Nezglyal – Package Leaflet: InfoCons – Consumer Protection presents, in an accessible format, the official package leaflet of the medicine Nezglyal, 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 giving this medicine to your child because it contains important information for you and your child . − Keep this leaflet. You may need to read it again. − If you have any further questions, ask your child’s doctor. − This medicine has been prescribed for your child only. Do not pass it on to others. It may harm them, even if their signs of illness are the same as those of your child. − If your child gets any side effects, talk to your child’s doctor . This includes any possible side effects not listed in this leaflet. See section 4.
ℹ️ 1. What Nezglyal is and what it is used for
Nezglyal contains the active substance leriglitazone. It is part of a group of medicines known as peroxisome proliferator -activated receptor gamma ( PPARγ ) agonists.
Nezglyal is a treatment for a rare brain disease in boys called cerebral adrenoleukodystrophy (cALD).
Nezglyal is used in boys aged 2 to 12 years with cALD , who have brain lesions that do not show disruption of the blood brain barrier, refe rred as “Gd negative ” lesions (these are areas that do not take up the contrast agent gadolinium and therefore do not light up on Magnetic Resonance Imaging ( MRI )) and who have either no or mild clinical symptoms (meaning a Neurological Functiona l Score (NFS) of 0 or 1 ).
Cerebral adrenoleukodystrophy (cALD) is a form of an inherited disease called adrenoleukodystrophy in which fatty substances known as ‘very long chain fatty acids’ build up in tissues around the body, mainly in the brain, spinal cord and adrenal glands (two glands situated above the kidneys) . In cALD, a build- up of these substances in the brain causes inflammation and destruction of the protective sheath (myelin) that insulates and helps signalling by nerve cells. This leads to brain lesions.
People with ALD may develop cALD in childhood . People can develop Gd negative lesions, with either no or mild clinical symptoms . These lesions can progress further to more serious disease which can be life-threatening.
The active substance in Nezglyal, leriglitazone , works by attaching to and activating a target (receptor) in the body known as PPAR γ receptor. PPAR γ receptors play a role in regulating the function of mitochondria (energy- producing structures in cells), how cells respond to oxidative stress (damage caused by toxic oxygen- containing molecules known as free radicals) and inflammation . Leriglitazone protects nerve cells from damage caused by cALD by reducing inflammation, improving the function of mitochondria and protecting against damage from free radicals .
⚠️ 2. What you need to know before you use Nezglyal
Do not use Nezglyal
− if your child is allergic to leriglitazone or any of the other ingredients of this medicine (listed in section 6). − if your child is allergic to a type of medicine known as ‘thiazolidinediones’ such as pioglitazone (used to lower blood sugar levels in people with type 2 diabetes). − if your child ha s or ever had heart failure.
Warnings and precautions
Talk to your child’s doctor before giving Nezglyal to your child. − Side effects with Nezglyal treatment have been reported more often during the first six months of treatment . In particular, side effects of weight gain and water retention leading to oedema with signs such as swelling of legs, feet or ankles. Your child’s doctor will monitor this and your child’s treatment with Nezglyal may be stopped if severe symptoms of oedema or cardiac failure occur. − Some patients who experience water retention may develop heart failure. Inform your child’s doctor as soon as possible if your child experiences signs of heart failure such as unusual shortness of breath, increased swelling of legs, feet, or ankles in spite of measures taken by the doctor, discomfort or swelling in the abdomen, trouble sleeping with breathlessness when lying flat in bed , chest pain or palpitations, frequent dry, hacking cough . − Treatment with Nezglyal must be stopped at least 5 days before your child starts the conditioning medicines used in haematopoietic stem cell transplantation , a procedure where a patient’s bone marrow is cleared of cells and replaced by stem cells to form new bone marrow. − If your child has impaired liver function, their doctor will carefully consider whether this medicine is suitable for them and may wish to monitor them carefully. − Inform your child’s doctor immediately if you notice any blood in your child’s urine or experience painful or difficult urination during treatment. Before giving Nezglyal to your child, your child will have a blood sample taken to check their liver function. This check may be repeated at regular intervals. Inform your child’s doctor straight away if your child develops symptoms of liver disease such as unexplained nausea, vomiting or abdominal pain, weight loss, dark urine, or yellowing of the skin (jaundice). − If your child has osteoporosis or are at risk of bone fracture, talk to their doctor. Medicines in the same class have been associated with an increased risk of broken bones. It is not known whether Nezglyal may affect bone development or bone strength in children. The doctor will monitor your child’s growth and bone health during treatment. − Nezglyal must not be used together with any blood‑glucose‑lowering medicines, because this may increase the risk of low blood sugar (hypoglycaemia). If your child has diabetes type 1 or 2 and is taking any anti -diabetic medicine, their doctor will carefully consider whether this medicine is suitable for them and may wish to monitor them carefully. − If your child has a special type of eye disease called macular oedema (swelling of the back of the eye), their doctor may refer them for an eye examination and decide whether treatment should be continued.
