Leriglitazone gets positive EU opinion for rare neurodegenerative disease

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended granting marketing authorisation approval under exceptional circumstances for Nezglyal (leriglitazone) as a treatment for male cALD patients, aged two to 12 years with gadolinium (Gd) negative brain lesions.  

European Commission approval is expected by the end of September 2026. 

The opinion is based on results from the Phase II/III NEXUS1 study and additional real-world evidence from compassionate use programmes.  

Cerebral adrenoleukodystrophy (cALD) is characterised by demyelinating brain lesions that can progress rapidly, leading to acute neurological decline and death in three to four years. There are currently no approved pharmacological treatments for cALD in the EU. 

“The positive CHMP opinion is a regulatory validation, and we are very pleased that we will soon be able to provide a new therapeutic option to boys suffering from cALD. I would like to take the opportunity to thank physicians, patients, their families and the wider ALD community, we are enormously grateful for the support we received throughout these years,” said Marc Martinell, CEO, Minoryx. “The journey continues towards US approval and future European label-expansion, as we generate new data in adult male cALD patients with gadolinium enhancing lesions from the ongoing CALYX 2 trial.” 

Minoryx and Neuraxpharm entered into a license agreement under which Neuraxpharm will commercialise the product in Europe following marketing authorisation. 

The development programme continues, including the ongoing CALYX Phase III trial in adult male cALD patients with gadolinium enhancing lesions and the TREE Phase IIa trial in paediatric patients with Rett syndrome, with read-outs expected by early 2028 and end of 2026 respectively. 

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