New Drug Approval for Friedreich's ataxia
New York, NY, Feb. 28, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) approval of Omaveloxolone (Skyclarys), the first ever treatment for Friedreich’s Ataxia (FA), a rare neuromuscular disease on Rare Disease Day today, February 28. Skyclarys will be made available in the United States (US) and marketed by Reata Pharmaceuticals.
The approval of SKYCLARYS is supported by the efficacy and safety data from the MOXIe Part 2 trial and a post hoc Propensity-Matched Analysis of the open-label MOXIe Extension trial.
“This is an incredible milestone for the Friedreich’s ataxia community,” said Sharon Hesterlee, Ph.D., Chief Research Officer, MDA. “Any therapy with the potential to alter the course of disease progression offers hope for this disease to so many families we serve’.
MDA is proud to have provided steadfast support over a period of five years to principal investigator David Lynch from Children’s Hospital of Philadelphia, an MDA Care Center, for the establishment of a clinical research network for FA. Dr. Lynch is one of the lead investigators of the MOXIe trial who has published seminal manuscripts reporting on the safety and efficacy of Omaveloxolone for viewing here and here.
MDA’s pivotal support for this network has helped towards collecting clinical data and biospecimens of FA patients over the years; an infrastructure that has aided in the clinical testing of Skyclarys in patients. Additionally, clinical trials of Skyclarys took place at MDA Care Center Network locations including UCLA, University of Florida Neurology, Emory University Hospital, The Ohio State University, and Children’s Hospital of Philadelphia.
Since the inception of MDA, the organization has invested more than $20 million in FA research.
“The approval of this groundbreaking, first-ever treatment for Friedreich’s Ataxia is both an answer to prayer and the result of hard work,” said Tom Henry, a volunteer MDA National Vice President and proud member of the MDA family as a father of two young adult sons living with the disease. “When my children were diagnosed, we partnered with our community of friends, business colleagues, and other MDA families to enable research and accelerate treatments through fundraising and volunteer work. Today, it is beyond gratifying to see the fruits of our labors starting to pay off —giving families like ours hope that their loved ones with genetic, neuromuscular diseases may live long and pain-free lives. Many thought it was impossible to develop treatments for rare genetic diseases, but our family, alongside the Muscular Dystrophy Association, has never wavered in our efforts to make a day like today possible. We are grateful to be surrounded by a community that will never give up on each other, and we are so excited about this new treatment. With that being said, this is just the beginning, because we do not plan to stop working until all genetic diseases are cured.”