Loargys 5 mg/ml solution for injection/infusion – patient leaflet, side effects, dosage

1. What Loargys is and what it is used for

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

Thi pe Th (39

3.

Lo

pro Lo

aged 2 years and older.

Patients with ARG1-D have low levels of an enzyme called arginase. This enzyme helps the body control levels of arginine, an amino acid needed by your body to make proteins. If arginine is not controlled it can build up in the body and cause symptoms, like problems with muscle control.

Loargys is used in combination with other ways to manage the disease. These may include;

• a diet that is low in protein

  Th pe Th do Yo blo Lo vei ap Yo

• food supplements with essential amino acids

• medicines to manage other symptoms of the

  disease, such as medicines that lower levels of ammonia in your body.

How Loargys works

Pegzilarginase, the active substance in Loargys, acts similarly to the natural enzyme arginase, which is lacking or not working properly in patients with ARG1-D. This lowers arginine levels in the blood,

ho tra yo

Al

lea yo

yo

If y

Yo

thereby reducing the disease symptoms.

2. What you need to know before you are given Loargys

You must not be given Loargys

• if you have had a severe allergic reaction to

  pegzilarginase or any of the other ingredients of this medicine (listed in section 6).

Warnings and precautions

am Lo

low dia tha sh

tre

If y

If y

Loargys may cause allergic reactions. This is most likely to occur after the first few doses, but may also occur later in treatment.

If possible, stop the administration immediately and follow your doctor's advice, including seeking immediate medical support if you experience any of the following symptoms of a severe allergic reaction: hives, generalised itching, tightness of the chest, difficulty breathing or low blood pressure. Your doctor

do Yo a f be

If y

Yo Lo lev

y decide you need additional medical treatment to her prevent or treat an allergic reaction. ring your treatment, your doctor will do blood tests check what dose of Loargys is right for you.

ildren and adolescents

e medicine should not be used in children under ears of age, because it is not known if Loargys is e and effective in this age group.

her medicines and Loargys

l your doctor if you are taking, have recently taken might take any other medicines.

egnancy, breast-feeding and fertility ou are pregnant or breast-feeding, think you may pregnant or are planning to have a baby, ask your ctor for advice before using this medicine. Loargys not recommended for use if you are pregnant.

s not known if the medicine passes into breast milk. ou are breast-feeding, ask your doctor for advice fore taking this medicine. Your doctor will help you cide whether to stop breast-feeding or to stop atment.

iving and using machines

argys has no or negligible influence on the ability to ve and use machines.

argys contains sodium and potassium s medicine contains less than 1 mmol sodium (23 mg) r dose, that is to say essentially ‘sodium-free’. is medicine contains potassium, less than 1 mmol mg) per dose, i.e. essentially ‘potassium-free’.

How Loargys is given

argys will be given to you by a healthcare fessional. Your doctor will decide the amount of argys given to you.

e recommended starting dose of Loargys is 0.1 mg r kilogram of your body weight taken once per week. e dose may be increased or decreased by your ctor to keep your blood arginine levels under control. ur doctor will do regular blood tests to check your od arginine levels and change your dose if needed. argys is given as an infusion (drip) directly into your n or as an injection under the skin, as considered propriate by your doctor.

ur doctor may decide you can be given Loargys at me, as an injection under the skin. After being ined by the doctor or nurse, you can inject Loargys urself, see instructions in section 7 below. ways use this medicine exactly as described in this flet or as your doctor, pharmacist or nurse has told u. Check with your doctor, pharmacist, or nurse if u are not sure.

ou are given more Loargys than you should ur doctor will ensure that you receive the right ount of Loargys. If you have been given too much argys, your blood arginine level may become too . Symptoms may include nausea, vomiting, rrhoea and tiredness. If you or your doctor suspects t you have been given more Loargys than you ould, you should be closely monitored and given atment as needed.

ou forget to use Loargys

ou have missed a dose of Loargys, contact your ctor to schedule the next dose as soon as possible. u should not be given a double dose to make up for orgotten dose and there should be at least 4 days tween doses.

ou stop using Loargys

ur doctor will decide if you should stop using argys. If you stop using Loargys, your blood arginine el is likely to increase again.

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

4. Possible side effects

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

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

• Injection site reaction. Symptoms may include pain, swelling, irritation, redness and rash around the site of injection.
• Allergic reaction (hypersensitivity). Symptoms may include swelling of your face, skin rash, sudden redness of the skin (flushing) and difficulty breathing (dyspnoea).

