2 mg / ml concentrate for solution for infusion
patisiran
What Onpattro is and what it is used for
The active substance in Onpattro is patisiran.
Onpattro is a drug that treats a hereditary disease called hereditary transthyretin amyloidosis (hATTR amyloidosis, Skellefte’s disease).
HATTR amyloidosis is caused by a problem with a protein in the body called ‘transthyretin’ (TTR).
- This protein is mainly made by the liver and carries around vitamin A and other substances in the body.
- In people with this disease, abnormally shaped TTR proteins can clump together and create deposits called “amyloid”.
- Amyloid can accumulate around nerves, the heart and other places in the body and prevent the organs from functioning properly. This causes the symptoms of the disease.
Onpattro works by reducing the amount of TTR protein that the liver produces.
- This means that there is less TTR protein in the blood that can form amyloid.
- This can help reduce the effects of this disease.
Onpattro is only used for adults.
What you need to know before using Onpattro
Do not use Onpattro
- if you have ever had a severe allergic reaction to patisiran or any of the other ingredients of this medicine (listed in section 6). If you are not sure, talk to your doctor or nurse before using Onpattro.
Warnings and cautions
Infusion-related reactions
Onpattro is given as a drip into a vein (called an “intravenous infusion”). Reactions to this infusion may occur during treatment with Onpattro. Before each infusion, you will be given medicines that help reduce the risk of infusion-related reactions (see “Medicines given during treatment with Onpattro” in section 3).
If you get any signs of an infusion-related reaction, tell your doctor or nurse immediately. These characters are listed at the beginning of section 4.
If you get an infusion-related reaction, your doctor or nurse may slow down or stop your infusion or you may need to take other medicines to keep your symptoms under control. When these reactions stop or get better, your doctor or nurse may decide to start the infusion again.
Vitamin A deficiency
Treatment with Onpattro lowers the amount of vitamin A in your blood. Your doctor will measure your vitamin A levels and if they are too low they should have returned to normal and any symptoms of vitamin A deficiency should have passed before starting treatment with Onpattro. Symptoms of vitamin A deficiency may include:
- Impaired night vision, dry eyes, poor eyesight, foggy or cloudy eyesight.
If you have vision problems or any other eye problems while using Onpattro, talk to your doctor. Your doctor may refer you to an ophthalmologist for examination if needed.
Your doctor will ask you to take a vitamin A supplement daily during treatment with Onpattro.
Both too high and too low levels of vitamin A can impair the development of unborn children. Therefore, women of childbearing potential should not become pregnant after starting treatment with Onpattro and must use an effective method of contraception (see section “Pregnancy, breast-feeding and contraception” below).
Tell your doctor if you are planning to become pregnant. Your doctor may tell you to stop taking Onpattro. Your doctor will check that your vitamin A levels have returned to normal before trying to conceive.
Tell your doctor if you have become unplanned pregnant. Your doctor may tell you to stop taking Onpattro. During the first three months of pregnancy, your doctor may tell you to stop taking vitamin A supplements. During the last six months of pregnancy, you should resume the vitamin A supplement if your vitamin A levels have not yet returned to normal. due to an increased risk of vitamin A deficiency during the last three months of pregnancy.
Children and young people
Onpattro is not recommended for children and adolescents under 18 years of age.
Other medicines and Onpattro
Tell your doctor or nurse if you are taking, have recently taken, or might take any other medicines. You must tell your doctor or nurse if you are taking any of the following medicines, as your doctor may need to change the dose:
- Bupropion, a drug used to treat depression or to help quit smoking
- Efavirenz, a medicine used to treat HIV infection and AIDS .
Pregnancy, breastfeeding and fertility
If you are pregnant or breast-feeding, think you may be pregnant, or are planning to have a baby, ask your doctor for advice before using this medicine.
Women of childbearing potential
Onpattro will lower the level of vitamin A in the blood. Vitamin A is important for the normal development of your unborn baby. If you are a fertile woman, you should use an effective method of contraception during treatment with Onpattro. Talk to your doctor or nurse about appropriate contraceptive methods. Pregnancy should be ruled out before starting treatment with Onpattro.
Pregnancy
You should not use Onpattro if you are pregnant unless your doctor advises you to do so. If you are fertile and intend to use Onpattro, use an effective method of contraception.
Breast-feeding
The ingredients in Onpattro can be transferred to breast milk. Talk to your doctor about stopping breast-feeding or stopping treatment with Onpattro.
Driving and using machines
Onpattro is considered to have no or negligible effect on the ability to drive and use machines. Your doctor will tell you if your condition allows you to drive vehicles and use machines safely.
Onpattro contains excipients
This medicine contains 3.99 milligrams (mg) sodium (the main ingredient in table salt/table salt) per milliliter (ml). This corresponds to 0.2% of the maximum recommended daily intake of sodium for adults.
