In the monthly newsletter from the Friedreich's Ataxia Research Alliance (FARA), the FA community received great news about a promising drug in development.
Edison Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug status to vatiquinone (EPI-743) for the treatment of Friedreich’s ataxia. Orphan status brings additional momentum and acceleration to the clinical development of EPI-743 now underway at three sites in the FARA Collaborative Clinical Research Network.
"This is an important step forward in moving EPI-743 towards approval for the treatment of Friedreich's ataxia," said Ron Bartek, FARA co-founder and President. "FARA has been working closely with Edison, the FDA and National Institutes of Health to advance EPI-743 to this very promising stage of development. We are grateful to Edison, our government partners, the dedicated teams of our Collaborative Clinical Research Network, and to the patients and patient families that have enabled us to recruit the 60 EPI-743 clinical trial participants in a matter of hours."
For more information, see the full press release here. Thank you for your continued support! Together we will cure Friedreich’s Ataxia!