Leriglitazone is a PPAR-gamma agonist and metabolite of the glitazone pioglitazone. It 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 cerebral adrenoleukodystrophy progression and consequently decreasing neuroinflammation, enhancing BBB integrity, promoting myelination and improving mitochondrial function.
The most common side effects in children were weight increase, eyelid edema, leukopenia, and neutropenia.
History The benefits of leriglitazone were shown in an open-label study which measured the effects of leriglitazoneon disease progression prior to hematopoietic stem cell transplantation (HSCT), in boys aged 2 to 12 years old with cerebral adrenoleukodystrophy.
Society and culture Legal status In January 2024, the European Medicines Agency (EMA) recommended the refusal of the marketing authorization for leriglitazone (Nezglyal) requested by Minoryx Therapeutics S.L. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged. In May 2024, the EMA confirmed its recommendation to refuse marketing authorization for leriglitazone.
In July 2026, the Committee for Medicinal Products for Human Use of the EMA adopted a positive opinion recommending the granting of a marketing authorization under exceptional circumstances for the medicinal product Nezglyal, intended for the treatment of cerebral adrenoleukodystrophy. The applicant for this medicinal product is Minoryx Therapeutics.
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