Leriglitazone Marketing Authorization Application submitted for treatment of cerebral adrenoleukodystrophy has been validated by European Medicines Agency

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Leriglitazone Marketing Authorization Application submitted for treatment of cerebral adrenoleukodystrophy has been validated by European Medicines Agency

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Barcelona, Spain and Düsseldorf, Germany – 23 July 2025 – Minoryx Therapeutics, a late-stage biotech company focused on the development of therapies for orphan central nervous system (CNS) disorders and Neuraxpharm Group (Neuraxpharm), a leading European specialty pharmaceutical company focused on the treatment of CNS disorders, today announce that the Marketing Authorization Application (MAA) for Minoryx’s lead candidate leriglitazone (NEZGLYAL®) has been submitted to the European Medicines Agency (EMA) for the treatment of paediatric and adult male patients with cerebral adrenoleukodystrophy (cALD). EMA has now validated the MAA file and it is under review by the Committee for Medicinal Products for Human Use (CHMP).

This new MAA is based on recent data from the successfully concluded NEXUS study, a 96-week, pivotal, open-label study designed to evaluate the efficacy and safety of once-daily oral dosing of leriglitazone in paediatric patients with cALD. The study met the primary endpoint and all 20 evaluable patients remained clinically stable whilst on treatment and 7 out of 20 evaluable patients (35%) met the arrested disease criteria, which is significantly greater than the 10% self-arrest rate that would be expected from natural history (p<0.05). Leriglitazone was well tolerated with no discontinuations due to adverse events.

The submission is further supported by data from the previously concluded ADVANCE study, a 116 patient, double-blind, placebo-controlled study conducted in Europe and the United States in which leriglitazone reduced the incidence and progression of cerebral lesions in adults. Furthermore, data from the ongoing extensive compassionate use program in adult and paediatric patients with cALD have also been submitted. Leriglitazone was generally well tolerated in the ADVANCE study and compassionate use programme.

“We believe the new and positive data from Nexus provide compelling evidence for the efficacy of leriglitazone giving hope to patients and their families that a new treatment will be available in the near future,”said Marc Martinell, CEO, Minoryx“Leriglitazone, if approved, would be the only pharmacological treatment for patients suffering from this devastating orphan disease with a major unmet medical need.”

“We are very pleased that the clinical development programme and compassionate use data have shown the efficacy of leriglitazone across all age ranges,”said Arun Mistry, Chief Medical Officer, Minoryx. “We will continue to work closely with the EMA through the evaluation process to maximize the chance for this therapy to reach all patients in desperate need.”

Dr. Jörg-Thomas Dierks, CEO, Neuraxpharm said; “The EMA’s validation of the leriglitazone MAA submission is a key step forward as we look to bring this much needed treatment to patients. Given the terrible impact of this devastating disease and the lack of effective options, we remain hopeful of a positive outcome from the CHMP.”

The development of leriglitazone continues with the ongoing CALYX trial in adult patients with cALD and the TREE study in paediatric Rett patients. Both studies are open to recruitment.