Tryngolza (Olezarsen)
Antisense oligonucleotide for familial chylomicronemia syndrome (FCS)
Quick Facts About Tryngolza
Key Takeaways About Tryngolza
- Targeted for FCS: Tryngolza is specifically approved for adults with genetically confirmed familial chylomicronemia syndrome, a rare condition affecting approximately 1-2 per million people
- Once-monthly injection: A single 80 mg subcutaneous injection given on the same day each month makes for a convenient dosing schedule
- Novel mechanism: Olezarsen blocks production of apolipoprotein C-III (apoC-III), which normally slows triglyceride clearance, helping reduce dangerously high triglyceride levels
- Reduces pancreatitis risk: By lowering triglyceride levels, Tryngolza helps reduce the frequency of acute pancreatitis episodes, a potentially life-threatening complication of FCS
- Continue low-fat diet: Tryngolza must be used alongside dietary fat restrictions; it does not replace the need for a strict low-fat diet in FCS patients
What Is Tryngolza and What Is It Used For?
Tryngolza (olezarsen) is a lipid-modifying medicine that changes the way your body breaks down fats. It is used together with dietary fat restrictions to treat adults aged 18 and older with familial chylomicronemia syndrome (FCS), a rare genetic disorder causing extremely high blood triglyceride levels that can lead to recurrent, potentially life-threatening pancreatitis.
Familial chylomicronemia syndrome is an inherited condition in which the body cannot properly break down certain fats called triglycerides. People with FCS carry mutations in genes responsible for triglyceride metabolism, most commonly in the lipoprotein lipase (LPL) gene. As a result, triglyceride-rich lipoproteins called chylomicrons accumulate in the bloodstream, sometimes reaching levels above 10,000 mg/dL (compared to the normal range below 150 mg/dL). This extreme hypertriglyceridemia puts patients at constant risk of acute pancreatitis, a painful and dangerous inflammation of the pancreas.
Olezarsen belongs to a class of medicines known as antisense oligonucleotides. It works by targeting the messenger RNA (mRNA) that carries instructions for producing a protein called apolipoprotein C-III (apoC-III). Under normal circumstances, apoC-III acts as a brake on the body's ability to clear triglycerides from the blood. By blocking the production of apoC-III, olezarsen effectively releases this brake, allowing the body to remove triglycerides more efficiently. This dual mechanism works through both lipoprotein lipase-dependent and independent pathways, making it particularly suitable for FCS patients who lack functional LPL.
Tryngolza is prescribed only after genetic testing has confirmed a diagnosis of FCS. It is typically considered when other approaches, including strict adherence to a very low-fat diet (typically less than 20 grams of fat per day) and other lipid-lowering medications, have not achieved adequate triglyceride reduction. The medicine does not cure FCS, but by significantly lowering triglyceride levels it can reduce the frequency and severity of acute pancreatitis episodes, thereby improving quality of life for patients living with this challenging rare disease.
FCS affects approximately 1-2 people per million worldwide. Unlike the more common multifactorial chylomicronemia syndrome (MCS), FCS is caused by specific genetic mutations (most often in the LPL, GPIHBP1, LMF1, APOC2, or APOA5 genes) and is present from birth. Accurate genetic diagnosis is essential because treatment strategies differ significantly between FCS and MCS.
What Should You Know Before Taking Tryngolza?
Before starting Tryngolza, tell your doctor about any liver or kidney problems, low platelet counts, allergies, pregnancy, or breastfeeding. Tryngolza is not approved for use in children under 18 years. You must have genetically confirmed FCS to receive this treatment.
Contraindications
You must not use Tryngolza if you are allergic (hypersensitive) to olezarsen or any of the other ingredients in this medicine. The inactive ingredients include sodium dihydrogen phosphate, disodium hydrogen phosphate, sodium chloride, water for injections, sodium hydroxide, and hydrochloric acid. If you have experienced a previous allergic reaction to any antisense oligonucleotide therapy, discuss this with your doctor before starting Tryngolza.
Warnings and Precautions
Speak to your doctor, pharmacist, or nurse before using Tryngolza if you have any of the following health conditions:
- Liver problems: Any form of liver disease or impaired liver function, as the medicine is metabolised in the liver and may affect liver enzyme levels
- Kidney problems: Renal impairment of any severity, as the medicine is partially excreted through the kidneys
- Low platelet count (thrombocytopenia): Platelets are blood cells that help your blood clot. Low platelet counts may increase the risk of bleeding, and Tryngolza may affect platelet levels
Your doctor will perform regular blood tests to monitor your liver function, kidney function, and platelet counts during treatment. These monitoring tests are essential to ensure the medicine is not causing harmful effects on these organs and blood components.
