Minoryx Therapeutics granted FDA Orphan Drug Designation in Friedreich’s Ataxia for new drug leriglitazone Minoryx Therapeutics is a company that specializes in the development of innovative treatments for rare diseases of the central nervous system (CNS), the part of the nervous system that contains the brain and spinal cord. It recently announced that its lead drug candidate, leriglitazone (MIN-102), has been given an Orphan Drug Designation for Friedreich’s Ataxia by the US Food and Drug Administration (FDA); a status given to medicines developed for rare conditions (for a full definition see here). Leriglitazone is in the late stages of being tested in humans as a treatment for Friedreich’s Ataxia and another neuro-degenerative disorder called adrenomyeloneuropathy or ‘AMN’. The potential for leriglitazone to alter the course of these disorders has been demonstrated in multiple animal and cell models, along with its unique method of action. It has been shown to have an anti-oxidant, anti-inflammatory and neuroprotective effect, and to improve mitochondrial function and delay the progression of neurological disability. Marc Martinell, CEO of Minoryx, said: “Orphan Drug Designation by the FDA for Friedreich’s Ataxia is yet another important milestone for the company. It is recognition of the disease-modifying potential of leriglitazone and of our commitment to changing the lives of patients suffering from severe orphan diseases with high unmet medical needs.” The full press release from Minoryx can be viewed here.