The EMA’s CHMP has recommended approval of NEZGLYAL for boys aged 2-12 with cerebral adrenoleukodystrophy, a rare neurodegenerative disease with no approved drug treatment in the EU.

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorisation for NEZGLYAL (leriglitazone) as a treatment for male patients aged 2-12 with cerebral adrenoleukodystrophy (cALD) and Gadolinium-negative brain lesions.

The recommendation, issued under exceptional circumstances, is based on data from the Phase 2/3 NEXUS1 study and additional real-world evidence from compassionate use programmes.

The European Commission is expected to make a final decision on marketing authorisation by the end of September 2026.

Cerebral adrenoleukodystrophy is a rare neurodegenerative disease characterised by demyelinating lesions in the brain. The condition can progress rapidly, causing neurological decline and death within three to four years.

There are currently no approved pharmacological treatments for cALD in the European Union.

Marc Martinell, chief executive officer of Minoryx Therapeutics, 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 positive opinion represents a regulatory milestone for Minoryx and Neuraxpharm Group, which entered into a licensing agreement under which Neuraxpharm will commercialise NEZGLYAL in Europe if marketing authorisation is granted.

The companies said the CHMP recommendation follows results from the NEXUS1 study, which evaluated leriglitazone in boys with cALD, alongside evidence collected through compassionate use programmes.

Martinell added: “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.”

The development programme for leriglitazone is continuing in other patient populations.

The ongoing CALYX Phase 3 trial is evaluating the treatment in adult male patients with cALD and Gadolinium-enhancing brain lesions. The companies expect data from the study in 2028.

Leriglitazone is also being investigated in the TREE3 Phase 2a trial in paediatric patients with Rett syndrome, with data expected by the end of 2026.

Jörg Thomas Dierks, chief executive officer 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.”

If the European Commission follows the CHMP recommendation, NEZGLYAL would provide a new pharmacological treatment option for eligible boys with cALD in the European Union.

The potential authorisation would mark a significant regulatory development for the treatment of the rare disease, which can cause rapid neurological deterioration and has limited treatment options.

Minoryx and Neuraxpharm said they will continue to progress the development programme for leriglitazone, including studies intended to expand its potential use in other cALD patient populations.