Friedreich Ataxia
Recent clinical, regulatory, research and industry developments relating to this disease.
Emerging therapies for childhood-onset movement disorders.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
- 4 clinical trials expected to report results, the earliest in Q1 2027.
- Q1 2027An Open-Label Extension Study to Assess the Long-Term Safety, Efficacy, Pharmacodynamics, Pharmacokinetics, and Tolerability of Subcutaneous CTI-1601 in Subjects With Friedreich's Ataxia
- Q4 2027A Phase 3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Omaveloxolone (BIIB141) in Participants With Friedreich's Ataxia Aged 2 to < 16 Years
- Q4 2027Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia
- Q4 2028Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia
Clinical MilestonesView all 9Hide
- 2026-04-13A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study With Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich Ataxia (MOVE-FA)Results posted
- 2026-01-23A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Friedreich's Ataxia (MOXIe)Results posted
- 2026-06-16A Phase 3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Omaveloxolone (BIIB141) in Participants With Friedreich's Ataxia Aged 2 to < 16 YearsResults expected Q4 2027
- 2026-05-13An Open-Label Extension Study to Assess the Long-Term Safety, Efficacy, Pharmacodynamics, Pharmacokinetics, and Tolerability of Subcutaneous CTI-1601 in Subjects With Friedreich's AtaxiaResults expected Q1 2027
- 2026-03-31Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's AtaxiaResults expected Q4 2028
- 2025-10-30Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich AtaxiaResults expected Q4 2027
- 2025-12-01A Randomized, Double-blind, Placebo-controlled, Parallel-group, Multicentre Study of the Efficacy and Safety of Nicotinamide in Patients With Friedreich AtaxiaWithdrawn
- 2025-10-22A Multicenter, Open-Label, Dose Escalation, Phase 1b Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of ASP2016 for Friedreich Ataxia CardiomyopathyWithdrawn
- 2026-06-16ClinicalA Phase 3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Omaveloxolone (BIIB141) in Participants With Friedreich's Ataxia Aged 2 to < 16 YearsResults expected Q4 2027
- 2026-05-13ClinicalAn Open-Label Extension Study to Assess the Long-Term Safety, Efficacy, Pharmacodynamics, Pharmacokinetics, and Tolerability of Subcutaneous CTI-1601 in Subjects With Friedreich's AtaxiaResults expected Q1 2027
- 2026-04-13ClinicalA Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study With Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich Ataxia (MOVE-FA)Results posted
- 2026-03-31ClinicalPhase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's AtaxiaResults expected Q4 2028
- 2026-01-23ClinicalA Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Friedreich's Ataxia (MOXIe)Results posted
- 2025-12-01ClinicalA Randomized, Double-blind, Placebo-controlled, Parallel-group, Multicentre Study of the Efficacy and Safety of Nicotinamide in Patients With Friedreich AtaxiaWithdrawn
- 2025-10-30ClinicalLong-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich AtaxiaResults expected Q4 2027
- 2025-10-22ClinicalA Multicenter, Open-Label, Dose Escalation, Phase 1b Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of ASP2016 for Friedreich Ataxia CardiomyopathyWithdrawn
- 2025-09-30ClinicalAn Open-Label, Phase 1, Study to Evaluate the Pharmacokinetics, Safety, Tolerability, Pharmacodynamics, and Efficacy of Omaveloxolone in Participants ≥2 to <16 Years of Age With Friedreich's AtaxiaPrimary completion
Therapeutic landscape
Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.
Approval — The treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older. (2024)
Clinical trials
The current development programme across all trial phases.
Regulatory timeline
Drug regulatory events matched to this condition by indication — EMA.
European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Major themes1
- Movement Disorders1
Leading journals1
- Current opinion in pediatrics1
Leading researchers3
- Ebrahimi-Fakhari D1
- Quiroz V1
- Vogt L1
Affiliations (unnormalised)2
- F.M. Kirby Neurobiology Center1
- The Hospital for Sick Children1
Reference
Authoritative identity, definition & identifiers.
An autosomal recessive disease, usually of childhood onset, characterized pathologically by degeneration of the spinocerebellar tracts, posterior columns, and to a lesser extent the corticospinal tracts. Clinical manifestations include GAIT ATAXIA, pes cavus, speech impairment, lateral curvature of spine, rhythmic head tremor, kyphoscoliosis, congestive heart failure (secondary to a cardiomyopathy), and lower extremity weakness. Most forms of this condition are associated with a mutation in a gene on chromosome 9, at band q13, which codes for the mitochondrial protein frataxin. (From Adams et al., Principles of Neurology, 6th ed, p1081; N Engl J Med 1996 Oct 17;335(16):1169-75) The severity of Friedreich ataxia associated with expansion of GAA repeats in the first intron of the frataxin gene correlates with the number of trinucleotide repeats. (From Durr et al, N Engl J Med 1996 Oct 17;335(16):1169-75)
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
- Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
- Related entities are derived from literature co-mention (studied together) — associative, not causal.