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).
Minoryx Therapeutics and Neuraxpharm Group’s Nezglyal is a treatment for male cerebral adrenoleukodystrophy (cALD) patients aged 2-12 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 2/3 NEXUS1 study and additional real-world evidence from compassionate use programmes. Cerebral adrenoleukodystrophy (cALD) is a debilitating disease 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.
Marc Martinell, CEO of Minoryxm said: “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. 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.”
Jörg Thomas Dierks, CEO of Neuraxpharm, said: “cALD is a severe neurodegenerative disease and we look forward to bringing Nezglyal, a long-awaited treatment, to European patients following EC approval later this year.”
Minoryx and Neuraxpharm entered into a licence agreement under which Neuraxpharm will commercialise the product in Europe following marketing authorisation.
The development programme continues, including the ongoing CALYX phase 3 trial in adult male cALD patients with Gadolinium enhancing lesions and the TREE3 phase 2a trial in paediatric patients with Rett syndrome, with read-outs expected by early 2028 and the end of 2026 respectively.