EMA validates Minoryx' filing for lead candidate leriglitazone

Spanish CNS specialist Minoryx Therapeutics said today it has filed a Marketing Authorization Application (MAA) for its lead candidate leriglitazone to the European Medicines Agency (EMA) for the treatment of adult male patients with X-linked adrenoleukodystrophy (X-ALD).

The EMA has now validated the MAA file and it is under review by the Committee for Medicinal Products for Human Use (CHMP).

Leriglitazone is a novel, brain penetrant and selective PPAR gamma agonist. The MAA is based on data from  Minoryx’s pivotal ADVANCE study, a double-blind, placebo-controlled study conducted in Europe and the USA in 116 patients. ADVANCE is the first large and definitive study to target adult male patients with X-ALD. In ADVANCE, leriglitazone reduced the progression of cerebral lesions and incidence of cALD, as well as the progression of myelopathy symptoms, such as balance deterioration. These data are supported by the ongoing open label extension of ADVANCE and NEXUS, an open-label registration trial assessing leriglitazone in boys with cALD.