EMA Recommends Approval for Nezglyal, First Pharmacological Treatment for Pediatric Cerebral Adrenoleukodystrophy in EU

The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending marketing authorization under exceptional circumstances for Nezglyal (leriglitazone). This treatment is intended for male pediatric patients aged 2-12 years with cerebral adrenoleukodystrophy (cALD) who have Gadolinium-negative brain lesions. If approved by the European Commission, Nezglyal would be the first pharmacological treatment available for cALD in the EU, a rare and debilitating genetic disorder.