Minoryx Therapeutics has submitted the marketing authorisation application (MAA) for its drug leriglitazone to the European Medicines Agency (EMA) for the treatment of adult males with X-linked adrenoleukodystrophy (X-ALD). The EMA has already validated the application dossier and it is now in the process of being reviewed by the Committee for Medicinal Products for Human Use (CHMP).

X-ALD is a rare hereditary neurodegenerative disease. The greatest risk for patients with X-ALD, both paediatric and adult, is the development of the acute cerebral form, cALD. Currently, there are no pharmacological treatments available for patients with X-ALD.

Leriglitazone is a new drug from Minoryx that is able to cross the blood-brain barrier and has shown significant potential for treating X-ALD and other central nervous system disorders. The marketing application is based on data from the ADVANCE clinical trial, so far the largest international study ever conducted in adult patients with X-ALD. In this study, leriglitazone has shown a reduction in the progression of brain damage and the incidence of cALD, as well as a slower progression of loss of balance, one of the characteristic symptoms of AMN. These data are supported by NEXUS, a second Minoryx study, currently underway, which evaluates the use of leriglitazone in children with cALD.

"The recent funding and submission of the MAA are two important milestones in Minoryx's preparations for the approval and marketing of leriglitazone for adult X-ALD patients in the European Union," says Dr. Marc Martinell, CEO of Minoryx. If approved, leriglitazone would be the first therapeutic option for these patients suffering from this devastating minority disease. During the EMA review of the MAA, we will continue to investigate the benefits of leriglitazone in other X-ALD patient populations," explains Martinell.

Minoryx is currently in discussions with the Food and Drug Administration (FDA) to define the steps necessary to achieve approval of leriglitazone in the United States. Leriglitazone has received the designation of gold standard for the treatment of X-ALD from both the EMA and the FDA. It has also been granted Rare Paediatric Disease Designation and Fast Track Designation by the FDA.