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Research Pipeline

The Friedreich's Ataxia Treatment Pipeline is a visual tool for communicating the progress of research and development on lead therapeutic candidates.

Along the vertical axis lead candidates are grouped based on mechanism of action or approach to treatment, e.g., where or how each drug might work in the cell, technological approach, or problem being addressed. The horizontal axis indicates the stage of the research - where the candidate is in development.

The first two stages, discovery and pre-clinical, take place in the research laboratory, and represent early discovery and development. The IND stage is an important milestone – this is when there is a regulatory filing with the United States Food and Drug Administration (FDA) to begin human studies. The stages “Phase 1” through “Phase 3” are phases of clinical trials/studies, when a drug is tested in individuals for safety and efficacy. With sufficient evidence proving a drug benefit, a new drug application (NDA) can be filed with the FDA. Once an NDA is approved, the drug then becomes available to individuals in their pharmacies.

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FARA is supporting the advancement of these treatments with financial resources, advocacy, patient engagement and/or fostered collaboration. FARA believes that there is merit in each of these approaches and that effective treatment will come in the form of a "cocktail approach" - a combination of two or more therapies.

This pipeline has been updated as of May 2021 and focuses on drugs that are already in preclinical development or have advanced to human clinical trial.


Currently on the Research Pipeline:

+ Omaveloxolone (RTA 408)
+ PTC-743
+ RT001 (dPUFAs)
+ NAD+ and Exercise in FA (ExRx in FA)
+ MIB-626
+ MIN-102
+ Dimethyl Fumarate
+ CTI-1601 (TAT-Frataxin)
+ Etravirine
+ Resveratrol
+ Gene-Tac (Syn-TEFS)
+ Oligonucleotides
+ Gene Replacement and Editing


Candidates below are not presently on the pipeline displayed above

These compounds have been previously tested in animal or cellular models of FA or in prior human clinical trials
+ ALCAR (Acetyl-L-Carnitine)
+ BMN 290 / HDACi
+ Calcitriol (Vitamin D3)
+ CAT-4001
+ Crestor
+ Cytokine Mediated Therapy - GCSF
+ Deferiprone
+ EGb-761
+ EPI-A0001
+ Epicatechin
+ EPO-mimetics
+ Erythropoietin (EPO)
+ Idebenone / Catena
+ Incretin analogs
+ Interferon gamma
+ Lu AA24493 carbamylated EPO
+ Methylprednisolone
+ Nicotinamide
+ Nutritional approach
+ Pioglitazone
+ SHP622 (formally VP20629 or OX1)
+ TAK-831
+ Ubiquitin Competitors
+ Varenicline (Chantix®)



FA Treatment Pipeline Webinar
FARA hosted a 90 minute webinar on the FA Treatment Pipeline on June 2, 2021, providing updates on therapeutic approaches in the pipeline with Jen Farmer, FARA's Chief Executive Officer; Dr. Barbara Tate, Chief Scientific Officer; and Susan Walther, Director of Patient Engagement. They reviewed the overall approach and strategy of the pipeline along with progress and new results for specific therapeutics approaches.


Clinical Trials

Clinical Trials - Active - Enrolling

Clinical Trials - Active - Enrollment Closed

Clinical Trials - Completed


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