What the approval covers
The European Commission has granted marketing authorisation under exceptional circumstances for NEZGLYAL (leriglitazone) as a treatment for cerebral adrenoleukodystrophy (cALD), a rare neurodegenerative disease. It is the first pharmacological treatment approved for the condition in the European Union.
The indication covers male cALD patients aged 2 to 12 years with Gadolinium-negative brain lesions and a neurological functional score of 0 or 1, meaning the disease is caught at an early stage. The approval is valid in all 27 EU member states, plus Norway, Iceland and Liechtenstein.
A daily pill instead of a transplant
Leriglitazone is an orally bioavailable, brain-penetrant selective PPAR gamma agonist, taken once a day. Until now, treatment options were extremely limited. Haematopoietic stem cell transplantation can halt progression in selected patients, but it is an invasive procedure that is not suitable or available for everyone.
The European Commission based its decision on results from the Phase 2/3 NEXUS1 study and additional real-world evidence from compassionate use programmes.
"This approval recognises years of research and collaboration between clinicians and patient organisations," said Marc Martinell, chief executive of Minoryx Therapeutics, the Barcelona company that developed the drug. He added that the company is generating more data with a view to expanding the label within X-linked adrenoleukodystrophy and other rare indications.
Launch and reimbursement
Minoryx and Neuraxpharm signed a licence agreement under which Neuraxpharm will commercialise the product in Europe. The first European launch is expected in Germany by the end of the year, with further launches to follow as national reimbursement negotiations are completed.