Neuraxpharm Receives EU Approval for Cerebral Adrenoleukodystrophy Treatment
News related to:Neuraxpharm Group · 1 min read
BARCELONA, Spain and DÜSSELDORF, Germany, Sept. 25, 2026 /CourierPR/ -- Neuraxpharm Group and Minoryx Therapeutics have announced that the European Commission has granted marketing authorisation for NEZGLYAL® (leriglitazone), the first pharmacological treatment for cerebral adrenoleukodystrophy (cALD) in the European Union. The approval, valid across all 27 EU Member States, Norway, Iceland, and Liechtenstein, is for male cALD patients aged 2 to 12 years with non-Gadolinium (Gd)-enhancing brain lesions.
The therapy, a brain-penetrant, orally bioavailable, selective PPAR gamma agonist, was approved based on results from the Phase 2/3 NEXUS1 study and additional real-world evidence from compassionate use programs. The first European launch is expected in Germany by the end of the year, with additional launches anticipated pending completion of national reimbursement negotiations. Neuraxpharm is also evaluating appropriate access pathways for eligible patients.
Dr. Caroline Sevin, MD, PhD, of CRMR LeukoFrance, Hôpital du Kremlin Bicêtre, France, highlighted the significance of this approval.
Dr. Jörg Thomas Dierks, CEO of Neuraxpharm, expressed the company's commitment to advancing innovative medicines for CNS diseases with significant unmet clinical need.
The development programme for NEZGLYAL® 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.
Minoryx, founded in 2011 and headquartered in Spain, has raised more than €150 million. The company is backed by a syndicate of experienced investors, including Columbus Venture Partners, CDTI Innvierte, Criteria BioVentures, Fund+, Ysios Capital, Roche Venture Fund, Kurma Partners, Chiesi Ventures, S.R.I.W, Idinvest Partners / Eurazeo, SFPI-FPIM, HealthEquity, and Sambrinvest. Neuraxpharm, a leading European specialty pharmaceutical company, has more than 1,000 employees and develops and commercialises CNS products through a direct presence in more than 20 countries in Europe, two in Latin America, one in the Middle East, in Australia, and globally via partners in more than 50 countries.