What potential treatments are in development?

Drug Development Pipeline

The pipeline is a visual tool for communicating the progress of potential therapeutics.

To download a printable PDF of the pipeline, click here.

Lead candidates are grouped based on mechanism of action or approach to treatment. The horizontal axis indicates the stage of the research (in the country where the candidate is furthest in its development process).

FARA supports the advancement of treatments with financial resources, advocacy, patient engagement and/or fostered collaboration. FARA believes that an effective treatment for FA will come in the form of a “cocktail approach” — a combination of two or more therapies. The descriptions of programs shared here are for informational purposes only and are not medical advice or an endorsement of any program.

To learn more about the stages of drug development, click here.

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Discovery
Finding potential therapies / drugs
Pre-clinical development
Testing in laboratory
Regulatory Submission
Permission to enter clinic
Phase I
Human safety trial
Phase II
Human safety and efficacy trial
Phase III
Definitive trial
Request for Approval
Permission to enter market
Available to patients

Improve Mitochondrial Function & Reduce Oxidative Stress

SKYCLARYS (omaveloxolone)

Biogen

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Available to patients *
Available to patients *

Improve Mitochondrial Function & Reduce Oxidative Stress

Vatiquinone (PTC-743)

PTC Therapeutics

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Phase III
Phase III

Improve Mitochondrial Function & Reduce Oxidative Stress

NAD+ and Exercise in FA

Children’s Hospital of Philadelphia

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Phase II
Phase II

Improve Mitochondrial Function & Reduce Oxidative Stress

Elamipretide

Children’s Hospital of Philadelphia

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Phase I/II
Phase I/II

Modulation of Frataxin Controlled Metabolic Pathways

Leriglitazone (MIN-102)

Minoryx Therapeutics

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Phase II
Phase II

Modulation of Frataxin Controlled Metabolic Pathways

Dimethyl Fumarate

University of Federico II

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Phase II
Phase II

Frataxin Replacement, Stabilizers, or Enhancers

Nomlabofusp (CTI-1601)

Larimar Therapeutics

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Phase II
Phase II

Increase Frataxin Gene Expression

DT-216P2

Design Therapeutics

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Phase I
Phase I

Cell Therapy

PPL-001

Papillon Therapeutics

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Discovery
Discovery

Gene Replacement

LX2006 Gene Therapy

Lexeo Therapeutics

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Phase I/II
Phase I/II

Gene Replacement

ASP2016

Astellas Gene Therapies

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Phase I
Phase I

Gene Replacement

AVB-202-TT Gene Therapy

Solid Biosciences

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Pre-clinical
Pre-clinical

Gene Replacement

Neurocrine Biosciences / Voyager Therapeutics AAV-Gene Therapy

Neurocrine Biosciences / Voyager Therapeutics

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Discovery
Discovery

Gene Editing

AAV Gene Editing

Prime Medicine

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Discovery
Discovery

Discontinued

Acetyl-L-Carnitine or ALCAR

University of South Florida

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Discontinued

Discontinued

BMN 290

BioMarin/RepliGen

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Discontinued

Discontinued

Calcitriol (vitamin D3)

Berta Alemany, Institut d’Inverstigacio Biomedica de Girona

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Discontinued

Discontinued

Chantix® (varenicline)

FARA, University of South Florida

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Discontinued

Discontinued

Crestor® (rosuvastatin)

Children’s Hospital of Philadelphia (CHOP)

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Discontinued

Discontinued

Deferiprone

Apopharma

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Discontinued

Discontinued

EPI-A0001

Edison Pharmaceuticals

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Discontinued

Discontinued

Epicatechin

Mayo Clinical and Epirium Bio (formerly Cardero Therapeutics)

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Discontinued

Discontinued

Etravirine

Fratagene

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Discontinued

Discontinued

EPO (epoetin a)

Department of Neurology, Innsbruck Medical University, Innsbruck, Austria; Federico II University

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Discontinued

Discontinued

Exenatide (incretin analogs)

Universite Libre de Bruxelles, Brussels, Belgium

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Discontinued

Discontinued

GCSF

University of Bristol

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Discontinued

Discontinued

Idebenone (Raxone®/Catena®)

Santhera Pharmaceuticals

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Discontinued

Discontinued

Interferon gamma (Actimmune®)

Horizon Therapeutics

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Discontinued

Discontinued

LU AA24493

Lundbeck

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Discontinued

Discontinued

Methylprednisolone

Children’s Hospital of Philadelphia

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Discontinued

Discontinued

Nicotinamide

Richard Festenstein, Imperial College; Jorg Schulz, Aachen University

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Discontinued

Discontinued

OXIGON (OX1 aka SHP622, VP20629)

Shire (Viro Pharma)

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Discontinued

Discontinued

Pioglitazone

Pierre Rustin, Hospital Robert Debre

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Discontinued

Discontinued

Resveratrol

Jupiter Orphan Therapeutics, Murdoch Children's Research Institute

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Discontinued

Discontinued

RT001

Retrotope

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Discontinued

Discontinued

TAK-831

Takeda Pharmaceuticals

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Discontinued