Friedreich’s Ataxia (FA)

Friedreich’s ataxia (FA) is a debilitating and life-shortening neurodegenerative disease that affects approximately 5,000 people in the United States and over 20,000 people worldwide. FA results in the progressive loss of coordination and muscle strength which then leads to motor incapacitation, wheelchair dependence, and ultimately early death most commonly due to cardiomyopathy. There are currently no approved treatments for FA. 

Retrotope is currently developing RT001, its lead clinical candidate, for the treatment of FA. The company completed enrollment in its ongoing pivotal Phase 2/3 trial of RT001 in FA in late 2020.  Data from this trial are expected to read out by the end of 2021.  Proof-of-concept for RT001 in FA was demonstrated in the company’s previously completed Phase 1/2 placebo-controlled trial.

RT001 has been granted rare pediatric disease designation, orphan drug designation and Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of FA.

Additional patient and healthcare provider resources on Friedreich’s ataxia are available in the Patient Resources section.

“The entire team at FARA is grateful for the dedication that Retrotope has demonstrated in its effort to develop a novel treatment for FA.  We are excited that the company has completely enrolled its ongoing pivotal study of RT001 in patients with FA and look forward to the read out of data from the trial.”

– Jennifer Farmer, chief executive officer, Friedreich’s Ataxia Research Alliance (FARA).