Tryngolza can cause serious allergic reactions (hypersensitivity) that may be life-threatening. Stop using Tryngolza and seek immediate medical attention if you experience difficulty breathing, tightness in the throat, swelling of the face, lips, mouth, tongue or throat, skin redness, or chills. These symptoms may indicate anaphylaxis, which requires emergency treatment.
Children and Adolescents
Tryngolza must not be used in patients under 18 years of age. Olezarsen has not been studied in children and adolescents, and the safety, efficacy, and appropriate dosing in this population are unknown. If FCS is diagnosed in a child or teenager, the treating physician will recommend alternative management strategies until the patient reaches adulthood.
Drug Interactions
Tell your doctor or pharmacist if you are taking, have recently taken, or might take any other medicines. Tryngolza can be used alongside other lipid-lowering medications, including statins and fibrates. However, because all of these medicines affect fat metabolism through different pathways, your doctor needs to know your complete medication list to monitor for any cumulative effects on liver function or muscle health.
While no clinically significant drug interactions have been identified in clinical trials, olezarsen is an antisense oligonucleotide that does not undergo cytochrome P450 metabolism. This means it is less likely to interact with medicines that are metabolised through this common liver enzyme pathway. Nevertheless, always inform your healthcare team about all prescription and over-the-counter medicines, herbal supplements, and vitamins you are taking.
| Drug Class | Interaction Level | Clinical Note |
|---|---|---|
| Statins (e.g., atorvastatin, rosuvastatin) | Low - can be co-administered | Monitor liver function tests; combined lipid-lowering effect |
| Fibrates (e.g., fenofibrate, gemfibrozil) | Low - can be co-administered | Both lower triglycerides; monitor for myopathy if combined with statins |
| Anticoagulants (e.g., warfarin) | Monitor | Monitor platelet count; Tryngolza may affect platelets |
| Antiplatelet agents (e.g., aspirin, clopidogrel) | Monitor | Increased bleeding risk if platelets are reduced |
| Omega-3 fatty acids | Low - can be co-administered | Complementary triglyceride-lowering mechanisms |
Pregnancy and Breastfeeding
If you are pregnant or breastfeeding, think you may be pregnant, or are planning to have a baby, ask your doctor for advice before using this medicine. The safety of Tryngolza during pregnancy has not been established, and it is not known whether olezarsen can harm an unborn baby.
As a precautionary measure, it is preferable to avoid using Tryngolza during pregnancy. Women of childbearing potential should use effective contraception throughout treatment. If you become pregnant while taking Tryngolza, stop the medicine and contact your doctor immediately.
It is unknown whether olezarsen passes into breast milk or whether it could affect a breastfeeding infant. Your doctor will weigh the benefits of treatment against the potential risks to the nursing infant and advise whether you should continue treatment or breastfeeding, but not both simultaneously.
Driving and Using Machines
Tryngolza has no or negligible effect on the ability to drive and use machines. You do not need to take special precautions regarding driving or operating machinery while using this medicine.
This medicine contains less than 1 mmol sodium (23 mg) per 80 mg dose, which means it is essentially sodium-free. This is relevant for patients on a controlled sodium diet.
What Is the Correct Dosage of Tryngolza?
The recommended dose of Tryngolza is 80 mg given as a subcutaneous injection once a month, on the same day each month. The injection is given under the skin of the abdomen, front of the thigh, or back of the upper arm (by a healthcare professional or caregiver only). Continue your prescribed low-fat diet during treatment.
Always use this medicine exactly as your doctor, pharmacist, or nurse has instructed. Do not change the dose or stop treatment without consulting your healthcare team. Tryngolza is designed to be used as a long-term treatment, and consistent monthly dosing is important for maintaining reduced triglyceride levels.
Adults (18 years and older)
Dose: 80 mg subcutaneous injection once monthly
Schedule: Same day each month
Administration: Pre-filled single-dose pen, for single use only
Injection sites: Abdomen (at least 5 cm from navel), front of thigh, or back of upper arm (caregiver/healthcare professional only)
Children and Adolescents (under 18 years)
Not recommended. Tryngolza has not been studied in patients under 18 years of age. Safety and efficacy have not been established in this age group.
Elderly Patients
No dose adjustment required. The same 80 mg once-monthly dose applies. Your doctor may perform more frequent monitoring of liver function, kidney function, and platelet counts.
How to Inject Tryngolza
Each Tryngolza pre-filled pen contains one dose of 80 mg in 0.8 mL of solution. The pen is designed for single use and must be discarded after the injection. Before your first self-injection, your healthcare provider will demonstrate the correct technique.
