approved REDEMPLO based on evidence from a clinical trial (Trial 1; NCT05089084) of 75 patients with FCS. The trial was conducted at 54 sites in 20 countries in North America, Europe, Australia, Asia, and South America. Of the 75 patients, 9 patients were from trial sites in the United States.

The benefits and side effects of REDEMPLO for patients with FCS were evaluated in the same single clinical trial. Additional trials in patients with hypertriglyceridemia were used to support the safety assessment. The number of patients representing efficacy findings may differ from the number of patients representing safety findings due to different pools of study participants analyzed for efficacy and safety.

How were the trials designed?

REDEMPLO was evaluated in one clinical trial of 75 patients with FCS.

Enrolled participants were already using other treatments to lower triglycerides, including a low-fat diet and drugs (such as fenofibrates, omega-3 fatty acids, and statins). Participants were randomly assigned to receive REDEMPLO or placebo every three months for one year. Neither the participants nor the health care providers knew which treatment was being given.

The trial measured percent change in triglyceride from baseline (before treatment) to Month 10 and compared REDEMPLO to placebo.

How were the trials designed?

The efficacy of REDEMPLO was demonstrated in a randomized, placebo-controlled, double-blinded clinical trial in adult patients with genetically or clinically diagnosed FCS and fasting triglyceride levels ≥880 mg/dL (Trial 1; NCT05089084). In this trial, for clinically diagnosed FCS, patients needed at least one of the following: recurrent episodes of pancreatitis not caused by alcohol or gallstones, recurrent hospitalizations for severe abdominal pain without other explainable cause, childhood pancreatitis, or a family history of pancreatitis caused by high triglycerides. After a ≥4-week stabilization period where patients continued to follow a diet with ≤20 grams of fat per day, patients were randomly assigned to receive doses every three months of REDEMPLO 25 mg (n=26) or placebo (n=25) via subcutaneous injection over a 12-month treatment period. The primary endpoint was percent change in fasting triglyceride from baseline to Month 10 (average of two assessments, two to seven days apart) compared to placebo.

DEMOGRAPHICS SNAPSHOT

Figure 1 summarizes how many male and female patients were enrolled in the clinical trial used to evaluate the efficacy of REDEMPLO.

Figure 1. Baseline Demographics by Sex, Full Analysis Set

Source: Adapted from FDA Review

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

Figure 2. Baseline Demographics by Race, Full Analysis Set

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 REDEMPLO.

Figure 3. Baseline Demographics by Age, Full Analysis Set

Source: Adapted from FDA Review

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

Figure 4. Baseline Demographics by Ethnicity, Full Analysis Set

Source: Adapted from FDA Review

Who participated in the trials?

Table 1. Baseline Demographics, Trial 1, Full Analysis Set

Baseline Demographics REDEMPLO 25 mg
N=26		 REDEMPLO 50 mg*
N=24		 Placebo
N=25
Sex, n (%)      
Female 14 (53.8) 13 (54.2) 11 (44.0)
Male 12 (46.2) 11 (45.8) 14 (56.0)
Age, years      
Mean (SD) 47.9 (14.4) 42.6 (10.9) 47.4 (13.9)
Median (min, max) 46.5 (22, 76) 40.5 (24, 68) 44 (26, 71)
Age group, years, n (%)      
18 to <65 22 (84.7) 22 (91.7) 22 (88.0)
≥65 4 (15.4) 2 (8.3) 3 (12.0)
Race, n (%)      
White 19 (73.1) 17 (70.8) 19 (76.0)
Asian 7 (26.9) 5 (20.8) 4 (16.0)
American Indian or Alaska Native 0 1 (4.2) 0
Other 0 1 (4.2) 2 (8.0)
Ethnicity, n (%)      
Hispanic or Latino 0 1 (4.2) 1 (4.0)
Not Hispanic or Latino 26 (100) 22 (91.7) 24 (96.0)
Unknown 0 1 (4.2) 0

Source: Adapted from FDA Review
* REDEMPLO 50 mg is not an approved dosing regimen
Abbreviations: SD, standard deviation

What are the benefits of this drug?

In the clinical trial, participants who received REDEMPLO had a 59% reduction in triglycerides compared to participants who received placebo after 10 months of treatment.

What are the benefits of this drug (results of trials used to assess efficacy)?

Table 1 summarizes changes in triglycerides at Month 10 for REDEMPLO compared to placebo.