Children under the age of 2 years This medicine should not be given to children under the age of 2 years as it has not been tested i n this age group.
Other medicines and Nezglyal
Tell your child’s doctor if your child is taking, has recently taken or might take any other medicines.
The following medicines may increase the risk of side effects of Nezglyal:
- gemfibrozil (used to treat high cholesterol)
- itraconazole (used to treat fungal infections) 27 – clarithromycin (used to treat bacterial infections)
- metformin (used to treat diabetes) or other medicines that lower blood sugar , such as insulin, gliclazide, glimepiride or glibenclamide The following medicines may reduce the effectiveness of Nezglyal:
- carbamazepine (used to treat certain types of seizures in people with epilepsy, pain in trigeminal neuralgia, or mania in bipolar disorder)
- rifampicin (used to treat certain bacterial infections, including tuberculosis )
- phenytoin (used to treat epilepsy)
- phenobarbital (used to treat epilepsy) The following medicines may require special monitoring when taken with Nezglyal :
- rosuvastatin (used to treat high cholesterol)
- methotrexate (used to treat certain inflammatory diseases and some cancers )
- sulfasalazine (used to treat inflammatory bowel disease ) Your child’s doctor will decide whether these medicines can be taken together and may monitor your child more closely.
Driving and using machines
This medicine may have minor influence on the ability to drive, ride a bicycle and use machines. If your child experiences side effects affecting their vision, they should not ride a bicycle or use machines until their vision is back to normal (see section 4 “Possible side effects”).
Nezglyal contains sorbitol, sodium and sodium benzoate
This medicine contains 8000 mg sorbitol in each 100 mL which is equivalent to 80 mg/mL. Sorbitol is a source of fructose. If your child’s doctor has told you that your child ha s an intolerance to some sugars or if your child has been diagnosed with hereditary fructose intolerance (HFI), a rare genetic disorder in which a person cannot break down fructose, talk to their doctor before your child take s or receive s this medicine.
This medicine contains 200 mg sodium (main component of cooking/table salt) in each 100 mL which is equivalent to 2 mg/mL. This is equivalent to 0.10% of the recommended maximum daily dietary intake of sodium for an adult.
This medicine contains 100 mg sodium benzoate in each 100 mL which is equivalent to 1 mg/mL.
💊 3. How to take Nezglyal
Always give this medicine to your child exactly as your child’s doctor has told you. Check with their doctor if you are not sure.
Treatment with this medicine should be started and supervised by a doctor experienced in the treatment of diseases that cause nerve cells to stop functioning properly.
Nezglyal is available as a suspension that is taken by mouth once daily.
The recommended daily dose will be calculated by your child’s doctor based on your child’s age, weight and height. Your child will need to attend regular appointments with their doctor to ensure they continue to receive the correct dose as they grow.
Give Nezglyal to your child at the same time every day, preferably after breakfast.
28 Your child’s doctor or pharmacist will show you how to measure the correct dose. Use the dosing syringe which is provided with Nezglyal to measure your or your child’s daily dose.
Instructions for use
- Always shake the bottle well for about 30 seconds before each dose.
- Put the bottle on a horizontal, stable surface and remove the cap.
- Insert the dosing syringe which is provided in the pack into the bottle and withdraw the recommended daily dose.
- Empty the contents of the oral dosing syringe into your child’s mouth and make sure your child swallows the dose.
- After giving the dose to your child , close the bottle. Wash the oral dosing syringe with clean water and dry with a paper towel. Place the bottle and oral dosing syringe back in its original package in order to protect from light .
If you give more Nezglyal to your child than you should If you accidentally give your child too much medicine, or if someone else or a nother child takes this medicine, talk to a doctor or pharmacist immediately and take this package leaflet with you .
If you forget to give Nezglyal to your child If your child misses a dose , just carry on with the next dose as normal. Do not give a double dose to make up for a forgotten dose.
If you r child stop s taking Nezglyal Nezglyal should be taken every day to work properly. Talk to your child’s doctor before stopping this treatment.
If you have any further questions on the use of this medicine, ask your child’s doctor.
❗ 4. Possible side effects
Like all medicines, this medicine can cause side effects, although not everybody gets them.
Some side effects are more common at the beginning of treatment and may require monitoring by your child’s doctor.
The following side effects have been reported in children treated with Nezglyal:
Very common (may affect more than 1 in 10 people)
− Swelling due to fluid retention, including swelling of your child’s lower legs , face and/or eyelids (oedema)
Common (may affect up to 1 to 10 people)
− Changes in blood tests, such as: o Low levels of red blood cells (anaemia) o Unusual balance of different types of white blood cells o Blood takes longer to clot (increased international normalised ratio (INR)) o Low levels of white blood cells (leukopenia ) o Low levels of lymphocytes, a type of white blood cell (lymphopenia) o Low levels of neutrophils, a type of white blood cell (neutropenia) o Increased levels of prothrombin ( a protein required for the blood to clot) o Increased levels of a heart -related blood test marker (N-terminal prohormone brain natriuretic peptide ) − Increased appetite − Weight gain − Headache 29 − Night sweats
Reporting of side effects
If your child gets any side effects, talk to your child’s doctor or surgeon. This 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 Nezglyal
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 carton and the label after EXP. The expiry date refers to the last day of that month.
