Prospect: information for the patient

Loargys 5 mg/ml injectable solution and for perfusion

pegzilarginase

This medicine is subject to additional monitoring, which will expedite the detection of new information about its safety. You may contribute by reporting any adverse effects you may experience. The final part of section 4 includes information on how to report these adverse effects.

Read this prospect carefully before starting to take this medicine, as it contains important information for you.

• Keep this prospect, as you may need to refer to it again.
• If you have any doubts, consult your doctor or nurse.
• If you experience adverse effects, consult your doctor or nurse,even if they are adverse effects not listed in this prospect. See section4.

1.What Loargys is and for what it is used

2.What you need to know before starting to take Loargys

3.How Loargys is administered

4.Possible adverse effects

5.Storage of Loargys

6.Contents of the package and additional information

7.Usage instructions

Loargys contains the active ingredient pegzilarginase, which is a modified human enzyme produced by recombinant DNA technology. The medication is used to treat arginase 1 deficiency (ARG1-D), also known as hyperargininemia, in adults, adolescents, and children aged 2 years and older.

Patients with ARG1-D have low levels of an enzyme called arginase. This enzyme helps the body control arginine levels, an amino acid the body needs to produce proteins. If arginine is not controlled, it can accumulate in the body and cause symptoms, such as muscle control problems.

Loargys is used in combination with other forms of treatment. These may include:

• a low-protein diet
• food supplements with essential amino acids
• medications to treat other disease symptoms, such as medications that reduce ammonia levels in the body.

How Loargys works

The pegzilarginase, the active ingredient in Loargys, acts similarly to the natural arginase enzyme, which is lacking or not functioning correctly in patients with ARG1-D. This reduces blood arginine levels, thereby reducing disease symptoms.

You should not receive Loargys

• if you have had a severe allergic reaction to pegzilarginase or any of the other components of this medication (listed in section6).

Warnings and precautions

Loargys may cause allergic reactions. This is more likely to occur after the first two doses.

Stop the injection immediately and contact your doctor or emergency services if you experience any of the following severe allergic reaction symptoms: hives, generalized itching, chest tightness, difficulty breathing, or low blood pressure. Your doctor may decide that you need additional medical treatment to prevent or treat an allergic reaction.

During your treatment, your doctor will perform blood tests to check what dose of Loargys is suitable for you.

Children and adolescents

The medication should not be used in children under 2 years of age, as it is not known if Loargys is safe and effective in this age group.

Use of Loargys with other medications

Inform your doctor if you are taking, have taken recently, or may need to take any other medication.

Pregnancy, breastfeeding, and fertility

If you are pregnant or breastfeeding, think you may be pregnant, or plan to become pregnant, consult your doctor before using this medication. Loargys is not recommended for use during pregnancy.

It is not known if the medication passes into breast milk. If you are breastfeeding, consult your doctor before taking this medication. Your doctor will help you decide whether to stop breastfeeding or interrupt treatment.

Driving and operating machinery

The influence of Loargys on the ability to drive and operate machinery is negligible or insignificant.

Loargys contains sodium and potassium

This medication contains less than 1mmol of sodium (23mg) per dose; essentially 'sodium-free'. This medication contains potassium, less than 1mmol (39mg) per dose, essentially 'potassium-free'.

Loargys will be administered by a healthcare professional. Your doctor will decide the amount of Loargys that you will receive.

The recommended initial dose of Loargys is 0.1 mg per kilogram of body weight, administered once a week. Your doctor may increase or decrease the dose to maintain arginine levels in the blood under control. Your doctor will prescribe periodic blood tests to check your arginine levels in the blood and adjust your dose as needed.

Loargys is administered through infusion (drip) directly into a vein or through subcutaneous injection, as your doctor considers appropriate.

Your doctor may decide that you can receive Loargys at home, in the form of a subcutaneous injection. After receiving training from your doctor or nurse, you may be able to administer Loargys yourself (see instructions in section7).

Always use this medication exactly as described in the present leaflet or as indicated by your doctor, pharmacist, or nurse. If in doubt, consult your doctor, pharmacist, or nurse.

If you receive more Loargys than you should

Your doctor will ensure that you receive the correct amount of Loargys. If you have received too much Loargys, your arginine level in the blood may become too low. Symptoms may include nausea, vomiting, diarrhea, and fatigue. If you or your doctor suspect that you have received more Loargys than you should, you will be closely monitored and receive the necessary treatment.

If you forget to use «Loargys»

If you have forgotten a dose of Loargys, contact your doctor to schedule the next dose as soon as possible. Do not receive a double dose to compensate for the missed doses, and at least 4 days should pass between doses.

If you interrupt treatment with Loargys

Your doctor will decide if you should stop using Loargys. If you stop treatment with Loargys, it is likely that your arginine level in the blood will increase again.

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

Like all medications, this medication may produce adverse effects, although not all people will experience them.

Very Frequent(may affect more than 1 in 10 people)

• Reaction at the injection site. Symptoms may includepain,swelling,irritation,redness, and skin rash around the injection site.
• • Allergic Reaction (Hypersensitivity) Symptoms may include facial swelling, skin rash, and sudden skin redness (flushing).