Table 2. Percent Change in Triglycerides After 10 Months of Treatment in Participants With FCS on Other Triglyceride-Lowering Therapies, Trial 1, Efficacy Population

Parameter REDEMPLO 25 mg
N=26		 Placebo
N=25		 Treatment Difference 
(95% CI)
Median percent change in triglycerides -80 -17 -59 (-90, -28)

Source: Adapted from FDA Review
Abbreviations: CI, confidence interval; FCS, familial chylomicronemia syndrome; N, total number of subjects

Figure 5 shows the absolute change in triglycerides over time.

Figure 5. Fasting Triglycerides (mg/dL) Over Time, Trial 1

Source: Adapted from FDA Review

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

  • Sex: The observed effect of REDEMPLO 25 mg was similar for females and males.
  • Race: The observed effect of REDEMPLO 25 mg was similar between White and Asian patients. However, the number of patients of races other than White and Asian was small, therefore differences in how REDEMPLO 25 mg worked among races could not be determined.
  • Age: The number of patients older than 65 years of age was small, therefore differences in how REDEMPLO 25 mg worked in patients younger and older than 65 years of age could not be determined.
  • Ethnicity: The number of patients with an ethnicity other than Not Hispanic was low, therefore differences in how REDEMPLO 25 mg worked by a patient’s ethnicity could not be determined.

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

Table 3. Efficacy Results by Subgroup for REDEMPLO 25 mg, Trial 1, Efficacy Population

Group Subgroup Treatment Effect*
Mean (95% CI)
Sex  Female  -62 (-96, -26)
Male  -63 (-94, -32)
Race White -54 (-86, -23)
Asian -53 (-90, -18)

Source: Adapted from FDA Review
* Treatment differences and credible intervals include the relevance of outcomes from other subgroups.
Abbreviations: CI, credible interval

What are the possible side effects?

The most common side effects include high blood sugar, headache, nausea, and injection site reactions.

What are the possible side effects (results of trials used to assess safety)?

Table 4. Adverse Reactions Occurring in ≥10% of Patients Treated With REDEMPLO and >5% More Than Treated With Placebo, Trial 1, Safety Population

Adverse Reaction  Total REDEMPLO
N = 50
n (%)		 Placebo
N = 25
n (%)
Hyperglycemiaa 10 (20%) 2 (8%)
Headache 8 (16%) 2 (8%)
Nausea 7 (14%) 2 (8%)
Injection site reactiona 5 (10%) 1 (4%)

Source: Adapted from FDA Review
a Grouped terms composed of several similar terms

The laboratory measures and the direction of change associated with REDEMPLO treatment include:

  • Glucose levels may increase
  • Liver enzymes may increase
  • LDL-C may increase

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

Important differences in side effects across subgroups was not observed. However, the number of patients was small and therefore the ability to detect meaningful treatment differences across these subgroups is limited.

  • Sex: The occurrence of side effects was similar in females and males.
  • Race: The occurrence of side effects of REDEMPLO 25 mg was similar between White and Asian patients. However, the number of patients of races other than White and Asian was small; therefore differences in side effects among other races could not be determined.
  • Age: The number of patients older than 65 years was low, therefore differences in side effects by age could not be determined.
  • Ethnicity: The number of patients with an ethnicity other than Not Hispanic was low, therefore differences in the occurrence of side effects by ethnicity could not be determined.

Were there any differences in side effects of the clinical trials among sex, race, and age groups?

Table 5. Side Effects by Subgroup, Trial 1, Safety Population

Characteristic REDEMPLO 25 mg
N=26
n/Ns (%)		 Placebo
N=25
n/Ns (%)
Sex    
Female 13/14 (92.9) 11/11 (100)
Male 10/12 (83.3) 9/14 (64.3)
Age group, years    
<50 14/15 (93.3) 10/14 (71.4)
50 to <65 6/7 (85.7) 7/8 (87.5)
≥65 3/4 (75.0) 3/3 (100)
Race    
American Indian or Alaska Native 0/0 (NA) 0/0 (NA)
Asian 7/7 (100) 3/4 (75.0)
Other 0/0 (NA) 2/2 (100)
White 16/19 (84.2) 15/19 (78.9)

Source: Adapted from FDA Review
Abbreviations: N, number of patients in treatment arm; n, number of patients with adverse event; NA, not applicable; Ns, total number of patients for each specific subgroup and were assigned to that specific arm

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.