Familial chylomicronemia syndrome (FCS) is a rare genetic condition that makes it hard for your body to break down fats called triglycerides. When these fats build up in your blood, your triglyceride levels can get very high. This can lead to serious problems like pancreatitis and cause damage to other parts of the body. If you have FCS, you will need to follow a strict low-fat diet and take medicine to lower your triglyceride levels. Redemplo (plozasiran) is a medicine to help lower your triglycerides. Redemplo is given as an injection under your skin (subcutaneously) once every three months. You or a caregiver can be trained to give these injections at home.
How Does Redemplo Work for FCS?
Redemplo lowers how much apolipoprotein C-III (APOC-III) your body makes through a process called RNA interference. APOC-III normally lowers the amount of triglycerides your body can break down. When Redemplo lowers APOC-III, it allows your body to break down more triglycerides. This lowers triglyceride levels in your blood. By lowering triglycerides, this may help reduce your risk of pancreatitis or other problems linked to high levels of triglycerides.
You must stay on a low-fat diet while using Redemplo. Your diet should include no more than 20 grams of fat each day.
How Do I Use Redemplo?
Redemplo is supplied in a prefilled syringe that can be stored in the refrigerator or at room temperature.
- If you store Redemplo at room temperature, it must be used within 30 days.
- If you store Redemplo in the refrigerator, it can be used before the expiration date. You should remove it from the refrigerator 30 minutes before using it.
You, a caregiver, or a health care provider will inject Redemplo under your skin (subcutaneously) every three months (or as your health care provider instructs you to) at any of the following three sites:
- Thigh
- Stomach (at least 2 inches away from your belly button)
- Upper arm (You should not give yourself injections using this site. The back of the upper arm should only be used if a caregiver or health care provider is giving you the injection.)
Do not inject Redemplo into skin that has scars, stretch marks, or bruises. Clean the injection site with an alcohol wipe before injecting the medicine. Follow these steps when you are ready to inject Redemplo.
- Pinch the skin and insert the needle at a 45- to 90-degree angle.
- Inject the medicine.
- After you have injected all of the medicine, remove the needle from the skin.
- Dispose of the syringe and needle in a proper disposal container.
How Was Redemplo Studied for FCS?
A clinical trial called PALISADE was done to examine the safety and effectiveness of Redemplo for the treatment of FCS. To be able to participate in the trial, people had to have very high triglyceride levels, such as fasting triglyceride levels higher than 1,000 milligrams per deciliter (mg/dL) on at least three occasions. Although the triglyceride levels are suggestive of FCS, the participants did not need to have FCS confirmed by a genetic test for this study. People with diabetes and uncontrolled blood sugar levels, those using steroid medicines, and those who had long-term kidney problems were not allowed in the study.
People in the study were either given Redemplo 25 mg, Redemplo 50 mg, or a placebo containing no medicine. The treatment was given subcutaneously every three months for one year. Neither the investigators nor the participants knew who was getting Redemplo or a placebo.
There were 75 people included in the PALISADE study. People in the study had the following characteristics at the beginning of the study:
- 51% were women.
- 73% were White, 21% were Asian, about 1% were American Indian or Alaskan Native, and the rest did not specify their race.
- The median (middle when lined up from low to high) triglyceride level was 2,044 mg/dL.
- 59% of people had FCS confirmed with genetic testing, and 41% had clinically diagnosed FCS that was not confirmed with genetic testing.
- 45% were taking a statin medicine.
- 67% were taking a fibrate medicine.
- 37% had diabetes or prediabetes.
- 89% had a history of pancreatitis.
What Benefits Were Seen in the Study?
How well Redemplo worked was measured by the change in fasting triglyceride levels from the beginning to 10 months after the study started. People treated with Redemplo had greater reductions in their triglyceride levels than people treated with a placebo:
- 80% decrease in median triglyceride levels with Redemplo 25 mg
- 78% decrease in median triglyceride levels with Redemplo 50 mg
- 17% decrease in median triglyceride levels with placebo
Other important benefits of Redemplo included fewer episodes of acute pancreatitis and lower apolipoprotein C-III levels.
- About 4% of people treated with Redemplo had acute pancreatitis compared with 20% treated with placebo (dose of Redemplo not specified).
- Apolipoprotein C-III levels decreased by a median of 1% with placebo, 93% with Redemplo 25 mg, and 96% with Redemplo 50 mg after 10 months of treatment.
Your results may differ from what was seen in clinical studies.
How Can I Prevent and Manage Common Side Effects of Redemplo?
The most common side effects of Redemplo include high blood sugar (hyperglycemia), headache, nausea, and injection site reactions. Although not all side effects can be prevented, you may be able to prevent or control some side effects.
Injection site reactions. Injection site reactions can include pain, redness, or swelling where you inject Redemplo. You can help reduce your risk for injection site reactions by carefully washing your hands and cleaning the injection site with an alcohol swab before injecting. You should remove Redemplo from the refrigerator 30 minutes before injection to help reduce pain when injecting. You should avoid injecting in areas of the skin that are irritated, scarred, or bruised.
You can also try these things to provide relief if you have any injection site reactions:
- Apply a cold or warm compress.
- Avoid scratching or rubbing the area.
- Ask your health care provider about using an over-the-counter pain medicine or antihistamines to help reduce pain and itching.
Hyperglycemia. In the PALISADE study, hyperglycemia occurred in some people who had diabetes or prediabetes. It is not known why this occurs, but it may be related to the breakdown of triglycerides. If you have symptoms of high blood sugar levels, such as thirst, headache, blurred vision, frequent peeing, tiredness, weight loss, or trouble concentrating, you should contact your health care provider.
How Long Does It Take for Redemplo to Work?
In the clinical study, people began to see improvements in their fasting triglyceride levels right away, with the largest change occurring in the first month. These results continued through 12 months.
Your results may differ from what was seen in clinical studies.
How Do I Get Redemplo?
Redemplo is available exclusively through a specialty pharmacy.
Your health care provider will get you started with Redemplo by completing the Redemplo Start Form. This form contains all the information your health plan needs to determine coverage.
You can enroll in the Rely On Redemplo Patient Support Program. This program provides you with a starter kit, additional injection training, and access to other resources. They can assist you with your insurance and provide information about any financial help that may be available. You can call 844-733-3675 or visit this website for support.