Approval is based on NEXUS data and follows positive CHMP opinion in July; first European launch is expected by end of 2026
Barcelona, Spain and Düsseldorf, Germany – 25 September, 2026 – Neuraxpharm Group and Minoryx Therapeutics, today announce that the European Commission (EC) has granted marketing authorisation under exceptional circumstances for NEZGLYAL® (leriglitazone), an orally bioavailable, brain penetrating, selective PPAR gamma agonist, as a treatment for male cALD patients, aged 2-12 years with Gadolinium (Gd)-negative brain lesions.
The therapy is the first approved pharmacological treatment for cALD in the European Union (EU). The approval is based on results from the Phase 2/3 NEXUS1 study and additional real-world evidence from compassionate use programmes.
The first European launch is expected in Germany by the end of the year, with additional launches in Europe anticipated pending completion of national reimbursement negotiations. Neuraxpharm is also evaluating appropriate access pathways for eligible patients.
“With childhood cALD, neurodegeneration is irreversible, so it is critical to halt disease progression early, ideally, before symptoms surface and signs of neuroinflammation appear. Until now, there were no pharmacological treatment options for early intervention. Invasive procedures, such as hematopoietic stem cell transplantation, are available for more progressed patients. However, they are donor-dependent and can only be applied within a very narrow time window,” said Dr. Caroline Sevin, MD, PhD, of CRMR LeukoFrance, Hôpital du Kremlin Bicêtre, France: “That we now have a pharmacological treatment for early intervention is a major advance in our treatment of cALD.”
“cALD is a rapidly progressing neurodegenerative disease which severely impacts the lives of patients and their families, underlining the critical need for treatments which can halt or slow disease progression and improve quality of life,” said Dr. Jörg Thomas Dierks, CEO of Neuraxpharm. “Today’s announcement reinforces our commitment at Neuraxpharm to advancing innovative medicines that target CNS diseases with significant unmet clinical need. We’re looking forward to working with the local authorities, and the medical and patient communities to bring NEZGLYAL® to eligible individuals across the region as quickly as possible.”
“This approval represents a significant milestone for the cALD community and recognises years of breakthrough research and collaboration between clinicians and patient organisations. We are very grateful for their continued support,” said Marc Martinell, CEO of Minoryx. “Our development efforts continue as we generate more data towards expanding the label within X-ALD and other orphan indications.”
Cerebral adrenoleukodystrophy (cALD) is a debilitating neurodegenerative disease characterised by demyelinating brain lesions that can progress rapidly, leading to acute neurological decline and death in three to four years. It predominantly affects the brain and is an aggressive form of X-linked adrenoleukodystrophy (X-ALD), which has an incidence of approximately 6-8/100,000 live births.
NEZGLYAL® (leriglitazone) is an oral medicine taken daily and offers a non-invasive yet disease-modifying cALD treatment option for Gd-negative children.
The EC approval is valid across all 27 European Union (EU) Member States, as well as Norway, Iceland and Liechtenstein.
Minoryx and Neuraxpharm entered into a license agreement under which Neuraxpharm will commercialise the product in Europe following marketing authorisation.
The development programme continues, with enrolment now completed in the CALYX2 Phase 3 trial in adult male cALD patients with Gd-enhancing lesions, and the ongoing TREE3 Phase 2a trial in paediatric patients with Rett syndrome. Read-outs are expected in early 2028 and by the end of 2026, respectively.
About the European Commission (EC) approval
On 21 September 2026, the European Commission (EC) granted marketing authorisation for NEZGLYAL® (leriglitazone). On 23 July 2026 the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the granting of a marketing authorisation under exceptional circumstances for the medicinal product NEZGLYAL®, indicated for the treatment of Cerebral Adrenoleukodystrophy (cALD), in males with Adrenoleukodystrophy (ALD) aged 2 to 12 years with non-Gadolinium (Gd)-enhancing lesions (i.e. Gd-negative) in brain Magnetic Resonance Imaging (MRI), with a Neurological Functional Score (NFS) of 0 or 1.
For more information, please visit Nezglyal | European Medicines Agency (EMA)
For further information please contact:
Optimum Strategic Communications
Nick Bastin / Charlotte Hepburne-Scott / Nellie Stephens / Ben Cowe
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Forward-Looking Statements
This press release contains forward-looking statements that involve risks and uncertainties that could cause actual results to differ materially.
References
1NEXUS, a phase 2/3, open-label clinical study designed to assess the efficacy and safety of leriglitazone in male paediatric patients with early stage cALD.
https://www.clinicaltrialsregister.eu/ctr-search/search?query=2019-000654-59
2CALYX, a phase 3, multicentre, randomised (1:1), double-blind, placebo-controlled, clinical study, designed to assess the efficacy and safety of leriglitazone in male adult cALD patients with Gd-positive lesions.
https://clinicaltrials.gov/study/NCT05819866)
3TREE, a phase 2a, randomised (1:1), double-blind, placebo-controlled, clinical study, designed to evaluate the safety (and efficacy) of leriglitazone in paediatric female patients with Rett syndrome.
https://euclinicaltrials.eu/ctis-public/view/2024-514684-26-00?lang=en
4ADVANCE, a pivotal phase 2/3 randomised, double-blind, placebo-controlled, clinical study with an open-label extension, was designed to assess the efficacy and safety of leriglitazone in male patients with AMN with or without cALD.
https://www.thelancet.com/journals/laneur/article/PIIS1474-4422(22)00495-1/abstract