How to use Onpattro
How much Onpattro is given
- Your doctor will calculate how much Onpattro should be given to you – this depends on your body weight.
- The usual dose of Onpattro is 300 micrograms per kilogram (kg) body weight and is given once every three weeks.
How Onpattro is given
- Onpattro will be given to you by a doctor or nurse.
- It is given as a drip into a vein (“intravenous infusion”), usually for about 80 minutes.
If you have no problems with your infusions at the clinic, your doctor can discuss with you that healthcare professionals give you your infusions at home.
Medicines are given during treatment with Onpattro
Before each infusion of Onpattro, you will be given medicines that will help reduce the risk of infusion-related reactions. These include antihistamines, a corticosteroid (an anti-inflammatory drug ), and a painkiller.
How long to use Onpattro
Your doctor will tell you how long you need to use Onpattro. Do not stop taking Onpattro unless your doctor tells you to.
If you take more Onpattro than you should
This medicine is given to you by a doctor or nurse. In the unlikely event that you are given too much (an overdose), your doctor or nurse will monitor you for side effects.
If you miss your dose of Onpattro
If you miss an appointment to receive Onpattro, ask your doctor or nurse when to book your next treatment.
If you have any further questions on the use of this product, ask your doctor or nurse.
Possible side effects
Like all medicines, this medicine can cause side effects, although not everybody gets them.
Infusion-related reactions
Infusion-related reactions are very common (may affect more than 1 in 10 people).
Tell your doctor or nurse immediately if you get any of the following signs of an infusion-related reaction during treatment. Infusion one may need to be slowed down or discontinued and you may need to take other medicines to manage the reaction.
- Stomach pain
- Nausea
- Pain or pain in the body, including the back, neck or joints
- Headache
- Fatigue (exhaustion)
- Overindulge
- Dizziness
- Cough, shortness of breath or other breathing problems
- Redness of the face or body (hot flashes), feeling of warmth in the skin or rash
- Chest discomfort or chest pain
- Fast heartbeat
- Low or high blood pressure ; Some patients have fainted during infusion one due to low blood pressure
- Pain, redness , burning or swelling at or near the infusion site
- Swelling of the face
Other side effects are
Tell your doctor or nurse if you notice any of the following side effects:
Very common: may affect more than 1 in 10 people
- Swelling of the arms or legs (peripheral edema )
Common: may affect up to 1 in 10 people
- Joint pain ( arthralgia )
- Muscle spasms
- Indigestion ( dyspepsia )
- Shortness of breath ( dyspnoea )
- Redness of the skin ( erythema )
- Feeling dizzy or fainting (dizziness)
- Clogged or runny nose ( rhinitis )
- Irritation or infection of the respiratory tract (sinusitis, bronchitis )
Uncommon: may occur in up to 1 in 100 infusions
- Leakage of the drug into the surrounding tissue at the infusion site which may cause swelling and redness
Tell your doctor or nurse if you notice any of the side effects listed above.
How to store Onpattro
Keep this medicine out of the sight and reach of children.
Do not use this medicine after the expiry date which is stated on the carton after “EXP”. The expiration date is the last day of the specified month.
Store in a refrigerator (2 ° C – 8 ° C). Do not freeze.
If refrigerator storage is not available, Onpattro can be stored at room temperature (maximum 25 ° C) for up to 14 days.
Medicines should not be disposed of via wastewater or household waste. Ask your pharmacist how to dispose of medicines no longer required. These measures will help to protect the environment.
Contents of the package and other information
Content declaration
- The active substance is patisiran.
- Each ml contains patisiran sodium equivalent to 2 mg patisiran.
- Each 5 ml vial contains patisiran sodium equivalent to 10 mg patisiran.
- The other ingredients are Dlin ‑ MC3 ‑ DMA ((6Z, 9Z, 28Z, 31Z) ‑heptatria account ‑ 6,9,28,31 ‑ tetraene ‑ 19 ‑ yl ‑ 4‑ (dimethylamino) ‑butanoate), PEG 2000 ‑C ‑ DMG (α- (3 ‘- {[1,2-di (myristyloxy) propanoxy] carbonylamino} propyl) methoxy, polyoxyethylene), DSPC (1,2-distearoyl- sn -glycero-3-phosphocholine), cholesterol , disodium hydrogen phosphate heptahydrate, anhydrous potassium dihydrogen phosphate, sodium chloride and water for injections (see “Onpattro contains sodium” in section 2).
What the medicine looks like and contents of the pack
- Onpattro is a white to off-white, opalescent, homogeneous concentrate for solution for infusion .
- Onpattro is delivered in cartons, each containing a vial .
Marketing Authorization Holder and Manufacturer
Alnylam Netherlands BV
Antonio Vivaldi Street 150
1083 HP Amsterdam
Netherlands