Loargys 5 mg/ml solution for injection and for infusion

Spain
Brand name Loargys 5 mg/ml solution for injection and for infusion
Form solution for injection and for infusion
Active substance / Dosage
PEGZILARGINASE · 2 mg
Prescription type Hospital Use Only
ATC code
A16A A16AB A16AB24
Registration number 1231774001
Manufacturer Immedica Pharma Ab
Loargys 5 mg/ml solution for injection and for infusion solution for injection and for infusion

Table of Contents

Package leaflet: Information for the patient

Introduction

Package leaflet: information for the patient

Loargys 5 mg/ml solution for injection and infusion

pegzilarginase

This medicine is subject to additional monitoring, which will allow rapid detection of new safety information. You can help by reporting any side effects you may experience. The end of section 4 includes information on how to report these side effects.

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

  • Keep this leaflet, as you may need to read it again.
  • If you have any questions, consult your doctor or nurse.
  • If you experience any side effects, consult your doctor or nurse, even if they are side effects not listed in this leaflet. See section 4.

Leaflet contents

  1. What Loargys is and what it is used for
  2. What you need to know before receiving Loargys
  3. How Loargys is administered
  4. Possible side effects
  5. How to store Loargys
  6. Contents of the pack and other information
  7. Instructions for use

1. What Loargys is and what it is used for

Loargys contains the active substance pegzilarginase, which is a modified human enzyme produced using recombinant DNA technology. The medicine is used to treat arginase 1 deficiency (ARG1-D), also known as hyperargininemia, in adults, adolescents, and children from 2 years of age.

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

Loargys is used in combination with other treatments for the disease, which may include:

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

How Loargys works

Pegzilarginase, the active substance in Loargys, works similarly to the natural enzyme arginase, which is missing or not functioning properly in patients with ARG1-D. This reduces arginine levels in the blood, thereby reducing the symptoms of the disease.

2. What you need to know before starting Loargys

Do not take Loargys

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

Warnings and precautions

Loargys may cause allergic reactions. This is more likely to occur after the first doses, although they may also appear later during treatment.

If possible, immediately stop administration and follow your doctor's advice, including seeking immediate medical attention if you experience any of the following symptoms of a severe allergic reaction: hives, widespread itching, tightness in the chest, difficulty breathing, or low blood pressure. Your doctor may decide that you require additional medical treatment to prevent or treat an allergic reaction.

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

Children and adolescents

This medicine should not be used in children under 2 years of age, as it is not known whether Loargys is safe and effective in this age group.

Use of Loargys with other medicines

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

Pregnancy, breastfeeding, and fertility

If you are pregnant or breastfeeding, think you may be pregnant, or are planning to become pregnant, consult your doctor before using this medicine. The use of Loargys is not recommended during pregnancy.

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

Driving and using machines

The influence of Loargys on the ability to drive and use machines is none or negligible.

Loargys contains sodium and potassium

This medicine contains less than 1 mmol of sodium (23 mg) per dose, i.e., essentially "sodium-free". This medicine contains potassium, less than 1 mmol (39 mg) per dose, i.e., essentially "potassium-free".

3. How Loargys is administered

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

The recommended initial dose of Loargys is 0.1 mg per kilogram of body weight, given once a week. Your doctor may increase or decrease the dose to keep your blood arginine levels under control. Your doctor will prescribe periodic blood tests to monitor your blood arginine levels and adjust your dose if necessary.

Loargys is administered either as an intravenous infusion (drip) directly into a vein or as a subcutaneous injection, as determined appropriate by your doctor.

Your doctor may decide that you can receive Loargys at home as a subcutaneous injection. After receiving training from your doctor or nurse, you may self-inject Loargys (see instructions in section 7).

Always use this medicine exactly as described in this leaflet or as your doctor, pharmacist, or nurse has advised. 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 been given too much Loargys, your blood arginine level may become too low. Symptoms may include nausea, vomiting, diarrhoea, and fatigue. If you or your doctor suspect that you have received more Loargys than you should have, you must be closely monitored and receive any necessary treatment.

If you forget to use Loargys

If you have missed a dose of Loargys, contact your doctor to schedule the next dose as soon as possible. You must not be given a double dose to make up for missed doses, and at least 4 days must elapse between doses.

If you stop treatment with Loargys

Your doctor will decide whether you should stop using Loargys. If you interrupt treatment with Loargys, your blood arginine level is likely to rise again.

