Europe Approved Leriglitazone for Boys With cALD Two Years After the EMA Recommended Refusal

The European Commission granted marketing authorisation under exceptional circumstances for Nezglyal (leriglitazone) on 25 September 2026, for males aged 2 to 12 with cerebral adrenoleukodystrophy (cALD) and brain lesions that do not enhance on gadolinium imaging. Minoryx Therapeutics and Neuraxpharm say it is the first approved drug treatment for cALD in the EU.
Leriglitazone is a once-daily oral, brain-penetrant PPAR gamma agonist. The approval rests on the Phase 2/3 NEXUS study plus real-world evidence from compassionate use. The CHMP recommended approval on 23 July 2026; EMA records show it had recommended refusal of the same product and confirmed that on re-examination in May 2024.
cALD is the aggressive cerebral form of X-linked adrenoleukodystrophy, which occurs in about 6 to 8 per 100,000 live births, and can lead to rapid neurological decline and death within three to four years.
Neuraxpharm will commercialise in Europe, starting in Germany by the end of the year, with other markets pending national reimbursement negotiations. The Phase 3 CALYX trial in adult men with gadolinium-enhancing lesions completed enrolment on 9 September and reads out in early 2028.

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A drug the EMA refused two years ago is now approved, on a narrow label and with Europe ahead of the US.

Exceptional circumstances is what changed. This route applies when complete data cannot be generated, and it comes with ongoing obligations to the EMA. Adding compassionate-use real-world evidence to NEXUS turned a confirmed refusal into an authorisation. For sponsors in ultra-rare paediatric neurology, that is the precedent to watch.
The label covers a specific window. Boys aged 2 to 12 without gadolinium-enhancing lesions are patients before active inflammation shows on imaging. Adults with enhancing lesions, a larger group with more advanced disease, wait for CALYX in early 2028.
Europe leads, and access is country by country. Minoryx says it is working towards US approval, but no FDA filing has been announced. The EU licence covers 30 countries, yet the only confirmed launch is Germany by year end; everywhere else waits on national price negotiations for a drug with a thin evidence base.