AMVUTTRA 25 mg Injectable Solution in Pre-filled Syringe

Introduction

Package Leaflet: Information for the Patient

Amvuttra 25mg solution for injection in pre-filled syringe

vutrisiran

This medicinal product is subject to additional monitoring, which will allow for the quick identification of new safety information. You can help by reporting any side effects you may get. The last section of the leaflet contains information on how to report side effects.

Read all of this leaflet carefully before you start using this medicine, because it contains important information for you.

• Keep this leaflet, you may need to read it again.
• If you have any further questions, ask your doctor, pharmacist, or nurse.
• This medicine has been prescribed for you only. Do not pass it on to others. It may harm them, even if their signs of illness are the same as yours.
• If you get any side effects, talk to your doctor, pharmacist, or nurse. This includes any possible side effects not listed in this leaflet. See section 4.

Contents of the pack

1. What is Amvuttra and what is it used for
2. What you need to know before you use Amvuttra
3. How to use Amvuttra
4. Possible side effects
5. Storage of Amvuttra
6. Contents of the pack and further information

1. What is Amvuttra and what is it used for

The active substance of Amvuttra is vutrisiran.

What Amvuttra is used for

Amvuttra is used to treat a disease called “ATTR amyloidosis”. This disease can be inherited and can also be caused by aging. ATTR amyloidosis is caused by problems with a protein in the body called “transthyretin” (TTR). This protein is mainly formed in the liver and carries vitamin A and other substances through the body.

In people with this disease, small fibers of the TTR protein cluster together to form deposits called “amyloid”. Amyloid can build up around nerves, the heart, and other parts of the body, or inside them, and prevent them from working normally. This causes the symptoms of the disease.

How Amvuttra works

Amvuttra works by reducing the amount of TTR protein produced by the liver, which means there is less TTR protein in the blood that can form amyloid. This can help reduce the effects of this disease.

Amvuttra is only used in adults.

2. What you need to know before you use Amvuttra

Do not use Amvuttra

• if you have ever had a severe allergic reaction to vutrisiran or any of the other ingredients of this medicine (listed in section 6).

If you are not sure, consult your doctor, pharmacist, or nurse before using this medicine.

Warnings and precautions

Reduced levels of vitamin A in the blood and vitamin A supplements

Amvuttra reduces the amount of vitamin A in the blood.

Your doctor will ask you to take a daily vitamin A supplement. Take the recommended dose of vitamin A as advised by your doctor.

Signs of low vitamin A levels may include: vision problems, especially at night, dry eyes, or blurred vision.

• If you notice any change in your vision or any other eye problems while using Amvuttra, consult your doctor. Your doctor may refer you to an eye specialist for a check-up.

Too high or too low levels of vitamin A can harm the development of the fetus. Therefore, pregnancy should be excluded in women of childbearing age before starting treatment with Amvuttra, and they should use effective contraceptive methods (see section “Pregnancy, breastfeeding, and contraception” below).

• Vitamin A levels may remain low for more than 12 months after the last dose of Amvuttra.
• Inform your doctor if you plan to become pregnant. Your doctor will advise you to stop using Amvuttra and the vitamin A supplement. Your doctor will also ensure that your vitamin A levels have returned to normal before you try to become pregnant.
• Inform your doctor in case of an unplanned pregnancy. Your doctor will advise you to stop using Amvuttra. During the first 3 months of pregnancy, your doctor may advise you to stop taking the vitamin A supplement. During the last 6 months of pregnancy, your doctor may advise you to resume the vitamin A supplement if your vitamin A levels in the blood have not yet returned to normal, due to a higher risk of deficiency during the last 3 months of pregnancy.

Children and adolescents

Amvuttra is not recommended in children and adolescents under 18 years of age.

Other medicines and Amvuttra

Tell your doctor, pharmacist, or nurse if you are using, have recently used, or might use any other medicines.

Pregnancy, breastfeeding, and contraception

If you are pregnant or breastfeeding, think you may be pregnant, or are planning to have a baby, consult your doctor or pharmacist before using this medicine.

Pregnancy

Do not use Amvuttra if you are pregnant.

Women of childbearing age

Amvuttra will reduce the level of vitamin A in your blood, which is important for the normal development of the fetus (see “Warnings and precautions” above).

• If you are a woman who can become pregnant, you must use an effective contraceptive method during treatment with Amvuttra.
• Consult your doctor or nurse about suitable contraceptive methods.
• Pregnancy should be excluded before starting treatment with Amvuttra.
• Inform your doctor if you plan to become pregnant or in case of an unplanned pregnancy. Your doctor will advise you to stop taking Amvuttra.

Breastfeeding

It is not known whether vutrisiran can pass into breast milk. Your doctor will weigh the potential benefits of treatment for you against the risks of breastfeeding for the baby.