Reporting Adverse Effects

If you experience any type of adverse effect, consult your doctor or nurse,even if it is an adverse effect not listed in this prospectus. You can also report them directly through thenational notification system included intheAppendixV.By reporting adverse effects, you can contribute to providing more information about the safety of this medication.

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

Do not use this medication after the expiration date listed on the label. The expiration date is the last day of the month indicated.

Store in refrigerator (between 2°C and 8°C). Do not freeze. Store in original packaging to protect it from light.

Once removed from the refrigerator, Loargys can be stored for 2 hours at ambient temperature not exceeding 25°C.

Medicines should not be disposed of through drains or in the trash. This will help protect the environment.

Composition of Loargys

• The active ingredient is pegzilarginase.
• Each vial of 0.4ml contains 2mg of pegzilarginase.
• Each vial of 1 ml contains 5 mg of pegzilarginase.

• Other ingredients are sodium chloride, potassium dihydrogen phosphate, dipotassium phosphate, glycerol, hydrochloric acid, sodium hydroxide, and water for injection.
  Loargys contains sodium and potassium (see section 2).

Appearance of the product and contents of the package

Loargys is a colorless to slightly yellow or slightly pink, clear to slightly opalescent liquid in a transparent glass vial.

Each package contains 1 vial with 0.4 ml or 1 ml of injection/infusion solution.

Only some package sizes may be marketed.

Marketing authorization holder and responsible person for manufacturing

Immedica Pharma AB

113 63 Stockholm

Sweden

Responsible person for manufacturing

Unimedic AB

Storjordenvägen 2

864 31 Matfors

Sweden

Last review date of this leaflet.

This medicine has been authorized under «exceptional circumstances». This authorization modality means that due to the rarity of this disease, it has not been possible to obtain complete information about this medicine.

The European Medicines Agency will review any new information available for this medicine annually and this leaflet will be updated as necessary.

Other sources of information

Further information on this medicine is available on the European Medicines Agency website: . There are also links to other websites on rare diseases and orphan medicines.

This leaflet is available in all languages of the European Union/European Economic Area on the European Medicines Agency website.

You can also find this leaflet and approved training material on this medicine on the websitehttp://www.loargyspatient.eu

7.Instructions for use

The following steps describe how to prepare and administer Loargys at home, as a subcutaneous injection. If you are going to inject this medicine yourself, your doctor or nurse will teach you how to prepare and inject Loargys.

Do not inject this medicine yourself unless you have received training and understand the steps you need to follow.

Your doctor will prescribe the correct dose and tell you what volume (in ml) to inject. You may need more than one vial to get the correct dose and you may need to divide the total dose into more than one injection. Your doctor or nurse will tell you exactly what is right for you.

Each vial is for single use, always use a new vial for each dose.

Loargys should not be mixed with other injection or infusion solutions.

Do not shake.

Preparation:

Make sure you have everything you need for the injection or injections:

• Vial(s) of Loargys
• A graduated syringe
• 1 large needle (e.g., 18 gauge) per vial, for extracting the dose
• 1 small needle (e.g., 26-27 gauge) per injection
• Alcohol wipes
• Gauze pad
• Tirita, if necessary
• Needle container

Retrieving the solution from the vial:

Administration of the dose:

Keep track of the date of the injection and all the locations where it was injected, this will help you use a different injection site for the next injection.

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This information is intended only for healthcare professionals:

Loargys is indicated for intravenous infusion or subcutaneous injection.

Use aseptic technique when preparing and administering Loargys.

Do not shake.

Instructions for preparation

• Determine the total volume of Loargys to be administered (and the number of vials needed) based on the patient's weight and dose level.
• Remove the vial(s) from the refrigerator to allow them to reach room temperature.
• Before administration, visually inspect the vial for the presence of particles and discoloration.
• • Loargys is a colorless to slightly yellow or slightly pink, clear to slightly opalescent (pearly) liquid, essentially free of visible foreign particles.
  • Discard any vial that is not consistent with this appearance.
• Extract the planned dose from the syringe.

• The chemical and physical stability of the prepared dose has been demonstrated for 2hours when stored at room temperature up to 25°C or up to 4hours if stored refrigerated between 2°C and 8°C. If the product is not used within these timeframes, it should be discarded. From a microbiological point of view, the product should be used immediately after reconstitution.

For intravenous administration

• Dilute with sodium chloride 9mg/ml (0.9%) injection solution to obtain the desired infusion volume (maximum concentration of pegzilarginase 0.5mg/ml).
• Administer the intravenous infusion for at least 30 minutes.
• Do not mix other medications with Loargys or infuse other medications simultaneously through the same intravenous access line.

For subcutaneous administration

• Administer the undiluted solution by subcutaneous injection in the abdomen, lateral thigh, or lateral or posterior upper arm. Alternate injection sites between doses.
• Do not inject into scar tissue or areas that are red, inflamed, or swollen.
• Do not inject into the area directly around the navel if injecting into the abdomen.
• If more than 1 injection is needed for a single dose of Loargys, the injection sites should be at least 3 cm apart.

Discard any unused portion of the medicine.

No special disposal requirements are necessary.