EMA Validates Minoryx’s Marketing Application for Leriglitazone to Treat cALD
Minoryx Therapeutics, a clinical-stage biotech company focused on orphan central nervous system (CNS) diseases, has announced that the European Medicines Agency (EMA) has validated its Marketing Authorization Application (MAA) for leriglitazone (NEZGLYAL®). This milestone marks an important regulatory step toward approval of a first-in-class oral therapy for cerebral adrenoleukodystrophy (cALD) in pediatric and adult male patients.
cALD is a rare and devastating neurodegenerative disorder affecting males with X-linked adrenoleukodystrophy (X-ALD). It leads to inflammatory demyelination in the brain and is often rapidly progressive and fatal if untreated. Current treatment options are extremely limited, with hematopoietic stem cell transplantation (HSCT) being the only disease(modifying option), and only for a subset of patients.
The MAA is supported by two pivotal clinical trials:
- ADVANCE, a placebo-controlled, double-blind Phase 2/3 trial involving 116 adult male patients with early-stage cALD. The trial demonstrated that leriglitazone significantly slowed lesion progression, reduced the risk of disease conversion, and showed favorable safety and tolerability.
- NEXUS, an open-label, 96-week study in pediatric boys with early cALD, showed that a majority of patients remained clinically and radiologically stable, with approximately 35% achieving disease arrest, a marked improvement over what is typically seen in untreated patients.
Leriglitazone is a PPAR gamma agonist that crosses the blood-brain barrier and modulates pathways involved in neuroinflammation, mitochondrial function, and oxidative stress, all critical in the pathology of cALD.
Minoryx is collaborating with Neuraxpharm, its European commercial partner, to ensure access to this therapy across the EU if approved. They are also in discussions with regulatory authorities in other regions.
If approved, leriglitazone would be the first pharmacological treatment available for cALD, addressing a major unmet medical need in this patient population.
🔗 Read the full press release from Minoryx here
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