- Prepare: Remove the pen from the refrigerator and let it reach room temperature (up to 30°C) for 30 minutes. Do not use a microwave or hot water to warm it.
- Inspect: Check the expiration date. Look through the viewing window – the solution should be clear, colourless to yellow. Air bubbles are normal. Do not use if frozen, cloudy, or containing particles.
- Choose a site: Select the abdomen (avoiding 5 cm around the navel) or front of the thigh. Caregivers may use the back of the upper arm. Avoid bruised, red, tender, hard, scarred, or damaged skin.
- Clean: Wash hands with soap and water. Clean the injection site with an alcohol swab in a circular motion and let it air dry.
- Remove cap: Pull the clear cap straight off (do not twist). The needle is inside the orange needle shield.
- Inject: Place the orange needle shield against the skin at 90 degrees. Press firmly until you hear a click. Hold for 10 seconds.
- Check and dispose: Verify the orange plunger fills the entire window. Lift straight up. Dispose in a sharps container.
Do not inject into the area within 5 cm of the navel. Do not inject into skin that is bruised, red, tender, hard, scarred, or damaged. Do not re-cap or reuse the pen. Rotate injection sites between doses to minimise injection site reactions.
Missed Dose
If you miss a dose of Tryngolza, inject the next dose as soon as possible. Then continue with your monthly injections from the date the missed dose was taken. This means your dosing schedule may shift. If you have questions about your dosing schedule, contact your doctor, pharmacist, or nurse for guidance.
Overdose
If you inject too much Tryngolza, contact your doctor, pharmacist, or go to a hospital emergency department immediately, even if you do not have any symptoms. Bring the medicine carton or pen with you. You will be monitored and given supportive care as needed. There is no specific antidote for olezarsen overdose.
Stopping Treatment
Do not stop using Tryngolza without first discussing it with your doctor. Abrupt discontinuation may lead to a rapid rise in triglyceride levels, potentially increasing the risk of acute pancreatitis. Your doctor will advise on the safest way to manage any changes to your treatment plan.
What Are the Side Effects of Tryngolza?
Like all medicines, Tryngolza can cause side effects, although not everybody gets them. The most common side effects are headache, joint pain, redness at the injection site, and vomiting. Serious allergic reactions are possible but uncommon. Contact your doctor immediately if you experience breathing difficulties, throat swelling, or widespread skin reactions.
The side effects reported in clinical trials are categorised below by how frequently they occur. Understanding these frequencies can help you and your doctor make informed decisions about your treatment and know when to seek medical attention.
Serious Side Effects
- Allergic reactions (hypersensitivity): May be life-threatening. Symptoms include difficulty breathing, throat tightness, swelling of face/lips/mouth/tongue/throat, skin redness, and chills
Very Common Side Effects
- Headache
- Pain, tenderness, or stiffness in joints (arthralgia)
- Redness (erythema) at the injection site
- Vomiting
Common Side Effects
- Muscle pain (myalgia)
- Skin discolouration at the injection site
- Chills
- Pain at the injection site
- Swelling at the injection site
Most injection site reactions are mild to moderate in severity and tend to resolve on their own within a few days. Rotating injection sites between doses can help reduce the frequency and severity of local reactions. If injection site reactions persist or worsen, consult your doctor.
If you notice any side effects not listed here, or if any side effects become serious, tell your doctor, pharmacist, or nurse. You can also report suspected side effects directly to your national medicines regulatory authority to help monitor the ongoing safety of this medicine.
Contact your doctor promptly if you experience unexplained bruising or bleeding (which could indicate low platelets), yellowing of the skin or eyes (which could indicate liver problems), or any signs of allergic reaction. Regular monitoring blood tests will help detect potential issues early.
How Should You Store Tryngolza?
Store Tryngolza in a refrigerator at 2-8°C in its original packaging to protect from light. It can be kept at room temperature (up to 30°C) for up to 6 weeks. Keep out of sight and reach of children. Do not use after the expiration date or discard date.
Proper storage of Tryngolza is essential to maintain the medicine's effectiveness and safety. The pre-filled pen contains a biological product that can degrade if exposed to extreme temperatures or prolonged light.
| Storage Condition | Details |
|---|---|
| Refrigerated storage | 2–8°C (36–46°F). Keep in original carton to protect from light. |
| Room temperature storage | Up to 30°C (86°F) for a maximum of 6 weeks. Record discard date on carton. |
| Before injection | Allow to reach room temperature for 30 minutes before use. Do not use heat sources. |
| Do not use if | Solution appears frozen, cloudy, or contains particles. Only use if clear, colourless to yellow. |
| Disposal | Dispose of used pens in a sharps container. Do not discard in household waste or recycling. |
If you have taken Tryngolza out of the refrigerator for room temperature storage, write the discard date on the outer carton. The discard date is a maximum of 6 weeks from the date you removed it from the refrigerator. Do not use the pre-filled pen if either the expiration date on the pen label or the discard date on the carton has passed. Once removed from the refrigerator, do not return the pen to refrigerated storage.
