Skyclarys (omaveloxolone), the first and only oral medication approved in the United States and the European Union for the treatment of Friedreich's Ataxia (FA), 'is not available in Argentina.' The drug, which must be prescribed by a specialist, has been a global milestone since its FDA approval in February 2023, as it has 'demonstrated to be more effective than placebo' to 'slow the progression of the disease' in adults and adolescents from 16 years of age.
Mechanism of action and prescription requirement Skyclarys works by activating the Nrf2 pathway to reduce oxidative stress and improve mitochondrial function, a key mechanism to counteract cellular damage caused by frataxin deficiency. It is crucial to understand that access to the drug is 'obtained only through a medical prescription.' Patients must consult with their neurologist to 'determine if this treatment is suitable for them' and receive the necessary guidance for its use.
Status in Argentina: The fight for approval The unavailability of Skyclarys in Argentina implies that patients face regulatory and logistical barriers to its access. Patient and family organizations are actively working for the medication to be approved and imported into the country. Meanwhile, in Argentina, patients with FA 'are managed with other treatments and therapies that must be indicated by a physician,' focused on rehabilitation and managing complications. Access to the drug, which was already subject to an evidence review by the National Commission for Health Technology Assessment and Clinical Excellence (CONETEC), remains the main challenge for the Friedreich's Ataxia community in the country.