Store in the original package in order to protect from light .
After first opening: Use up to 66 days below 25 ºC .
Do not throw away any medicines via wastewater or household waste. Ask your pharmacist how to throw away medicines that your child no longer use s. These measures will help protect the environment.
📦 6. Contents of the pack and other information
What Nezglyal contains
The active substance is leriglitazone (as hydrochloride) .
Each mL of oral suspension contains 13.7 mg of leriglitazone (as hydrochloride).
One bottle (100 mL) contains 1 370 mg of leriglitazone (as hydrochloride).
The other ingredients are sorbitol (E 420), microcrystalline cellulose (E 460i) and carmellose sodium (E 466), saccharin sodium (E 954), sodium benzoate (E 211), sodium citrate (E 331), citric acid monohydrate (E 330), strawberry flavour, and purified water (see also section 2 ‘ Nezglyal contains sorbitol, sodium and sodium benzoate’) .
What Nezglyal looks like and contents of the pack
Nezglyal is a homogenous, white oral suspension.
Hydrolytic class III amber glass bottle of 100 mL with a white polyethylene child -resistant cap packaged with a polyethylene oral dosing syringe of 12 mL graduated at every 0.5 mL.
Pack size of one bottle.
Marketing Authorisation Holder
Minoryx Therapeutics S.L.
Av Ernest Lluch, 32, TCM3 08302 Mataró, Barcelona Spain Tel: +34 93 544 14 66 [email protected]
Manufacturer
Delpharm Huningue S.A.S.
26, Rue de la Chapelle 68330 Huningue France 30 For any information about this medicine, please contact the local representative of the Marketing Authorisation Holder:
België/Belgique/Belgien Neuraxpharm Belgium Tél/Tel: +32 (0)2 732 56 95 Lietuva Neuraxpharm Pharmaceuticals, S.L.
Tel: +34 93 475 96 00 България Neuraxpharm Pharmaceuticals, S.L.
Teл.: +34 93 475 96 00 Luxembourg/Luxemburg Neuraxpharm France Tél/Tel: +32 474 62 24 24 Česká republika Neuraxpharm Bohemia s.r.o. Tel: +420 739 232 258 Magyarország Neuraxpharm Hungary Kft.
Tel.: +36 (30) 542 2071 Danmark Neuraxpharm Sweden AB Tlf.: +46 (0)8 30 91 41 (Sverige) Malta Neuraxpharm Pharmaceuticals, S.L.
Tel: +34 93 475 96 00 Deutschland neuraxpharm Arzneimittel GmbH Tel: +49 2173 1060 0 Nederland Neuraxpharm Netherlands B.V Tel: +31 70 208 5211 Eesti Neuraxpharm Pharmaceuticals, S.L.
Tel: +34 93 475 96 00 Norge Neuraxpharm Sweden AB Tlf: +46 (0)8 30 91 41 (Sverige) Ελλάδα Brain Therapeutics ΙΚΕ Τηλ: +302109931458 Österreich Neuraxpharm Austria GmbH Tel: + 43 (0) 1 208 07 40 España Neuraxpharm Spain, S.L.U.
Tel: +34 93 475 96 00 Polska Neuraxpharm Polska Sp. z.o.o.
Tel.: +48 783 423 453 France Neuraxpharm France Tél: +33 1.53.63.42.90 Portugal Neuraxpharm Portugal, Unipessoal Lda Tel: +351 910 259 536 Hrvatska Neuraxpharm Pharmaceuticals, S.L.
Tel: +34 93 602 24 21 România Neuraxpharm Pharmaceuticals, S.L.
Tel: +34 93 475 96 00 Ireland Neuraxpharm Ireland Ltd.
Tel: +353 1 428 7777 Slovenija Neuraxpharm Pharmaceuticals, S.L.
Tel: +34 93 475 96 00 Ísland Neuraxpharm Sweden AB Sími: +46 (0)8 30 91 41 (Svíþjóð) Slovenská republika Neuraxpharm Slovakia a.s.
Tel: +421 255 425 562 31 Italia Neuraxpharm Italy S.p.A.
Tel: +39 0736 980619 Suomi/Finland Neuraxpharm Sweden AB Puh/Tel: +46 (0)8 30 91 41 (Ruotsi /Sverige) Κύπρος Brain Therapeutics ΙΚΕ Τηλ: +302109931458 Sverige Neuraxpharm Sweden AB Tel: +46 (0)8 30 91 41 Latvija Neuraxpharm Pharmaceuticals, S.L.
Tel: +34 93 475 96 00
This 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.
📄 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 Nezglyal (PDF, ec.europa.eu).
You can check this medicine at any time in the Union Register of medicinal products (EU number EU/1/26/2060).
⚠️ 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.