For those with adrenoleukodystrophy (ALD) and adrenomyeloneuropathy (AMN), Leriglitazone has yielded positive results in clinical trials.
Now, Minoryx Therapeutics and Neuraxpharm Group have annouced that European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended granting marketing authorisation approval under exceptional circumstances for NEZGLYAL® (leriglitazone) as a treatment for male cALD patients, aged 2-12 years with Gadolinium (Gd) negative brain lesions. European Commission approval is expected by the end of September 2026.
Although we could not be involved in the EMA assessment as the UK is no longer part of the European Union, our Leriglitazone Impact Report was accepted as evidence. We have also been invited to participate in The National Institute for Health and Care Excellence (NICE) Highly Specialised Technology Appraisal.
This recommendation marks an important milestone for the ALD community and reflects years of research, advocacy and collaboration between families, clinicians, researchers and industry. While there is still work to do to ensure timely access to new treatments, the progress made with leriglitazone offers renewed hope for children and families affected by cerebral ALD. We remain committed to representing the voices of the leukodystrophy community throughout the NICE appraisal process and will continue to work towards a future where effective treatments are available to all who need them.