Driving and using machines

Amvuttra is not expected to affect your ability to drive or use machines. Your doctor will tell you if your condition allows you to drive vehicles and use machines safely.

Amvuttra contains sodium

This medicine contains less than 1 mmol of sodium (23 mg) per ml; this is, essentially “sodium-free”.

3. How to use Amvuttra

Amvuttra can be self-administered or administered by a caregiver or healthcare professional.

Your doctor or healthcare professional will show you and/or your caregiver how to prepare and inject a dose of Amvuttra before you do it yourself.

To know how to use Amvuttra, read the “Instructions for use” at the end of this leaflet.

How much Amvuttra to use

The recommended dose is 25 mg once every 3 months.

Where to administer the injection

Amvuttra is administered by injection under the skin (“subcutaneous injection”) in the stomach area (abdomen), in the upper arm (if administered by another person), or in the thigh.

How long to use Amvuttra

Your doctor will tell you how long you need to use Amvuttra. Do not stop treatment with Amvuttra unless your doctor tells you to.

If you use more Amvuttra than you should

In the unlikely event that you use too much (an overdose), contact your doctor or pharmacist, even if you do not have any symptoms. Your doctor will check for any side effects.

If you forget to use Amvuttra

If you miss a dose, administer Amvuttra as soon as possible. From then on, resume administration every 3 months, counting from the last administered dose.

If you have any further questions on the use of this medicine, ask your doctor, pharmacist, or nurse.

4. Possible side effects

Like all medicines, this medicine can cause side effects, although not everybody gets them.

Tell your doctor, pharmacist, or nurse if you notice any of the following side effects:

Common:may affect up to 1 in 10 people

• Redness, pain, itching, bruising, or warmth at the injection site
• Blood tests showing increased levels of liver enzymes called alkaline phosphatase and alanine transaminase

Reporting of side effects

If you experience any side effects, talk to your doctor or nurse, even if it is possible side effects not listed in this leaflet. You can also report side effects directly through the national reporting system listed in Appendix V. By reporting side effects, you can help provide more information on the safety of this medicine.

5. Storage of Amvuttra

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 label, on the carton, and on the blister after EXP. The expiry date refers to the last day of the month stated.

Do not store above 30°C.

Do not freeze.

Medicines should not be disposed of via wastewater or household waste. Ask your pharmacist how to dispose of medicines no longer required. This will help protect the environment.

6. Container Contents and Additional Information

Composition of Amvuttra

• The active ingredient is vutrisiran.

Each prefilled syringe contains vutrisiran sodium equivalent to 25 mg of vutrisiran in 0.5 ml of solution.

• The other components are: sodium dihydrogen phosphate dihydrate, disodium phosphate dihydrate, sodium chloride, and water for injectable preparations. Sodium hydroxide and phosphoric acid may be used to adjust the pH (see "Amvuttra contains sodium" in section 2).

Appearance and Container Contents of the Product

This medicinal product is a clear, colorless to yellowish injectable solution. Each container contains a single-dose prefilled syringe.

Marketing Authorization Holder and Manufacturer

Alnylam Netherlands B.V.

Antonio Vivaldistraat 150

1083 HP Amsterdam

Netherlands

You can request more information about this medicinal product by contacting the local representative of the marketing authorization holder:

Date of Last Revision of this Leaflet:

Other Sources of Information

Detailed information on this medicinal product is available on the European Medicines Agency website: https://www.ema.europa.eu/.

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INSTRUCTIONS FOR USE

Amvuttra 25 mg solution for injection in a prefilled syringe

vutrisiran

Single-dose prefilled syringe with needle shield

Read these instructions before using this prefilled syringe.

Information about the Prefilled Syringe

The prefilled syringe (referred to as "syringe") is disposable and for single use only.

Route and Method of Administration

Each carton contains a single-dose Amvuttra syringe. Each Amvuttra syringe contains 25 mg of vutrisiran for injection under the skin (subcutaneous injection), once every 3 months.

Your doctor or healthcare professional will show you and/or your caregiver how to prepare and inject a dose of Amvuttra before you do it yourself. Contact your healthcare professional or doctor for more information and assistance if needed.

Keep these instructions until you have used the syringe.

Storage of Amvuttra

Do notstore above 30°C.

Do notfreeze.

Keep this medicinal product out of the sight and reach of children.

Important Warnings

Do notuse the medicinal product if the carton is damaged or shows signs of tampering.

Do notuse the syringe if it has been dropped onto a hard surface.

Do nottouch the plunger rod until you are ready to inject.

Do notremove the needle shield cap until just before injection.

Do notreplace the needle shield cap on the syringe at any time.