Onpattro

Onpattro is a prescription medication treatment for polyneuropathy in adults living with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis). This condition affects several parts of the body, including the nerves, heart, and digestive system, which means individuals may experience a range of symptoms that may not seem related to one another.

Onpattro decreases the amount of TTR protein made in the liver. This helps to decrease the amount of amyloid deposits and may result in fewer symptoms.

Onpattro is given by intravenous (IV) infusion once every 3 weeks by a healthcare professional in a doctor’s office or an infusion center.

The most common side effects of Onpattro are respiratory infections, such as colds, sinus infections, and nasal congestion, and infusion-related reactions.

Onpattro is a prescription medication used to treat polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR). Hereditary transthyretin-mediated amyloidosis is a rare genetic disease and can be life-threatening. It causes unwanted proteins to build up in nerves, the heart, and other organs. Onpattro is used in adults only.

Serious side effects have been reported with Onpattro. See "Onpattro Precautions" section.

Common side effects of Onpattro include:

• respiratory infections such as colds, sinus infections, and nasal congestion
• infusion-related reactions such as flushing of skin, back pain, nausea, abdominal pain, difficulty breathing, headache
• treatment with Onpattro lowers the amount of vitamin A in your blood. Your doctor will tell you to take a vitamin A supplement every day.

This is not a complete list of Onpattro side effects. Tell your doctor if you have any side effects that bother you or that do not go away. Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.

No drug interactions have been determined by the manufacturer. However, you should tell your doctor about all the medicines you take including prescription and non-prescription medicines, vitamins, and herbal supplements. Not all drug interactions are known or reported and new drug interactions are continually being reported.

Serious side effects have been reported with Onpattro including:

Infusion-related reactions: Onpattro is given as a drip into a vein (called an “intravenous infusion”). Reactions to this infusion may happen during treatment with Onpattro. Before each infusion you will be given medicines that help to lower the chance of infusion-related reactions.

Tell your doctor or nurse right away if you experience any of the following signs of an infusion-related reaction during treatment:

• Reddening of the face or body(flushing), skin warm
• Body aches or pain, including pain in the back, neck, or joints
• Feeling sick (nausea)
• Stomach pain
• Feeling short of breath, cough, or other breathing problems
• Headache
• Chest discomfort or chest pain
• Rash
• Chills
• Dizziness
• Feeling tired (fatigue)
• Rapid heart rate
• Facial swelling

If you have an infusion-related reaction, your doctor or nurse may slow down or stop your infusion, and you may need to take other medicines. When these reactions stop, or get better, your doctor or nurse may decide to start the infusion again.

Low Vitamin A levels
Treatment with Onpattro lowers the amount of vitamin A in your blood. Your doctor will tell you to take a vitamin A supplement every day. You should not take more than the amount of vitamin A recommended by your doctor.

Low vitamin A levels can affect vision. If you have problems with your vision (e.g., night blindness) while taking Onpattro, talk to your doctor. Your doctor may refer you to an eye specialist.

Do not take this medication if you are allergic to any of its ingredients.

Medications can interact with certain foods. In some cases, this may be harmful and your doctor may advise you to avoid certain foods. In the case of Onpattro, there are no foods to watch out for in your diet.

Before using Onpattro, tell your healthcare provider about all your medical conditions, including if you:

• are pregnant or plan to become pregnant. It is not known if Onpattro will harm your unborn baby.
• are breastfeeding or plan to breastfeed. It is not known if Onpattro passes into your breast milk. 

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Know the medicines you take. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine.

Tell your doctor if you are pregnant or plan to become pregnant.

There is no data on Onpattro use in pregnancy. Onpattro reduces vitamin A, which an unborn baby needs to grow. Onpattro should be used during pregnancy only if the benefits are more than the risks to the unborn baby. In animal studies, pregnant animals were given this medicine, and the babies did not grow and develop properly.

Tell your doctor if you are breastfeeding or plan to breastfeed.

It is not known if Onpattro crosses into human milk. Because many medications can cross into human milk and because of the possibility for serious adverse reactions in nursing infants with use of this medication, a choice should be made whether to stop nursing or stop the use of this medication. Your doctor and you will consider the benefits of breastfeeding and the need for Onpattro and its adverse effects to the baby, if any.

Onpattro is given by intravenous (IV) infusion once every 3 weeks by a healthcare professional in adoctor’s office or an infusion center. Some patients may be eligible for infusions at their home, if coveredby their insurance plan. Prior to the IV infusion, individuals will receive a combination of medications commonly used in order to reduce the risk of infusion-related reactions.

Take this medication exactly as prescribed by your doctor. Follow the directions on your prescription label carefully.

The dose your doctor recommends may be based on the following:

• Actual body weight
  • Patients who weigh less than 100 kg, the recommended dose is 0.3 mg/kg once every 3 weeks
  • Patients who weigh 100 kg or more, the recommended dose is 30 mg once every 3 weeks

If you take too much Onpattro, call your healthcare provider or local Poison Control Center, or seek emergency medical attention right away.

• Store Onpattro vials refrigerated at 36-46 °F (2-8 °C). Do not freeze. Throw away vial if it has been frozen.
• Keep this and all medicines out of the reach of children.

This page was written by Lindsey Loera, PharmD Candidate 2020 | UT Austin College of Pharmacy