STRENSIQ 40 mg/mL injectable solution

Introduction

Package Leaflet: Information for the User

Strensiq 40 mg/ml solution for injection

(12 mg/0.3 ml, 18 mg/0.45 ml, 28 mg/0.7 ml, 40 mg/1 ml)

asfotase alfa

This medicine is subject to additional monitoring, which will allow for quick identification of new safety information. You can help by reporting any side effects you may get. The last section of this leaflet includes 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 Strensiq and what is it used for
2. What you need to know before you use Strensiq
3. How to use Strensiq
4. Possible side effects
5. Storing Strensiq
6. Contents of the pack and other information

1. What is Strensiq and what is it used for

What is Strensiq

Strensiq is a medicine used to treat a rare inherited disease called hypophosphatasia that started in childhood. It contains the active substance asfotase alfa.

What is hypophosphatasia

Patients with hypophosphatasia have low levels of an enzyme called alkaline phosphatase, which is important for various body functions, including the proper strengthening of bones and teeth. Patients have bone growth and strength problems that can lead to bone fractures, bone pain, and difficulty walking, as well as breathing difficulties and a risk of seizures (epileptic fits).

What is Strensiq used for

The active substance in Strensiq can replace the missing enzyme (alkaline phosphatase) in hypophosphatasia. It is used as prolonged enzyme replacement therapy to treat the symptoms.

What benefits has Strensiq shown in clinical studies

Strensiq has shown benefits in bone mineralization and patient growth.

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2. What you need to know before you use Strensiq

Do not use Strensiq

If you are severely allergic to asfotase alfa (see section "Warnings and precautions" below) or to any of the other ingredients of this medicine (listed in section 6).

Warnings and precautions

Consult your doctor before using Strensiq.

• Patient treated with asfotase alfa have presented allergic reactions, including potentially life-threatening allergic reactions similar to anaphylaxis that required medical treatment. Patients who presented symptoms similar to anaphylaxis had difficulty breathing, feeling of suffocation, nausea, swelling around the eyes, and dizziness. Reactions occurred within minutes of asfotase alfa administration and can occur in patients who have been taking asfotase alfa for more than a year. If you experience any of these symptoms, stop Strensiq and seek medical attention immediately.

If you experience an anaphylactic reaction or an episode with similar symptoms, your doctor will discuss the measures to be taken next and the possibility of resuming Strensiq under medical supervision. Always follow the instructions provided by your doctor.

• During treatment, blood proteins against Strensiq, also called antibodies against the medicine, may develop. Inform your doctor if you experience reduced efficacy of Strensiq.
• Fat lumps or reduced fat tissue on the skin surface (localized lipodystrophy) at the injection sites have been reported after several months of treatment with Strensiq. Read section 3 carefully for injection recommendations. It is essential to change the injection site between the following body areas to reduce the risk of lipodystrophy: abdominal area, thigh, or deltoid.
• In studies, some eye-related side effects (e.g., calcium accumulation in the eye [conjunctival or corneal calcification]) have been reported in patients using Strensiq and those who did not, probably associated with hypophosphatasia. Inform your doctor if you experience vision problems.
• Premature fusion of the skull bones (craneosinostosis) has been reported in children under 5 years of age in clinical studies of infants with hypophosphatasia, with and without the use of Strensiq. Inform your doctor if you notice any change in the shape of your child's head.
• If you receive treatment with Strensiq, you may experience an injection site reaction (pain, nodule, skin rash, discoloration) during the injection of the medicine or during the hours following the injection. If you experience any severe injection site reaction, inform your doctor immediately.
• In studies, an increase in parathyroid hormone concentration and low calcium levels have been reported. Therefore, your doctor may ask you to take oral calcium and vitamin D supplements if necessary.
• Weight gain may occur during your treatment with Strensiq. Your doctor will provide dietary advice as needed.

Other medicines and Strensiq

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

If you need to have blood tests, inform your doctor that you are receiving treatment with Strensiq. Strensiq may affect the results of some blood tests. Therefore, if you are treated with Strensiq, you may need to have other types of tests.

Pregnancy

Strensiq must not be used during pregnancy. Effective contraceptive methods should be considered during treatment in women of childbearing age.

Breast-feeding

It is not known whether Strensiq can be excreted in breast milk. Consult your doctor if you are breast-feeding or plan to breast-feed. Your doctor will then help you decide whether to stop breast-feeding or stop treatment with Strensiq, taking into account the benefit of breast-feeding for the child and the benefit of treatment with Strensiq for the mother.

If you are pregnant or breast-feeding, think you may be pregnant, or plan to become pregnant, consult your doctor or pharmacist before using this medicine.

Driving and using machines

This medicine is not expected to affect your ability to drive or use machines.

