FDA Grants Orphan Drug Status to Friedreich’s Ataxia Therapy Candidate

The U.S. Food and Drug Administration’s (FDA) Office of Orphan Products Development recently granted orphan drug designation to Retrotope‘s stabilized fatty acid drug (RT001) for the treatment of Friedreich’s taxia (FA).

The FDA designation follows a statement from the company that RT001 was well tolerated by patients with FA without causing serious adverse reactions or dose limiting toxicities in the first cohort of its Phase 1/2 study.

FA is a rare inherited disease that causes nervous system damage and movement problems. The disorder usually first manifests during childhood beginning between the ages of 5 and 15, but can appear as early as 18 months or as late as age 30, and progresses to muscle coordination impairment (ataxia) that gradually worsens over time. In the U.S., Friedreich’s ataxia affects about 5,000 people, and worldwide it affects more than 20,000 people. There are currently no approved treatments for FA.

RT001 is a chemically stabilized formula of a natural fatty acid that provides resistance to lipid peroxidation in cellular and mitochondrial membranes through a new method. In FA, free iron is a catalyst for lipid peroxidation of exactly the type that can be mitigated with RT001.

Retrotope CEO Robert Molinari, Ph.D., said he was pleased the FDA granted the drug orphan drug status, with the benefits that come with the program. “We have been fortunate to have had the support of the Friedreich’s Ataxia Research Alliance (FARA) since the inception of our clinical program for RT001. We are also grateful to the patients and clinicians who are participating in our first-in-human clinical trial of RT001 for this devastating condition.”

Jennifer Farmer, MS, CGC, executive director of the Friedreich’s Ataxia Research Alliance (FARA), said FARA is pleased to continue its support of Retrotope. “We utilized our Patient Registry to help recruit patients for the Phase 1 that is now fully enrolled. We look forward to a continued partnership as Retrotope’s clinical development advances to further evaluate their new approach to treating FA,” Farmer said. “We are excited by the potential of RT001 and hope this new drug benefits FA patients.”

The company is currently conducting the trial at two clinical locations: the Collaborative NeuroSciences Network in Long Beach, California, and the University of South Florida Ataxia Research Center. The clinical trial is evaluating the safety, tolerability, and pharmacokinetics of RT001 in patients with Friedreich’s ataxia, and is fully enrolled.