Barcelona, Spain and Düsseldorf, Germany – July 24, 2026
Minoryx Therapeutics and Neuraxpharm Group have received a positive opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recommending marketing authorization under exceptional circumstances for NEZGLYAL® (leriglitazone) to treat male patients aged 2–12 years with cerebral adrenoleukodystrophy (cALD) and Gadolinium (Gd)-negative brain lesions. The recommendation is based on positive findings from the Phase 2/3 NEXUS study and supporting real-world evidence from compassionate use programs. If approved by the European Commission, expected by the end of September 2026, NEZGLYAL® will become the first approved pharmacological treatment for cALD in the European Union, addressing a significant unmet medical need for this rare and rapidly progressive neurodegenerative disorder.
CHMP Opinion Marks Major Milestone for Rare Disease Treatment
Cerebral adrenoleukodystrophy (cALD) is a rare, inherited neurodegenerative disease characterized by progressive demyelinating brain lesions that can rapidly lead to severe neurological deterioration and death within three to four years if left untreated. The CHMP recommendation validates the clinical benefits demonstrated in the NEXUS Phase 2/3 trial, supported by additional evidence collected through compassionate use programs. Minoryx Therapeutics CEO Marc Martinell described the positive opinion as an important regulatory milestone that will soon provide a long-awaited treatment option for young boys affected by cALD. He also highlighted the company’s continued commitment to expanding the therapy’s potential through ongoing development programs, including efforts toward U.S. regulatory approval and future European label expansion for additional patient populations.
Commercialization Plans Advance Across Europe
Following the anticipated European Commission approval, Neuraxpharm Group will commercialize NEZGLYAL® across Europe under its licensing agreement with Minoryx Therapeutics. Neuraxpharm CEO Dr. Jörg Thomas Dierks emphasized that the therapy represents a significant advancement for patients with severe central nervous system disorders and reinforces the company’s commitment to delivering innovative treatments for diseases with limited therapeutic options. The collaboration between the two companies combines Minoryx’s clinical development expertise with Neuraxpharm’s commercialization capabilities, aiming to ensure broad patient access after regulatory approval. Both organizations believe the partnership will help accelerate the availability of this novel treatment throughout European markets.
Advertisement
Ongoing Clinical Development Supports Future Expansion
The development of NEZGLYAL® continues beyond its initial cALD indication. Minoryx Therapeutics is currently conducting the Phase 3 CALYX trial in adult male cALD patients with Gadolinium-enhancing lesions, supporting potential future label expansion. In parallel, the company is evaluating leriglitazone in the Phase 2a TREE trial for pediatric Rett syndrome, with topline data expected by the end of 2026, while results from the CALYX Phase 3 study are anticipated in early 2028. These ongoing studies demonstrate the company’s long-term strategy to expand the clinical applications of leriglitazone across multiple rare neurological disorders while strengthening its position in the neurodegenerative disease treatment landscape.
Source: Minoryx Therapeutics press release