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

4. Possible adverse effects

Like all medicines, this medicine can cause adverse effects, although not everyone will experience them.

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

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

Reporting of adverse effects

If you experience any type of adverse effect, consult your doctor or nurse, even if it is an effect not listed in this leaflet. You may also report them directly through the national reporting system detailed in Appendix V. By reporting adverse effects, you can help provide more information on the safety of this medicine.

5. Storage of Loargys

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

Do not use this medicine after the expiry date stated on the label. The expiry date refers to the last day of the month indicated.

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

After removal from the refrigerator, Loargys may be stored for up to 2 hours at room temperature not exceeding 25 °C.

Medicines must not be disposed of via wastewater or household waste. Doing so helps protect the environment.

6. Contents of the container and other information

Composition of Loargys

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

Appearance of the product and contents of the pack

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

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

Only certain pack sizes may be marketed.

Marketing Authorisation Holder and Manufacturer

Immedica Pharma AB
113 63 Stockholm
Sweden

Date of the most recent review of this leaflet

This medicine has been authorised under "exceptional circumstances". This means that, because the disease is rare, complete information about this medicine could not be obtained.

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

Other sources of information

Detailed information on this medicine is available on the website of the European Medicines Agency: . Links to other websites on rare diseases and orphan medicinal products are also available.

This leaflet can be found on the European Medicines Agency website in all languages of the European Union/European Economic Area.

You can also find this leaflet and the training materials approved by health authorities for this medicine by scanning the QR code below with a smartphone or via the website http://www.loargyspatient.eu

Square QR code composed of black pixels on a white background with three large positioning squares at the corners
  1. Instructions for use

The steps below describe how to prepare and administer Loargys at home as a subcutaneous injection. If you are 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 been trained and understand the steps 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 obtain 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 appropriate for you.

Each vial is for single use only; always use new vial(s) for each dose.

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

Do not shake.

Preparation:

Ensure you have everything you need for the injection(s):

  • Loargys vial(s)
  • A graduated syringe
  • 1 large needle (e.g., gauge 18) per vial, to withdraw the dose
  • 1 small needle (e.g., gauge 26–27) per injection
  • Alcohol wipes
  • Gauze pad
  • Plaster, if needed
  • Sharps container
  1. Check the vial label to confirm the name and concentration to ensure it contains the correct medicine and that you have the correct number of vials. Check the expiry date on the packaging. Do not use if the product has expired.
  1. Remove the unopened Loargys vial(s) from the refrigerator 15 to 30 minutes before the scheduled injection to allow the solution to reach room temperature. Do not use external heat sources.
  1. Wash your hands.
  1. The solution in the vial should be colorless to slightly yellow or slightly pink, clear to slightly opalescent (pearly). Do not use if the solution is cloudy or contains visible particles.
  1. Place the vial on a clean, flat surface. Remove the plastic flip-off cap.
  1. Clean the top of the vial with an alcohol swab and allow it to air dry. Do not touch the top of the vial or allow it to come into contact with anything after cleaning.

Withdrawing the solution from the vial:

  1. Attach a large needle to the graduated syringe. Remove the needle cap.
  1. Pull back the plunger to draw air into the graduated syringe,
  2. equal to the volume to be withdrawn from the vial (in ml).

Two hands holding a syringe vertically with the needle pointing downward

  1. Place the vial on a flat surface and slowly insert the needle through the rubber stopper into the vial.
  • For single vial or first vial: The tip of the needle should not touch the solution to avoid foaming. Proceed to step 4.
  • For subsequent vial(s): Turn the vial upside down and ensure the needle tip is in the air space above the solution to avoid foaming.

One hand holding a syringe vertically with needle while the

Two hands holding a syringe with the needle pointing downward, while an upper hand grips the top and the

  1. Slowly push the plunger to inject the air into the vial.
  1. Keep the needle in the vial and hold it upside down. With the needle in the solution, slowly pull back the plunger to the mark corresponding to the required volume.

Two hands holding a syringe with a black arrow pointing downward indicating the plunger's downward pressure movement toward the

  1. Before removing the needle from the vial, check for air bubbles in the syringe solution. If bubbles are present, keep the vial upside down with the needle pointing upward. Gently tap the barrel of the syringe with your finger. Once all air bubbles have risen to the top, gently push the plunger to expel the air bubbles through the needle.

