Neuraxpharm and Minoryx Therapeutics have received European Commission marketing authorisation for Nezglyal (leriglitazone), an oral treatment for male patients aged two to 12 with cerebral adrenoleukodystrophy (cALD) and non-gadolinium-enhancing brain lesions.
The approval, granted under exceptional circumstances on 21 September, follows a positive opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) in July.
It is based on data from the Phase II/III NEXUS study and additional real-world evidence from compassionate-use programmes.
Leriglitazone is an orally bioavailable, brain-penetrating selective peroxisome proliferator-activated receptor gamma (PPARγ) agonist.
EMA describes the medicine as an oral suspension containing 13.66 mg/mL of the active substance.
The drug crosses the blood-brain barrier and is designed to modulate pathways associated with neuroinflammation, demyelination, oxidative stress and mitochondrial dysfunction.
The authorisation covers all 27 EU Member States, as well as Norway, Iceland and Liechtenstein.
Neuraxpharm plans to launch the product first in Germany by the end of 2026, with further European launches dependent on national reimbursement negotiations.
Implications for CNS drug development
The approval marks another milestone in Neuraxpharm’s strategy to expand its CNS portfolio through partnerships and specialist medicines.
Last year, the company acquired the Provigil and Nuvigil narcolepsy brands and entered a global co-development agreement with Dizlin Pharmaceuticals for the Parkinson’s drug-device therapy Infudopa SubC.
For Minoryx, Nezglyal provides a potential platform for expanding leriglitazone into additional CNS indications.
The companies completed enrolment in the Phase III CALYX trial this month, which is evaluating the drug in adult male cALD patients with gadolinium-enhancing lesions.
Top-line results are expected in early 2028 and could support a future expansion of the European indication and a US regulatory submission.
The companies are also continuing development of leriglitazone in Rett syndrome through the Phase IIa TREE3 study, with results expected by the end of this year.
For manufacturers and developers, the approval highlights the role of specialist commercial partnerships in bringing therapies for small patient populations through late-stage development and into European markets, while continued clinical development could broaden the manufacturing and supply requirements for leriglitazone with time.
