FDA Grants Special Designation to Friedreich's Ataxia Treatment

October 25, 2022

AUCD's network of Intellectual and Developmental Disability Research Centers (IDDRCs) consists of 16 Centers. Fifteen Centers currently receive funding from the Eunice Kennedy Shriver National Institute for Child Health and Human Development (NICHD). IDDRCs contribute to the development and implementation of evidence-based practices by evaluating the effectiveness of biological, biochemical, and behavioral interventions; developing assistive technologies; and advancing prenatal diagnosis and newborn screening.

A potential new therapy for the progressive neurological disorder, Friedreich's Ataxia (FA) was granted Fast Track Designation and Orphan Drug Designation by the U.S. Food and Drug Administration based on study findings led by a  neurologist at CHOP which is one of AUCD's Intellectual & Developmental Disabilities Research Centers.

The designation puts the treatment, called omaveloxolone, on an expedited process to become the first therapy approved for patients with FA. An oral, once-daily drug, omaveloxolone works by activating Nrf2, a transcription factor that induces molecular pathways that promote the resolution of neuroinflammation by restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling.

In collaboration with the biopharmaceutical company Reata Pharmaceutical and other colleagues around the globe, David Lynch, MD, PhD, director of the FA Program at CHOP, published results that show omaveloxolone has a potentially beneficial effect in slowing the progression of FA out to more than two years. In their study published in Neurodegenerative Disease Management, the researchers show that omaveloxolone modifies the long-term behavior of FA, and when analyzed in a delayed-start fashion, each trial cohort benefitted equally from the drug no matter when they started omaveloxolone.

Read more: https://www.chop.edu/news/fda-grants-special-designation-friedreich-s-ataxia-treatment-based-findings-study-led-chop