Nezglyal
Authorised
This medicine is authorised for use in the European Union
leriglitazone
MedicineHumanAuthorised
- Application under evaluation
- CHMP opinion
- European Commission decision
Overview
Nezglyal is a medicine used to treat cerebral adrenoleukodystrophy in boys from 2 to 12 years of age who have mild or no symptoms, based on a neurological functional score of 0 or 1.
It is used in patients whose brain lesions do not take up the contrast agent gadolinium (Gd-negative lesions) during MRI brain scans, indicating that the disease is in the early stages.
Cerebral adrenoleukodystrophy is a form of a genetic 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 cerebral adrenoleukodystrophy, 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.
Cerebral adrenoleukodystrophy is rare and Nezglyal was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 18 November 2016 for the treatment of adrenoleukodystrophy. Further information on the orphan designation can be found on the EMA website.
Nezglyal contains the active substance leriglitazone.
Nezglyal can only be obtained with a prescription, and treatment should be started and monitored by a doctor experienced in managing diseases in which cells of the central nervous system stop working or die.
The medicine is available as a suspension to be taken by mouth once a day. The dose is based on the child’s height and weight.
Before starting Nezglyal and during treatment, the doctor will carry out regular blood tests to check how well the patient’s liver is working. They may need to temporarily stop treatment if the tests show problems with the patient’s liver function.
For more information about using Nezglyal, see the package leaflet or contact your doctor or pharmacist.
The active substance in Nezglyal, leriglitazone, works by attaching to and activating receptors (targets) called 'PPAR gamma receptors', which are found inside cells, including nerve cells. PPAR gamma 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 is therefore expected to protect nerve cells from damage by reducing inflammation, improving the function of mitochondria and protecting against damage from free radicals.
A main study involved 23 boys from 2 to 12 years of age with cerebral adrenoleukodystrophy, including those with Gd-positive or Gd-negative brain lesions. All patients received Nezglyal in addition to their standard care and, if eligible, haematopoietic stem cell transplantation (HSCT) at any time during the study. HSCT is a procedure where the patient receives stem cells to help the bone marrow produce healthy blood cells. Nezglyal was not compared with another treatment. After 96 weeks or by the visit before HSCT, disease worsening was arrested (halted) in 35% of patients (7 out of 20 for whom sufficient data had been collected), compared with an expected disease arrest rate of 10% if the disease were left to progress naturally. Most of the patients (6 out of 7) who had disease arrest with Nezglyal had Gd-negative brain lesions at the start of treatment.
Studies carried out with Nezglyal are described in more detail in the medicine’s assessment report.
For the full list of side effects and restrictions with Nezglyal, see the package leaflet.
The most common side effects with Nezglyal (which may affect up to 1 in 10 children) include increased weight, eyelid oedema (swelling due to fluid retention), leucopenia (low levels of white blood cells) and neutropenia (low levels of neutrophils, a type of white blood cell that fights infection).
Nezglyal must not be used by people who have hypersensitivity (an allergy) to thiazolidinedione-containing medicines, which are used to treat type 2 diabetes. It must also not be used by people who have, or have previously had, heart failure.
At the time of approval, there were no authorised medicines to treat patients with Gd-negative cerebral adrenoleukodystrophy, a very rare, rapidly progressing disease. The only potential treatment option for progressive (worsening) cerebral adrenoleukodystrophy (usually with Gd-positive lesions) is HSCT.
Nezglyal has been shown to be effective at arresting disease progression in children with cerebral adrenoleukodystrophy who have Gd-negative lesions, an early disease stage in which treatment is expected to offer the greatest long-term benefit. In terms of safety, the side effects are considered manageable.
In reaching its decision, the European Medicines Agency also considered input from patient organisations and healthcare professional organisations.
The Agency decided that Nezglyal’s benefits are greater than its risks and that it can be authorised for use in the EU.
Nezglyal has been authorised under exceptional circumstances. This is because there is a high unmet medical need for the treatment of cerebral adrenoleukodystrophy and it has not been possible to obtain complete information about Nezglyal due to the rarity of the disease. The company must provide further data on Nezglyal. It must submit the results of a study looking at the safety of the medicine as well as the results of a registry-based study on the long-term effectiveness of Nezglyal. The company must also provide yearly updates on any new information about the safety and effectiveness of the medicine. Every year, the Agency will review any new information that becomes available.
Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Nezglyal have been included in the summary of product characteristics and the package leaflet.
As for all medicines, data on the use of Nezglyal are continuously monitored. Suspected side effects reported with Nezglyal are carefully evaluated and any necessary action is taken to protect patients.
Nezglyal received a marketing authorisation under exceptional circumstances valid throughout the EU on 21 September 2026.
Product information
This medicine’s product information is available in all official EU languages.
Select 'available languages' to access the language you need.
Product information documents contain:
- summary of product characteristics (annex I);
- manufacturing authorisation holder responsible for batch release (annex IIA);
- conditions of the marketing authorisation (annex IIB);
- labelling (annex IIIA);
- package leaflet (annex IIIB).
Product details
- Name of medicine
- Nezglyal
- Active substance
- leriglitazone
- International non-proprietary name (INN) or common name
- leriglitazone
- Therapeutic area (MeSH)
- Adrenoleukodystrophy
- Anatomical therapeutic chemical (ATC) code
- A16AX23
Pharmacotherapeutic group
Other alimentary tract and metabolism productsTherapeutic indication
Nezglyal is indicated for the treatment of Cerebral Adrenoleukodystrophy (cALD) in males with Adrenoleukodystrophy (ALD) aged 2 to 12 years with non Gadolinium (Gd) enhancing lesions (i.e. Gd negative) in brain Magnetic Resonance Imaging (MRI), with a Neurological Functional Score (NFS) of 0 or 1.
News on Nezglyal
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