Nezglyal

Opinion

EMA has issued an opinion on this medicine

leriglitazone

Overview

On 23 July 2026 the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the granting of a marketing authorisation under exceptional circumstances for the medicinal product Nezglyal, intended for the treatment of cerebral adrenoleukodystrophy (cALD).

The applicant for this medicinal product is Minoryx Therapeutics S.L.

Nezglyal will be available as oral suspension (13.66 mg/mL). The active substance of Nezglyal is leriglitazone (ATC code: A16AX23). Leriglitazone is an active metabolite of pioglitazone, and a selective peroxisome proliferator-activated receptor gamma (PPARγ) agonist that crosses the blood-brain barrier (BBB). Lerigltazone has been reported to have an antioxidant and neuroprotective activity in the central nervous system by modulating critical pathways involved in cALD progression and consequently decreasing neuroinflammation, enhancing BBB integrity, promoting myelination and improving mitochondrial function.

The benefits of Nezglyal were shown in an open-label study which measured the effects of leriglitazoneon disease progression prior to haematopoietic stem cell transplantation (HSCT), in boys aged 2 to 12 years old with cALD. 

Of the 20 boys who could be evaluated, seven (35%) showed no meaningful worsening of their condition (clinically and radiologically arrested disease) after receiving treatment for up to 96 weeks (or until they underwent transplantation). Of the nine boys who presented with gadolinium-negative cerebral lesions at baseline, six showed stabilisation (6/9 [66.7%]), while of the 11 boys who presented with gadolinium-positive cerebral lesions, one showed stabilisation (1/11 [9.1%.]).

This proportion was higher than what would normally be expected if the disease was left to progress naturally.

The most common side effects in children (2 to 12 years of age) were weight increase, eyelid oedema, leukopenia and neutropenia.  

The full indication is:

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.

Nezglyal should be initiated and monitored by a physician with experience in the management of neurodegenerative diseases.

Detailed recommendations for the use of this product will be described in the summary of product characteristics (SmPC), which will be published on the EMA website in all official European Union languages after the marketing authorisation has been granted by the European Commission.

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Notes:

• In exceptional circumstances, an authorisation may be granted subject to certain specific obligations, to be reviewed annually. This happens when the applicant can show that they are unable to provide comprehensive data on the efficacy and safety of the medicinal product, due to the rarity of the condition it is intended for, limited scientific knowledge in the area concerned, 
• This product was designated as an orphan medicine during its development. EMA will now review the information available to date to determine if the orphan designation can be maintained

CHMP summary of positive opinion for Nezglyal

Adopted Reference Number: EMADOC-1829012207-57936

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
• EMA product number:
  EMEA/H/C/006693
• Orphan:

  This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

• Marketing authorisation applicant:
  Minoryx Therapeutics S.L.
• Opinion adopted:
  23/07/2026
• Opinion status:
  Positive