Important information about some of the ingredients of Strensiq

This medicine contains less than 1 mmol of sodium (23 mg) per vial, which means it can be considered essentially sodium-free.

3. How to use Strensiq

Follow the administration instructions for the medication contained in this prospectus or as indicated by your doctor, pharmacist, or nurse. In case of doubt, ask your doctor, pharmacist, or nurse.

A doctor with experience in treating patients with metabolic or bone diseases will explain how to use Strensiq. After the doctor or specialized nurse has trained you, you can administer Strensiq at home.

Dose

• The dose you receive depends on your body weight.
• Your doctor will calculate the correct dose, which consists of a total of 6 mg of asfotase alfa per kg of body weight per week, administered as an injection of 1 mg/kg of asfotase alfa 6 times a week or as an injection of 2 mg/kg of asfotase alfa 3 times a week, depending on your doctor's recommendation. Each dose will be administered by subcutaneous injection (under the skin) (see the included dosage table for detailed information on the volume to be injected and the type of vials to use based on your weight).
• Your doctor should periodically adjust the dose as your weight changes.
• The maximum volume per injection should not exceed 1 ml. If more than 1 ml needs to be administered, you should perform several injections in a row, one after the other.

In case of injection 3 times a week In case of injection 6 times a week

Injection recommendations

• You may experience a reaction at the injection site. Read section 4 carefully before using this medication to know the adverse effects that may occur.
• When injecting regularly, you should change the injection site between different areas of the body to help reduce possible pain and irritation.
• Areas with a greater amount of fat under the skin (thighs, arms [deltoids], abdomen, and buttocks) are the most suitable areas for injection. Consult your doctor or nurse to determine the best places in your case.

Before injecting Strensiq, read the following instructions carefully

• Each vial is for single use and should only be punctured once. The Strensiq liquid should have a transparent, slightly opalescent, or opalescent appearance, colorless to slightly yellow, and may contain small translucent or white particles. Do not use it if the liquid is discolored or contains lumps or large particles; obtain a new vial. The disposal of unused medication and all materials that have come into contact with it will be carried out in accordance with local regulations.
• If you are going to inject the medication yourself, your doctor, pharmacist, or nurse will teach you how to prepare and inject the medication. Do not inject this medication yourself unless you have been trained and have understood the procedure.

How to inject Strensiq:

Step 1: Preparation of the Strensiq dose

1. Wash your hands thoroughly with water and soap.
2. Remove the Strensiq vial(s) from the refrigerator 15 to 30 minutes before administering the injection to allow the liquid to reach room temperature. Do not heat Strensiq in any other way (e.g., do not heat it in the microwave or in hot water). After removing the vial(s) from the refrigerator, Strensiq must be used within a maximum of 3 hours.
3. Remove the protective cap from the Strensiq vial(s). Remove the plastic protector from the syringe you will use.
4. Always use a new syringe with a plastic protector.
5. Place a larger gauge needle (e.g., 25G) on the empty syringe, keeping the protective cap, press and turn the needle onto the syringe in a clockwise direction until it is secure.
6. Remove the plastic protector that covers the needle of the syringe. Be careful not to hurt yourself with the needle.
7. Pull the plunger back to draw in the same amount of air into the syringe as your dose.

Step 2: Extraction of the Strensiq solution from the vial

1. After removing the air bubbles, check the dose of medication in the syringe to ensure you have drawn the correct amount. You may need to use multiple vials to draw the total volume you need for the correct dose.

Step 3: Placement of the needle on the syringe

1. Remove the needle from the vial. Put the cap back on with one hand by placing it on a flat surface; slide the needle into the cap, lift it, and snap it firmly into place using one hand.
2. Remove the larger gauge needle by pressing and turning it counterclockwise. Place the needle with the protective cap in the sharps container.
3. Place a smaller gauge needle (e.g., 27 or 29G) on the filled syringe, keeping the protective cap, press and turn the needle onto the syringe in a clockwise direction until it is secure. Remove the protective cap from the needle.
4. Hold the syringe with the needle facing upwards and gently tap the syringe barrel with your fingers to remove any possible air bubbles.

Visually check that the volume contained in the syringe is correct.

The volume per injection should not exceed 1 ml. If you need a larger volume, you should administer several injections in different locations.

You are now ready to inject the correct dose.

Step 4: Injection of Strensiq

If you need to administer a second injection to complete the prescribed dose, take another Strensiq vial and repeat steps 1 to 4.

Step 5: Disposal of used materials

Place the syringes, vials, and needles in a sharps container. Your doctor, pharmacist, or nurse will tell you how to obtain one of these containers.

If you use more Strensiq than you should

If you think you have accidentally received a higher dose of Strensiq than prescribed, contact your doctor for advice.

