On 21 September 2026, the European Commission (EC) granted marketing authorisation for NEZGLYAL® (leriglitazone). under exceptional circumstances for the medicinal product NEZGLYAL®, indicated for the treatment of Cerebral Adrenoleukodystrophy (cALD), in males with Adrenoleukodystrophy (ALD) aged 2 to 12 years with non-Gadolinium (Gd)-enhancing lesions (i.e. Gd-negative) in brain Magnetic Resonance Imaging (MRI), with a Neurological Functional Score (NFS) of 0 or 1.

Leriglitazone is a novel, selective, differentiated PPAR gamma agonist, which has shown preclinical proof-of-concept in animal models of multiple CNS diseases by modulating pathways leading to neuroinflammation, demyelination, mitochondrial dysfunction, oxidative stress, and axonal degeneration.

It has shown positive cALD outcomes in both paediatric cALD patients in the NEXUS clinical trial and adult AMN patients in the ADVANCE trial. Results from NEXUS demonstrate that paediatric cALD patients are clinically and radiologically stable after over 96 weeks of treatment or at a visit prior to Haematopoietic Stem Cell Transplantation (HSCT). Data from ADVANCE showed that leriglitazone reduced cALD progression in AMN patients. In the US, an agreement was reached with the FDA regarding a pivotal study in adult male cALD patients with Gd-positive brain lesions (CALYX). The study is currently on-going, and the first readout will be in 1Q 2028. Leriglitazone offers a strong potential for indication expansion into other CNS diseases. In ADVANCE leriglitazone showed clinically relevant impact on AMN endpoints and a proof-of-concept study in Friedreich’s Ataxia (FRDA) showed clinical benefit in this population. Finally, by the end of 2026 we will obtain read-out from our phase 2 trial (TREE) in Rett syndrome.