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Disease

Ataxia

Late-stage therapeutic developmentEmerging research
MeSHAtaxia
1
Publications
19
Clinical trials
2021
Latest publication
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this disease.

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Moderate
Key developments
  • 3 clinical trials expected to report results, the earliest in Q2 2027.
  • 2 industry developments reported.
Upcoming Milestones
Clinical Milestones9View all 9
Results posted 4
Results expected 3
Other updates 2
+1 more in the activity timeline below
Industry & Market2View
Activity timeline11

Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies
Omaveloxoloneapproved

Approval — The treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older. (2024)

Clinical trials

15 sponsors · 1 new · 1 completed in the last 12 months (net -1)

The current development programme across all trial phases.

Clinical programme
19
All trials
6
Active
15
Late-stage
6
Completed
Late-stage studies

A 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

Phase 3 · Recruiting · Biogen

Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T: A Phase III, Randomized, Placebo-controlled, Double-blind, Crossover Study

Phase 3 · Active, not recruiting · IntraBio Inc

A Randomized, Parallel-arm, Double Blind, Placebo-controlled Study to Assess the Efficacy of Fampridine for Patients With Spinocerebellar Ataxia SCA27B Caused by a GAA Expansion in the FGF14 Gene

Phase 3 · Recruiting · Assistance Publique - Hôpitaux de Paris

Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia

Phase 3 · Active, not recruiting · PTC Therapeutics

A Phase III, Long-Term, Randomized, Double-blind, Placebo-controlled Trial of Troriluzole in Adult Participants With Spinocerebellar Ataxia.

Phase 3 · Active, not recruiting · Biohaven Pharmaceuticals, Inc.

A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Neurological Effects of EryDex on Subjects With Ataxia Telangiectasia (NEAT)

Phase 3 · Completed · Quince Therapeutics S.p.A.
Recently completed

A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Neurological Effects of EryDex on Subjects With Ataxia Telangiectasia (NEAT)

Phase 3 · Completed · Quince Therapeutics S.p.A.

A 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)

Phase 2/3 · Completed · PTC Therapeutics

Pilot Trial About the Effects of Calcitriol's Treatment in the Neurological Function and Frataxin's Level in Friedreich's Ataxia Patients

Phase 4 · Completed · Berta Alemany

A Randomized, Double-Blind, Controlled, Phase 2/3 Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia

Phase 3 · Completed · Biojiva LLC

Response of Individuals With Ataxia-Telangiectasia to Metformin and Pioglitazone

Phase 4 · Completed · NHS Tayside

A Phase IIb/III, Randomized, Double-blind, Placebo-controlled Trial of Troriluzole in Adult Participants With Spinocerebellar Ataxia

Phase 2/3 · Completed · Biohaven Pharmaceuticals, Inc.

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

First approvals
2024emaApproval — Omaveloxolone· The treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older. source ↗

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

1 papers

Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.

Major themes1
  • Genetic Therapy1
Leading journals1
  • Human molecular genetics1
Leading researchers8
  • Adhikari A1
  • Anderson JS1
  • Beegle J1
  • Cameron DL1
  • Copping NA1
  • Deng P1
  • Fink KD1
  • O'Geen H1
Affiliations (unnormalised)4
  • Institute for Regenerative Cures1
  • MIND Institute1
  • UC Davis Genome Center1
  • University of California Davis School of Medicine1

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

Impairment of the ability to perform smoothly coordinated voluntary movements. This condition may affect the limbs, trunk, eyes, pharynx, larynx, and other structures. Ataxia may result from impaired sensory or motor function. Sensory ataxia may result from posterior column injury or PERIPHERAL NERVE DISEASES. Motor ataxia may be associated with CEREBELLAR DISEASES; CEREBRAL CORTEX diseases; THALAMIC DISEASES; BASAL GANGLIA DISEASES; injury to the RED NUCLEUS; and other conditions.

Identifiers
MeSHAtaxia
References & data sources
  • 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.