If you miss a dose of Strensiq

Do not inject a double dose to make up for the missed dose, and contact your doctor for advice.

If you have any other questions about the use of this medication, ask your doctor, pharmacist, or nurse.

4. Possible side effects

Like all medications, this medication can cause side effects, although not everyone will experience them.

If you are not sure what the following side effects are, ask your doctor to explain them to you.

The most serious side effects observed in patients treated with asfotase alfa have been allergic reactions, including potentially life-threatening allergic reactions similar to anaphylaxis that required medical treatment. This side effect is common (it can affect up to 1 in 10 people). Patients who experienced these severe allergic reactions had difficulty breathing, a feeling of suffocation, nausea, swelling around the eyes, and dizziness. The reactions occurred a few minutes after using asfotase alfa and can occur in patients who have been using asfotase alfa for more than a year. If you experience any of these symptoms, stop using Strensiq and go to the doctor immediately.

Additionally, other allergic reactions (hypersensitivity) may occur frequently, which can manifest as redness (erythema), fever (pyrexia), skin rash, itching (pruritus), irritability, nausea, vomiting, pain, chills (shivering), numbness of the mouth (oral hypoesthesia), headache, flushing (hot flashes), rapid heart rate (tachycardia), and cough. If you experience any of these symptoms, stop using Strensiq and go to the doctor immediately.

Very common: may affect more than 1 in 10 people

Reactions at the injection site during or after injection of the medication (which can cause redness, discoloration, itching, pain, fat lumps or less fatty tissue on the skin surface, hypopigmentation of the skin, and/or swelling)

Fever (pyrexia)

Irritability

Redness of the skin (erythema)

Pain in the hands and feet (pain in the extremities)

Bruising (contusion)

Headache

Common: may affect up to 1 in 10 people

Stretched skin, skin discoloration

Nausea

Numbness of the mouth (oral hypoesthesia)

Muscle pain (myalgia)

Scarring

Increased tendency to bruising

Hot flashes

Skin infection at the injection site (cellulitis at the injection site)

Lower calcium levels in the blood (hypocalcemia)

Kidney stones (nephrolithiasis)

Reporting side effects

If you experience any side effects, consult your doctor, pharmacist, or nurse, even if they are possible side effects not listed in this prospectus. You can also report them directly through the Spanish Medication Monitoring System for Human Use: www.notificaRAM.es. By reporting side effects, you can contribute to providing more information on the safety of this medication.

5. Storage of Strensiq

Keep this medication out of the sight and reach of children.

Do not use this medication after the expiration date stated on the box after CAD and on the vial label after EXP. The expiration date is the last day of the month indicated.

Store in a refrigerator (between 2°C and 8°C).

Do not freeze.

Store in the original packaging to protect it from light.

After opening the vial, the medication must be used immediately (within 3 hours at room temperature between 23°C and 27°C).

Medications should not be disposed of through wastewater or household waste. Ask your pharmacist how to dispose of the packaging and any unused medication. This will help protect the environment.

6. Container Content and Additional Information

Strensiq Composition

The active ingredient is asfotase alfa. Each ml of solution contains 40 mg of asfotase alfa.

Each 0.3 ml vial of solution (40 mg/ml) contains 12 mg of asfotase alfa.

Each 0.45 ml vial of solution (40 mg/ml) contains 18 mg of asfotase alfa.

Each 0.7 ml vial of solution (40 mg/ml) contains 28 mg of asfotase alfa.

Each 1 ml vial of solution (40 mg/ml) contains 40 mg of asfotase alfa.

The other components are sodium chloride, sodium phosphate monobasic monohydrate, sodium phosphate dibasic heptahydrate, and water for injectable preparations.

Product Appearance and Container Content

Strensiq is presented as a clear, slightly opalescent or opalescent, colorless to slightly yellowish aqueous injectable solution, in vials containing 0.3 ml, 0.45 ml, 0.7 ml, and 1 ml of solution. It may contain some small translucent or white particles.

Container sizes of 1 or 12 vials.

Only some container sizes may be marketed.

Marketing Authorization Holder

Alexion Europe SAS

103-105 rue Anatole France

92300 Levallois-Perret

France

Manufacturer

Alexion Pharma International Operations Limited

College Business and Technology Park, Blanchardstown

Dublin 15

Ireland

Date of Last Revision of this Leaflet: June 2021

This medicinal product has been authorized under "exceptional circumstances". This means that due to the rarity of the disease, it has not been possible to obtain complete information on this medicinal product.

The European Medicines Agency will review any new information on this medicinal product that may become available annually, and this leaflet will be updated as necessary.

Other Sources of Information

Detailed information on this medicinal product is available on the European Medicines Agency website: http://www.ema.europa.eu. There are also links to other websites on rare diseases and orphan medicines.