cerebral adrenoleukodystrophy

The European Commission has granted marketing authorization for NEZGLYAL (leriglitazone), making it the first pharmacological treatment approved for cerebral adrenoleukodystrophy, a rare and rapidly progressive neurodegenerative disease that primarily affects young boys. Until now, care options for cALD were limited mainly to stem cell transplantation in eligible patients. The approval is being highlighted as a significant milestone for families affected by the rare disease and for rare-disease drug development in Europe.

The European Commission has granted marketing authorisation for NEZGLYAL, the first pharmacological treatment for childhood cerebral adrenoleukodystrophy (cALD), a rare and severe genetic brain disorder. Until now, treatment options were largely limited to stem cell transplantation and supportive care. The approval offers a new therapy option for patients across the EU and is being noted in rare disease and biotech circles.