Loargys – Package Leaflet: InfoCons – Consumer Protection presents, in an accessible format, the official package leaflet of the medicine Loargys, as authorised in the European Union and published in the Union Register of medicinal products.
Before anything else
Read all of this leaflet carefully before you are given 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 or nurse.
- If you get any side effects, talk to your doctor, or nurse. This includes any possible side effects not listed in this leaflet. See section 4.
ℹ️ 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 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
- 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, 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
Loargys may cause allergic reactions. This is most likely to occur after the first few doses, but may also occur later in treatment .
26 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 may decide you need additional medical treatment to either prevent or treat an allergic reaction.
During your treatment, your doctor will do blood tests to check what dose of Loargys is right for you.
Children and adolescents
The medicine should not be used in children under 2 years of age, because it is not known if Loargys is safe and effective in this age group.
Other medicines and Loargys
Tell your doctor if you are taking, have recently taken or might take any other medicines.
Pregnancy, breast -feeding and fertility If you are pregnant or breast-feeding, think you may be pregnant or are planning to have a baby, ask your doctor for advice before using this medicine. Loargys is not recommended for use if you are pregnant.
It is not known if the medicine passes into breast milk. If you are breast-feeding, ask your doctor for advice before taking this medicine. Your doctor will help you decide whether to stop breast-feeding or to stop treatment.
Driving and using machines
Loargys has no or negligible influence on the ability to drive and use machines.
Loargys contains sodium and potassium
This medicine contains less than 1 mmol sodium (23 mg) per dose, that is to say essentially ‘sodium – free’. This medicine contains potassium, less than 1 mmol (39 mg) per dose, i.e. essentially ‘potassium- free’.
💊 3. How Loargys is given
Loargys will be given to you by a healthcare professional. Your doctor will decide the amount of Loargys given to you.
The recommended starting dose of Loargys is 0.1 mg per kilogram of your body weight taken once per week. The dose may be increased or decreased by your doctor to keep your blood arginine levels under control. Your doctor will do regular blood tests to check your blood arginine levels and change your dose if needed.
Loargys is given as an infusion (drip) directly into your vein or as an injection under the skin, as considered appropriate by your doctor.
Your doctor may decide you can be given Loargys at home, as an injection under the skin. After being trained by the doctor or nurse, you can inject Loargys yourself, see instructions in section 7 below.
Always use this medicine exactly as described in this leaflet or as your doctor, pharmacist or nurse has told you. Check with your doctor, pharmacist, or nurse if you are not sure.
If you are given more Loargys than you should
Your doctor will ensure that you receive the right 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 tiredness. If you or your doctor suspects that you have been given more Loargys than you should, you should be closely monitored and given treatment as needed.
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 should not be given a double dose to make up for a forgotten dose and there should be at least 4 days between doses.
If you stop using Loargys Your doctor will decide if you should stop using Loargys. If you stop using Loargys, your blood arginine level 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. T his includes any possible side effects not listed in this leaflet. You can also report side effects directly via 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. 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.
28 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
Manufacturer
Unimedic AB Storjordenvägen 2 864 31 Matfors Sweden T his leaflet was last revised in .
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 European Medicines Agency will review any new information on this medicine every year and this leaflet will be updated as necessary.
Other sources of information Detailed information on this medicine is available on the European Medicines Agency web site: https://www.ema.europa.eu . There are also links to other websites about rare diseases and treatments.
This leaflet is available in all EU/EEA languages on the European Medicines Agency website.
You can also find this leaflet and regulatory approved training material on this medicine when scanning the QR code below with a smartphone or via the website http://www.loargyspatient.eu .
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 29 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
1. 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.
2. 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.
3. Wash your hands.
4. 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.
5. Place the vial on a clean flat surface. Remove the plastic flip -off cap from the vial.
6. 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. Attach a large needle to the graduated syringe. Remove the needle cap.
2. Pull the plunger back to draw air into the graduated syringe, equal to the volume to withdraw from the vial (in ml).
3. Keep the vial on a flat surface, slowly insert the needle through the rubber seal into the vial. a) For single or first vial : The tip of the needle should not touch the solution to avoid forming foam. Continue to step 4. 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.
4. Slowly push the plunger to inject the air into the vial.
30 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.
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.
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.
8. Pull the needle from the vial , put the needle cap back on and dispose it in your sharps container.
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.
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. 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. 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. Clean the injection site using an alcohol swab and allow the skin to dry.
4. Gently pinch the skin of the chosen injection site between your thumb and index finger.
31 5. Hold the syringe like a pencil or dart. Insert the needle into the raised skin at a 45° to 90° angle.
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. 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.
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. ——————————————————————————————————————–
🩺 Information 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. o Loargys is a colourless to slightly yellow or slightly pink, clear to slightly opalescent liquid, essentially free of visible foreign particles. o 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 dis carded. 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 32 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.
ANNEX I V SCIENTIFIC CONCLUSIONS AND GROUNDS FOR THE VARIATION TO THE TERMS OF THE MARKETING AUTHORISATION (S) 34 Scientific conclusions Taking into account the PRAC Assessment Report on the PSUR (s) for pegzilarginase, the scientific conclusions of PRAC are as follows:
In view of available data on hypersensitivity from the active surveillance programme including two cases of hypersensitivity after subcutaneous administration, one of them severe, in a close temporal relationship and in view of a plausible mechanism of act ion, the PRAC considers an amendment of the concerned sections of the product information of medicinal products containing pegzilarginase necessary.
Having reviewed the PRAC recommendation, the CHMP agrees with the PRAC overall conclusions and grounds for recommendation.
Grounds for the variation to the terms of the Marketing Authorisation (s) On the basis of the scientific conclusions for pegzilarginase the CHMP is of the opinion that the benefit- risk balance of the medicinal product(s) containing pegzilarginase is unchanged subject to the proposed changes to the product information.
The CHMP recommends that the terms of the Marketing Authorisation(s) should be varied.
📄 Package Leaflet – official source
This article reproduces the information in the official package leaflet, as authorised in the European Union. You can consult the original document here: Product information Loargys (PDF, ec.europa.eu).
You can check this medicine at any time in the Union Register of medicinal products (EU number EU/1/23/1774).
⚠️ Important: This article is for information purposes only and does not replace medical advice or the package leaflet inside the medicine box. Read the leaflet carefully before use. For any questions about your treatment, talk to your doctor or pharmacist. If you experience side effects, tell your doctor or pharmacist and report them through your national reporting system.