Once in the bloodstream, leriglitazone can cross the blood-brain barrier and enter the central nervous system (CNS). It acts by binding to a protein called PPAR gamma, and in this way prevents processes associated with X-ALD, including problems with mitochondria (the power houses of cells), oxidative stress, neuroinflammation, loss of myelin (the protective coat surrounding nerve cells), and nerve cell degeneration.
The FDA’s Fast Track program aims to support the development, and speed up the review and approval of therapies for serious conditions with an unmet medical need.
“The granting of Fast Track Designation underscores the unique potential of leriglitazone to treat X-ALD, an orphan life-threatening and chronic neurodegenerative disorder for which there is currently no effective cure available,” Marc Martinell, CEO of Minoryx, said in a press release.
Currently, the efficacy and safety of leriglitazone is being evaluated in a Phase 2/3 clinical trial called ADVANCE (NCT03231878). The trial is a two-year, double-blind and placebo-controlled study that includes 116 adult male X-ALD patients with AMN, enrolled at centers across Europe and the U.S.
Its primary goal is to evaluate the effect of leriglitazone on AMN progression, which will be determined based on a motor function test after 96 weeks of treatment. Top-line results for the ADVANCE trial, due to conclude in October, are expected by year’s end.
“This FDA designation comes as we prepare to report pivotal results of the ADVANCE trial at the end of 2020, and to submit a New Drug Application in the U.S. We are working closely with the FDA to provide the first potential disease-modifying and effective therapy for patients with AMN,” María Pascual, chief regulatory officer of Minoryx, concluded.
Leriglitazone was previously designated an orphan drug by both the FDA and the European Medicines Agency (EMA) for X-ALD, a status that helps to support and accelerate the development of treatments targeting rare conditions.