Reporting of side effects

If you get any side effects, talk to your doctor or nurse. This includes any possible side effects not listed in this leaflet. You can also report side effects directly via the Yellow Card Scheme at: www.mhra.gov.uk/yellowcard or search for MHRA Yellow Card in the Google Play or Apple App Store.

By reporting side effects, you can help provide more information on the safety of this medicine.

5. How to store Loargys

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. The expiry date refers to the last day of that month.

Store in a refrigerator (2 °C – 8 °C). Do not freeze. Store in the original carton in order to protect from light. Once removed from the refrigerator, Loargys can be stored for 2 hours at room temperature up to 25 °C. Do not throw away any medicines via wastewater or household waste. These measures will help protect the environment.

6. Contents of the pack and other information

What Loargys contains

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

• The other ingredients are sodium chloride, potassium dihydrogen phosphate, dipotassium phosphate, glycerol, hydrochloric acid, sodium hydroxide and water for injections.

  Loargys contains sodium and potassium, see section 2.

What Loargys looks like and contents of the pack

Loargys is a colourless to slightly yellow or slightly pink, clear to slightly opalescent (pearly) liquid, in a clear glass vial.

Each pack contains 1 vial with either 0.4 ml or 1 ml solution for injection/infusion.

Not all pack sizes may be marketed.

Marketing Authorisation Holder and Manufacturer

Immedica Pharma AB

113 63 Stockholm

Sweden

This leaflet was last revised in 08/2025.

This medicine has been authorised under ‘exceptional circumstances’. This means that because of the rarity of this disease it has been impossible to get complete information on this medicine.

The Medicines and Healthcare products Regulatory Agency will review any new information on this medicine every year and this leaflet will be updated as necessary.

1. 7. 7. Instructions for use

The steps below describe how to prepare and give Loargys at home, as an injection under the skin. If you are injecting this medicine yourself, you will be trained how to prepare and inject Loargys by your doctor or nurse.

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

Your doctor will prescribe your correct dose and will 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 only, always use new vial(s) for each dose.

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

Do not shake.

Preparation:

Make sure you have everything you need for the injection(s):

• Loargys vial(s)
• A graduated syringe
• 1 large needle (e.g. 18 Gauge) per vial, to withdraw the dose
• 1 small needle (e.g. 26-27 Gauge) per injection
• Alcohol wipes
• Gauze pad
• Plaster, if required
• Sharps container
• Check the name and the strength of the vial(s) to make sure it contains the correct medicine and that you have the correct number of vials at hand. Check the expiry date on the carton. Do not use if the product has expired.
• Take the unopened Loargys vial(s) out of the refrigerator 15 to 30 minutes before the planned injection to allow the solution to reach room temperature. Do not use external heat.
• Wash your hands.
• The solution in the vial should be colourless to slightly yellow or slightly pink, clear to slightly opalescent (pearly). Do not use if the solution is cloudy or contains visible particles.
• Place the vial on a clean flat surface. Remove the plastic flip-off cap from the vial.
• Wipe the top of the vial with an alcohol swab and allow to air dry. Do not touch the top of the vial or allow it to touch anything else once wiped.

Withdrawing solution from the vial:

1. 1.

   Attach a large needle to the graduated syringe.

   Remove the needle cap.

2. 2. Pull the plunger back to draw air into the graduated syringe, equal to the volume to withdraw from the vial (in ml).

Product name: Loargys

Strength: 5 mg/ml

Formulation: solution for injection/infusion

Countries: UK

Languages: en

Component: PIL

N/A

Dimensions: 420 x 297 mm

Internal article no.: 0411PIL02

External article no.: PX1254/1Y

Version: 01

Date changed: 2025-07-10

0411PIL02

1. 3.

   Keep the vial on a flat

   surface, slowly insert the

   needle through the rubber

   seal into the vial.

   1. a)

      For single or first vial:

      The tip of the needle

      should not touch the

      solution to avoid

      forming foam. Continue

      to step 4.

   2. b)

      For subsequent vial(s):

      Turn the vial upside

      down, ensure the needle

      tip is in the air space

      above the solution to

      avoid forming foam.

2. 4.

   Slowly push the plunger to inject the air into the

   vial.

3. 5.

   Keep the needle in the vial

   and hold it upside down. With

   the needle in the solution,

   slowly pull the plunger to the

   mark equal to the volume

   needed.

4. 6.

