VivoSeq
Neurology

Friedreich Ataxia

22

Pipeline Programs↗

2

Active Trials↗

19

Companies Involved↗

Friedreich ataxia is an emerging landscape with 22 programs across 19 companies. The most active players are Design Therapeutics, Inc., Larimar Therapeutics, Inc., Auerbach Hematology Oncology Associates P C. 4 therapies are approved with 2 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

48/100

Pipeline Velocity

6/100

Mech. Concentration

22/100

Completion Rate

85/100

Attrition Rate

14/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

D

Design Therapeutics, Inc.

1 program

Phase 1L

Larimar Therapeutics, Inc.

1 program

Phase 1A

Auerbach Hematology Oncology Associates P C

1 program

Phase 1AstraZeneca logo

AstraZeneca

1 program

Phase 1Pfizer logo

Pfizer

1 program

Phase 1P

Pfizer's Upjohn has merged with Mylan to form Viatris Inc.

1 program

Phase 1

Pipeline

Programs by Phase

Analysis

Trial Status

22

clinical trials

Completed17 · 77%
Withdrawn2 · 9%
Active, not recruiting1 · 5%
Terminated1 · 5%
Recruiting1 · 5%

Clinical Trials

Trial Activity

22 trials

A Post-marketing, Observational, Descriptive Study to Assess the Risk Associated With Pregnancy, the Maternal Complications, and Adverse Effects on the Developing Fetus, Neonate, and Infant Among Individuals Exposed to Omaveloxolone During Pregnancy and/or Lactation
Recruiting·Biogen logoBiogen·N/A·NCT06628687·10 enrolled
A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
Active, not recruiting·PPTC Therapeutics·Phase 3·NCT05515536·130 enrolled
Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia
Completed·DDesign Therapeutics, Inc.·Phase 1·NCT05573698·32 enrolled
Study to Evaluate DT-216 in Adult Patients With Friedreich Ataxia
Completed·DDesign Therapeutics, Inc.·Phase 1·NCT05285540·39 enrolled
An Objective Double-blind Evaluation of Bupropion and Citalopram in an Individual With Friedreich Ataxia
Completed·UUniversity of Colorado, Denver·Phase 4·NCT01716221·1 enrolled
Methylprednisolone Treatment of Friedreich Ataxia
Completed·—·Phase 1·NCT02424435·11 enrolled
Micronised Resveratrol as a Treatment for Friedreich Ataxia
Completed·MMurdoch Childrens Research Institute·Phase 2·NCT03933163·25 enrolled
Rosuvastatin (Crestor) in Friedreich Ataxia
Completed·—·Phase 1·NCT02705547·12 enrolled
Safety and Efficacy of Etravirine in Friedreich Ataxia Patients
Completed·IIRCCS Eugenio Medea·Phase 2·NCT04273165·30 enrolled
Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia
Completed·Neurocrine Biosciences logoNeurocrine Biosciences·Phase 2·NCT03214588·67 enrolled
A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
Completed·PPTC Therapeutics·Phase 2·NCT05485987·5 enrolled
Efficacy of EGb761 in Patients Suffering From Friedreich Ataxia
Completed·Ipsen logoIpsen·Phase 2·NCT00824512·22 enrolled
Interferon Gamma-1b in Friedreich Ataxia (FRDA)
Completed·—·Phase 2·NCT01965327·12 enrolled
Open Trail of γIFN for Friedreich Ataxia
Completed·IIRCCS Eugenio Medea·Phase 2·NCT03888664·12 enrolled
Efficacy Study of Epoetin Alfa in Friedreich Ataxia
Completed·FFederico II University·Phase 2·NCT01493973·56 enrolled
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Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)