FDA approves Reata Pharmaceuticals’ first-in-class treatment for Friedreich’s Ataxia

pharmafile | March 1, 2023 | News story | Medical Communications  

US-based Reata Pharmaceuticals has announced that its first-in-class treatment for Friedreich’s Ataxia (FA), Skyclarys, has been given FDA approval. This approval came on Rare Disease Day.


FA is a rare neuromuscular disease which mainly affects the nervous system and heart. It afflicts roughly one in 50,000 people globally. The Muscular Dystrophy Association (MDA) has welcomed the news, after having a direct impact in the research of FA.


The MDA has invested more than $20m in FA research, as well as supported principal investigator David Lynch from Children’s Hospital of Philadelphia in the establishment of a clinical research network. This network aided the MOXIe Part 2 trial and a post hoc Propensity-Matched Analysis of the open-label MOXIe Extension trial, the efficacy and safety data from which led to the FDA’s approval. Clinical trials of Skyclarys also 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 Friedreich’s Ataxia is a devastating disease that results in loss of corradiated movement, speech and sensory deficits, as well as fatal heart disease; an impactful therapy like Skyclarys brings new hope to patient community served by MDA Care Centers in partnership with the Friedreich’s Ataxia Research Alliance (FARA). We at MDA are delighted to see this and other transformative therapies supported by MDA move to approval and become available to patients,” stated Barry Byrne, M.D., Ph.D., chief medical advisor, MDA and Associate Chair of Paediatrics and Director of the Powell Gene Therapy Center at the University of Florida. 


James Spargo

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