   Before removing the needle

   from the vial, check the

   solution in the syringe for air

   bubbles. If there are bubbles,

   continue to hold the vial

   upside down with the needle

   pointing upwards. Gently tap

   the barrel of the syringe with

   your finger. Once all the air

   bubbles are at the top, gently

   push the plunger to push out

   the bubbles through the

   needle.

5. 7.

   Check the volume to give (in ml), against the

   markings on the syringe to make sure that you have withdrawn the correct amount of solution.

6. 8.

   Pull the needle from the vial, put the needle cap

   back on and dispose it in your sharps container.

7. 9.

   You may need to use several vials to withdraw the

   complete volume (in ml). To do this, repeat steps 1-8 above for each vial needed to obtain the total dose (in ml) or as shown to you by your doctor or nurse. Always follow the instructions given to you by your doctor or nurse. Please note that for each new vial you also should use a new needle.

8. 10.

   Place a small needle on the filled syringe, do not

   remove the needle cap. Ensure the needle sits tight.

   Note: If the solution is not to be used immediately,

   protect the syringe from light. After preparation Loargys can be stored at room temperature (up to 25 °C) for up to 2 hours before administration. After this time, the prepared Loargys cannot be used anymore and must be discarded.

   Giving the dose:

   1. 1.

      Remove the needle cap. Hold the syringe with the

      needle pointing up and tap the barrel of the syringe with your finger to remove any air bubbles. Control visually that the volume contained in

      the syringe is correct. The volume per injection should not exceed 1 ml. If it is the case, multiple injections should be injected at different sites.

   2. 2.

      Choose an injection site

      (abdomen, side of the thigh,

      or the side or back of the

      upper arms). Rotate injection

      sites between doses.

      Do not inject into scar tissue

      or areas that are reddened,

      inflamed, or swollen. If

      injecting into the abdomen,

      avoid the area directly

      surrounding the navel.

      If more than 1 injection is

      needed for a single dose of

      Loargys, the injection sites

      should be at least 3 cm

      apart.

   3. 3.

      Clean the injection site using an alcohol swab and

      allow the skin to dry.

   4. 4.

      Gently pinch the skin of the

      chosen injection site

      between your thumb and

      index finger.

   5. 5.

      Hold the syringe like a pencil

      or dart. Insert the needle into

      the raised skin at a 45° to

      90° angle.

   6. 6.

      While continuing to pinch

      the skin, slowly push the

      plunger until you have

      injected the desired volume.

      Reminder: If you need to

      inject a volume of more than

      1 ml of Loargys, switch the

      injection site and make sure

      that the new injection site is

      more than 3 cm away.

      Slowly press the plunger

      down until you have injected

      the volume needed. Repeat

      until you have injected your

      total dose (in ml). Always

      use a new small needle for

      each injection.

   7. 7.

      Remove the syringe by pulling it straight out.

      Release the pinched skin and gently press a gauze pad over the injection site for a few seconds. Apply a plaster if needed.

      1. 8. Put your used syringe, needles and caps in the sharps container. Used vials, even if not empty, should be discarded according to your local guidelines.

Note the date of the injection and all the sites where you have injected. This helps you to use a different injection site for the next injection.

---------------------------------------------------------------

The following information is intended for healthcare professionals only:

Loargys is intended for intravenous infusion or subcutaneous injection.

Use aseptic technique when preparing and administering Loargys.

Do not shake.

Instruction 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 reach room temperature.

• Inspect the vial visually for particulate matter and discoloration prior to administration.

  ο Loargys is a colourless to slightly yellow or slightly pink, clear to slightly opalescent liquid, essentially free of visible foreign particles. ο Discard any vial(s) not consistent with this appearance.

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

For intravenous administration

• Dilute with sodium chloride 9 mg/ml (0.9 %)

  solution for injection to achieve the desired volume of infusion (maximum pegzilarginase concentration 0.5 mg/ml).

• Administer the intravenous infusion over at least

  30 minutes.

• Do not mix other medicines with Loargys or infuse other medicines concomitantly via the same intravenous access line.

For subcutaneous administration

• Administer the undiluted solution as subcutaneous injection into the abdomen, lateral part of the thigh, or the side or back of the upper arms. Rotate injection sites between doses.
• Do not inject into scar tissue or areas that are reddened, inflamed, or swollen.
• If injecting into the abdomen, avoid the area directly surrounding the navel.
• If more than 1 injection is needed for a single dose of Loargys, the injection sites should be at least 3 cm apart.

Discard unused portion of the medicine.

No special requirements for disposal.