Two hands holding a glass vial while a third hand inserts the

  1. Verify the volume to be administered (in ml) using the syringe markings to ensure the correct amount of solution has been withdrawn.
  1. Remove the needle from the vial, replace the needle cap, and dispose of it in a sharps container.
  1. You may need to use multiple vials to withdraw the full volume (in ml). To do so, repeat steps 1–8 above for each vial required to obtain the total dose (in ml), or as directed by your doctor or nurse. Always follow the instructions provided by your doctor or nurse. Note that for each new vial, a new needle must also be used.
  1. Attach a small needle to the filled syringe and do not remove the needle cap. Make sure the needle is securely attached.

NOTE: If the solution is not to be used immediately, protect the syringe from light. After preparation, Loargys may be stored at room temperature (up to 25°C) for a maximum of 2 hours prior to administration. After this time, the prepared Loargys must not be used and should be discarded.

Administration of the dose:

  1. Remove the needle cap. Hold the syringe with the needle pointing upward and tap the barrel of the syringe gently with your finger to remove any air bubbles.

Visually check that the volume contained in the syringe is correct. The injection volume must not exceed 1 ml. If necessary, multiple injections should be administered at different sites.

  1. Select an injection site (abdomen, outer side of the thigh, or outer or back side of the upper arms). Rotate injection sites between doses.

Do not inject into scar tissue or in areas that are red, inflamed, or swollen. If injecting into the abdomen, avoid the area immediately surrounding the navel.

If more than one injection is required for a single dose of Loargys, injection sites must be at least 3 cm apart from each other.

Stylized drawing of a male human body with several dark spots distributed across the arms, torso, and legs

  1. Clean the injection site with an alcohol swab and allow the skin to dry.
  1. Gently pinch the skin at the selected injection site between your thumb and index finger.

Line drawing of a hand pinching the skin of the arm to prepare the subcutaneous injection site

  1. Hold the syringe like a pencil or dart. Insert the needle into the pinched skin at an angle of 45° to 90°.

Technical illustration showing a hand inserting a needle with a syringe at a 90-degree or 45-degree angle into the skin

  1. While continuing to pinch the skin, slowly push the plunger until the desired volume has been injected.

Reminder: if you need to inject a volume greater than 1 ml of Loargys, change the injection site and ensure the new injection site is more than 3 cm away. Slowly press the plunger until the required volume has been injected. Repeat until your total dose (in ml) has been administered. Always use a new small needle for each injection.

A hand holding a syringe and inserting the

  1. 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 bandage if necessary.
  1. Place the syringe, needles, and caps used into a sharps container. Used vials, even if not empty, should be disposed of according to local regulations.

Keep a record of the date of the injection and all the sites where injections have been administered; this will help you use a different injection site for the next injection.

This information is intended for healthcare professionals only:

Loargys is indicated for intravenous infusion or subcutaneous injection.

Use an aseptic technique when preparing and administering Loargys.

Do not shake.

Preparation instructions

  • Determine the total volume of Loargys to be administered (and the number of vials required) based on the patient's weight and dose level.

  • Remove the vial(s) from the refrigerator and allow them to reach room temperature.

  • Before administration, visually inspect the vial for particles and discoloration.

    • Loargys is a colorless to slightly yellow or slightly pink, clear to slightly opalescent liquid, essentially free from visible foreign particles.
    • Discard any vial that does not conform to this appearance.

  • Withdraw the prescribed dose into a syringe.

  • The prepared dose has been shown to be chemically and physically stable for 2 hours when stored at room temperature up to 25°C, or for up to 4 hours if refrigerated between 2°C and 8°C. If the product is not used within these time frames, it must be discarded. From a microbiological standpoint, 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 infusion volume (maximum pegzilarginase concentration of 0.5 mg/mL).
  • Administer the intravenous infusion over at least 30 minutes.
  • Do not mix other medications with Loargys or infuse other drugs simultaneously through the same intravenous line.

For subcutaneous administration

  • Administer the undiluted solution by subcutaneous injection into the abdomen, the outer area of the thigh, or the side or back of the upper arms. Rotate injection sites between doses.
  • Do not inject into scar tissue or into areas that are red, swollen, or inflamed.
  • If injecting into the abdomen, avoid the area immediately surrounding the umbilicus.
  • If more than one injection is required to administer a single dose of Loargys, the injection sites should be separated from each other by at least 3 cm.

Discard any unused portion of the medication.

No special disposal requirements are needed.