Omaveloxolone (SKYCLARYS®): A Comprehensive Pharmacological and Clinical Monograph

I. Introduction and Drug Profile

Overview and Significance

Omaveloxolone, marketed under the brand name SKYCLARYS®, represents a landmark therapeutic advancement in the field of neurodegenerative diseases. It is the first and only medication approved by major global regulatory bodies, including the U.S. Food and Drug Administration (FDA) and the European Commission (EC), for the treatment of Friedreich's ataxia (FA).[1] This approval is indicated for adults and adolescents aged 16 years and older who are living with this rare, inherited, and relentlessly progressive condition.[3] FA is characterized by debilitating neurological and systemic symptoms, including ataxia, muscle weakness, loss of coordination, cardiomyopathy, and a significantly shortened lifespan, for which no disease-modifying therapies previously existed.[4] The advent of Omaveloxolone signals a paradigm shift, moving the management of FA beyond purely supportive and symptomatic care to an era of active pharmacological intervention designed to slow the underlying progression of the disease.[7] Its approval is a culmination of years of collaborative effort between patients, researchers, advocacy organizations, and industry, offering tangible hope to a community that has long faced a profound unmet medical need.[7]

Chemical Classification and Origin