Neuraxpharm and Minoryx have announced that the European Commission has granted marketing authorisation under exceptional circumstances for Nezglyal as a treatment for male cerebral adrenoleukodystrophy aged 2–12 years with Gd‑negative brain lesions.
It is the first pharmacological therapy approved in the EU for this aggressive neurodegenerative disease.
“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 of CRMR LeukoFrance, Hôpital du Kremlin Bicêtre, France.
She added: “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.
He explained: “Today’s announcement reinforces our commitment at Neuraxpharm to advancing innovative medicines that target CNS diseases with significant unmet clinical need.”
“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.
He concluded: “Our development efforts continue as we generate more data towards expanding the label within X-ALD and other orphan indications.”
cALD is characterised by demyelinating brain lesions that progress rapidly, leading to neurological decline and death within three to four years. It is the most aggressive form of X‑linked adrenoleukodystrophy, which has an incidence of 6–8 per 100,000 live births.
Nezglyal is an oral, once‑daily selective PPAR gamma agonist offering a non‑invasive treatment option for Gd‑negative children. The approval applies across all 27 EU Member States plus Norway, Iceland and Liechtenstein.
Neuraxpharm will commercialise the therapy in Europe, with the first launch expected in Germany by the end of 2026. Further launches will follow national reimbursement negotiations.
The development programme continues with the CALYX2 phase 3 trial in adult male cALD patients and the TREE3 phase 2a trial in paediatric Rett syndrome. Read-outs are expected in early 2028 and by the end of 2026.
