Catalan biotechnology company Minoryx Therapeutics, based at the TecnoCampus in Mataró, along with pharmaceutical group Neuraxpharm, have jointly announced the European approval of NEZGLYAL® (leriglitazone). This drug is the first pharmacological treatment authorized for cerebral adrenoleukodystrophy (cALD), a rare and very serious neurodegenerative disease.
cALD is an aggressive form of X-linked adrenoleukodystrophy (X-ALD) that causes progressive brain lesions and severe neurological deterioration, primarily affecting children between 2 and 12 years old. The approval was based on the results of the NEXUS clinical study and data from compassionate use programs.
Leriglitazone is indicated for male children aged 2 to 12 years with cALD presenting initial brain lesions without gadolinium uptake (Gd negative), meaning in early stages. Until now, the only therapeutic option was hematopoietic stem cell transplantation, an invasive procedure.
This new drug offers a less invasive therapeutic alternative with the potential to slow the disease's progression. The first European launch is expected to take place in Germany before the end of 2026, followed by other European countries.
According to the licensing agreement between Minoryx Therapeutics and Neuraxpharm, the latter will be responsible for marketing the drug in Europe. Clinical development will continue with additional trials for patients with more advanced cALD and Rett syndrome.