Do not dispose of medicines via wastewater or household waste. Ask your pharmacist how to dispose of medicines you no longer use. These measures help protect the environment and ensure safe handling of pharmaceutical waste.
What Does Tryngolza Contain?
Each Tryngolza pre-filled pen contains 80 mg olezarsen in 0.8 mL solution. The inactive ingredients include phosphate buffers, sodium chloride, and water for injections. The solution is clear, colourless to yellow, and comes in a single-use pre-filled pen.
Active Ingredient
The active substance is olezarsen. Each single-dose pre-filled pen delivers 80 mg of olezarsen in 0.8 mL of solution. Olezarsen is a 2'-O-(2-methoxyethyl)-modified antisense oligonucleotide that selectively targets apolipoprotein C-III mRNA in hepatocytes (liver cells), reducing the production of this protein and thereby enhancing triglyceride clearance from the bloodstream.
Inactive Ingredients (Excipients)
- Sodium dihydrogen phosphate (E 339) – buffering agent
- Disodium hydrogen phosphate (E 339) – buffering agent
- Sodium chloride – tonicity agent
- Water for injections – solvent
- Sodium hydroxide (E 524) – pH adjustment
- Hydrochloric acid (E 507) – pH adjustment
The final solution has a pH adjusted to physiological range for comfortable subcutaneous injection. The medicine contains less than 1 mmol sodium (23 mg) per dose and is essentially sodium-free.
Packaging
Tryngolza is supplied as a clear, colourless to yellow solution for injection in a single-dose pre-filled pen for single use. Each carton contains one pre-filled pen. The pen features a viewing window to inspect the solution, an orange needle shield with safety lock mechanism, and a clear cap that must be removed before injection.
The marketing authorisation holder and manufacturer is Swedish Orphan Biovitrum AB (publ), based in Stockholm, Sweden. For further information about this medicine, contact the marketing authorisation holder or consult the European Medicines Agency (EMA) website.
Frequently Asked Questions About Tryngolza
References and Sources
This article is based on the following evidence-based sources. All medical claims are supported by regulatory approval data and peer-reviewed research.
- European Medicines Agency (EMA). Tryngolza (olezarsen) – Summary of Product Characteristics (SmPC). European public assessment report. EMA/2025.
- Witztum JL, Gaudet D, Freedman SD, et al. Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome. N Engl J Med. 2019;381(6):531-542. doi:10.1056/NEJMoa1715944
- Hegele RA, Borren DR, Engelen L, et al. Familial Chylomicronemia Syndrome: An Under-Recognized Cause of Severe Hypertriglyceridemia. J Clin Lipidol. 2020;14(1):5-12.
- Stroes E, Moulin P, Parhofer KG, et al. Diagnostic algorithm for familial chylomicronemia syndrome. Atheroscler Suppl. 2017;23:1-7.
- Gaudet D, Brisson D, Tremblay K, et al. Targeting APOC3 in the Familial Chylomicronemia Syndrome. N Engl J Med. 2014;371(23):2200-2206.
- World Health Organization (WHO). Essential Medicines and Health Products – Guidelines for management of severe hypertriglyceridemia. WHO, 2023.
- British National Formulary (BNF). Treatment of Hypertriglyceridemia – Lipid-regulating drugs. NICE Evidence Services, 2025.
- Burnett JR, Hooper AJ, Hegele RA. Familial Lipoprotein Lipase Deficiency. In: Adam MP, et al., editors. GeneReviews. University of Washington, Seattle; 2017 (updated 2023).
About This Article
This article was written and reviewed by the iMedic Medical Editorial Team, comprising licensed physicians and pharmacology specialists with expertise in rare metabolic diseases and lipid disorders.
iMedic Medical Editorial Team – specialists in pharmacology and internal medicine with documented clinical and research experience in lipid metabolism disorders.
iMedic Medical Review Board – independent panel of medical experts who verify all content against current regulatory documentation and peer-reviewed evidence.
Level 1A evidence based on randomized controlled trials and EMA-approved product information. All claims verified against the Summary of Product Characteristics (SmPC).
No pharmaceutical company funding or advertising. All content is independently produced without commercial influence or conflicts of interest.