On this Rare Disease Day, February 28, the Muscular Dystrophy Association (MDA) celebrates the groundbreaking news from the US Food and Drug Administration (FDA): Omaveloxolone (Skyclarys) has become the first ever treatment approved for Friedreich’s Ataxia (FA). This momentous milestone will be made available in the United States (US) through Reata Pharmaceuticals.
SKYCLARYS has been approved based on the impressive evidence from the MOXIe Part 2 trial and a post hoc Propensity-Matched Analysis of the open-label MOXIe Extension trial. Both trials demonstrated that SKYCLARYS is both safe and effective, making it a reliable treatment option for those in need.
The Friedreich’s ataxia community is celebrating a momentous achievement with the potential of a new therapy to change the course of this disease. Sharon Hesterlee, Ph.D., Chief Research Officer of MDA, expresses her excitement, stating “This is an incredible milestone for the Friedreich’s ataxia community. Any therapy with the potential to alter the course of disease progression offers hope for this disease to so many families we serve.”
MDA has proudly supported David Lynch, Principal Investigator from Children’s Hospital of Philadelphia and MDA Care Center, over the past five years in establishing a clinical research network for FA. Dr. Lynch is a renowned leader in the MOXIe trial, and his groundbreaking research on the safety and efficacy of Omaveloxolone has been documented in two seminal publications which can be found here and here.
MDA has been a vital supporter of a network that has helped to collect clinical data and biospecimens of FA patients over the years, providing an invaluable infrastructure for the clinical testing of Skyclarys. Clinical trials of Skyclarys have taken place in MDA Care Center Network locations across the country, including UCLA, University of Florida Neurology, Emory University Hospital, The Ohio State University, and Children’s Hospital of Philadelphia. This network of support has been essential for advancing the treatment of FA patients.
Since its establishment, MDA has been dedicated to finding treatments and cures for FA and has invested a staggering $20 million in research to that end. The organization continues to be a leader in the fight against this debilitating disorder.
Today marks a momentous occasion for the MDA family, as we celebrate the approval of the first-ever treatment for Friedreich’s Ataxia. This moment is the result of years of hard work, prayer and passion from families like ours. When our children were diagnosed with genetic neuromuscular diseases, we partnered with our community to help raise funds and accelerate treatments. It is with great joy that we now see the fruits of our labor and the proof that no disease is too rare or too difficult to treat. We are grateful to be surrounded by a community that never gives up on each other, and we are overjoyed to have this new treatment that brings hope to so many families. This is only the beginning, as we will never stop working until all genetic diseases are cured.
At MDA, we are thrilled to see Skyclarys, an impactful therapy for Friedreich’s Ataxia, quickly gaining approval and becoming available to the patient community served by our Care Centers. This devastating disease is characterized by loss of corradiated movement, speech and sensory deficits, as well as fatal heart disease, and the introduction of Skyclarys brings newfound hope to those affected. We are proud to work with the Friedreich’s Ataxia Research Alliance (FARA) in giving those suffering from this disorder the opportunity to live a better quality of life.
Katherine Mathews, M.D., MDA Care Center Director and Professor of Pediatrics-General Neurology at University of Iowa Health Care, is thrilled to have a disease-modifying treatment available for her FA patients! With improved management options for many MDA-covered diseases, she has seen great success in providing the best care for those afflicted with this rare and complex condition. The MDA Care Center Network offers patients the confidence and assurance that their care is being provided by those who have experience in treating the disease.
About Friedreich’s Ataxia
Friedreich Ataxia (FA) is a rare, yet debilitating neuromuscular disorder that affects the nervous system and the heart. Discovered in 1863 by German physician Nikolaus Friedreich, it is the most common form of hereditary ataxias, affecting approximately one in 50,000 people worldwide. Though it may be confused with autosomal dominant spinocerebellar ataxias, Friedreich Ataxia is a distinct disorder with its own unique set of symptoms and challenges.
About Muscular Dystrophy Association
For seventy years, the Muscular Dystrophy Association (MDA) has been the leading health organization in the United States for those living with muscular dystrophy, ALS, and other neuromuscular diseases. We strive to give hope to individuals and families affected by these conditions by accelerating research for treatments, advocating for better services, and advancing care. It is our passion to empower those we serve to live longer, healthier, and more independent lives. Come join us in this mission and help us make a difference!