Friedreich Ataxia

Neurology
24
Pipeline Programs
11
Companies
35
Clinical Trials
5 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
9
5
8
1
1
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Peptide
267%
Small Molecule
133%
+ 31 programs with unclassified modality

Competitive Landscape

11 companies ranked by most advanced pipeline stage

PTC Therapeutics
3 programs
1
1
1
VatiquinonePhase 31 trial
VatiquinonePhase 2/31 trial
VatiquinonePhase 21 trial
Active Trials
NCT05485987Completed5Est. Aug 2024
NCT04577352Completed146Est. Oct 2023
NCT05515536Active Not Recruiting130Est. Dec 2027
Alliance Pharmaceuticals
12 programs
2
2
2
Epoetin alfaPhase 21 trial
Interferon Gamma-1bPhase 21 trial
ElamipretidePhase 1/2Peptide
ResveratrolPhase 1/21 trial
MethylprednisolonePhase 11 trial
+7 more programs
Active Trials
NCT03090789Active Not Recruiting2,000Est. Jan 2030
NCT06016946Recruiting3,000Est. Jan 2048
NCT06560346Withdrawn0Est. Dec 2028
+8 more trials
Larimar Therapeutics
Larimar TherapeuticsPA - Bala Cynwyd
6 programs
3
2
CTI-1601Phase 21 trial
CTI-1601Phase 21 trial
CTI-1601Phase 11 trial
CTI-1601Phase 11 trial
NomlabofuspPhase 11 trial
+1 more programs
Active Trials
NCT04255680Completed20Est. Jun 2020
NCT04519567Completed27Est. Mar 2021
NCT04176991Completed28Est. Oct 2020
+3 more trials
Ipsen
IpsenChina - Tianjin
1 program
1
EGb 761 120 mgPhase 21 trial
Active Trials
NCT00824512Completed22Est. Oct 2011
Aisa Pharma
Aisa PharmaMA - Boston
1 program
1
Epoetin alfaPhase 2
Takeda
TakedaTOKYO, Japan
1 program
1
TAK-831Phase 24 trials
Active Trials
NCT04234672Completed6Est. Apr 2020
NCT03706469Completed16Est. Dec 2018
NCT03214588Completed67Est. Dec 2018
+1 more trials
Design Therapeutics
4 programs
3
1
DT-216P2Phase 1/21 trial
DT-216Phase 11 trial
DT-216Phase 11 trial
DT-216P2Phase 11 trial
Active Trials
NCT05573698Completed32Est. Aug 2023
NCT05285540Completed39Est. Dec 2022
NCT06772870Not Yet Recruiting36Est. Dec 2025
+1 more trials
Lexeo Therapeutics
Lexeo TherapeuticsNY - New York
2 programs
1
Low dose LX2006Phase 1/21 trial
Clinical Course Of Disease In Participants With FA-CMN/A1 trial
Active Trials
NCT06865482Recruiting65Est. Sep 2027
NCT05445323Active Not Recruiting8Est. Sep 2029
Stealth BioTherapeutics
1 program
1
ElamipretidePhase 1/2Peptide1 trial
Active Trials
NCT05168774Completed20Est. Jul 2024
Astellas
AstellasChina - Shenyang
1 program
1
ASP2016Phase 11 trial
Active Trials
NCT06483802Withdrawn0Est. Oct 2025
Biogen
BiogenCAMBRIDGE, MA
2 programs
OmaveloxoloneN/A1 trial
OmaveloxoloneN/A1 trial
Active Trials
NCT06628687Recruiting20Est. Apr 2035
NCT06623890Recruiting300Est. Oct 2029

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
PTC TherapeuticsVatiquinone
PTC TherapeuticsVatiquinone
Larimar TherapeuticsCTI-1601
PTC TherapeuticsVatiquinone
Larimar TherapeuticsCTI-1601
TakedaTAK-831
Alliance PharmaceuticalsInterferon Gamma-1b
Alliance PharmaceuticalsEpoetin alfa
IpsenEGb 761 120 mg
Design TherapeuticsDT-216P2
Lexeo TherapeuticsLow dose LX2006
Stealth BioTherapeuticsElamipretide
Alliance PharmaceuticalsResveratrol
Design TherapeuticsDT-216P2
Larimar TherapeuticsNomlabofusp

Showing 15 of 35 trials with date data

Clinical Trials (35)

Total enrollment: 6,676 patients across 35 trials

NCT05515536PTC TherapeuticsVatiquinone

A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia

Start: Dec 2022Est. completion: Dec 2027130 patients
Phase 3Active Not Recruiting
NCT04577352PTC TherapeuticsVatiquinone

A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia

Start: Dec 2020Est. completion: Oct 2023146 patients
Phase 2/3Completed
NCT06447025Larimar TherapeuticsCTI-1601

An Open Label Extension Study of CTI-1601 in Subjects With Friedreich's Ataxia

Start: Jan 2024Est. completion: Jan 202775 patients
Phase 2Enrolling By Invitation
NCT05485987PTC TherapeuticsVatiquinone

A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia

Start: Oct 2022Est. completion: Aug 20245 patients
Phase 2Completed
NCT05579691Larimar TherapeuticsCTI-1601

A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's Ataxia

Start: Sep 2022Est. completion: Dec 202328 patients
Phase 2Completed
NCT03214588TakedaTAK-831

Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia

Start: Nov 2017Est. completion: Dec 201867 patients
Phase 2Completed
NCT01965327Alliance PharmaceuticalsInterferon Gamma-1b

Interferon Gamma-1b in Friedreich Ataxia (FRDA)

Start: Aug 2013Est. completion: Oct 201412 patients
Phase 2Completed
NCT01493973Alliance PharmaceuticalsEpoetin alfa

Efficacy Study of Epoetin Alfa in Friedreich Ataxia

Start: Jan 2013Est. completion: Jun 201556 patients
Phase 2Completed
NCT00824512IpsenEGb 761 120 mg

Efficacy of EGb761 in Patients Suffering From Friedreich Ataxia

Start: Jun 2008Est. completion: Oct 201122 patients
Phase 2Completed
NCT06874010Design TherapeuticsDT-216P2

A Multiple Ascending Dose Study of DT-216P2 in Patients With Friedreich's Ataxia

Start: Jun 2025Est. completion: Mar 202720 patients
Phase 1/2Recruiting
NCT05445323Lexeo TherapeuticsLow dose LX2006

Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia

Start: Aug 2022Est. completion: Sep 20298 patients
Phase 1/2Active Not Recruiting
NCT05168774Stealth BioTherapeuticsElamipretide

FRDA Investigator Initiated Study (IIS) With Elamipretide

Start: Mar 2022Est. completion: Jul 202420 patients
Phase 1/2Completed
NCT01339884Alliance PharmaceuticalsResveratrol

A Study of Resveratrol as Treatment for Friedreich Ataxia

Start: Apr 2011Est. completion: Dec 201227 patients
Phase 1/2Completed
NCT06772870Design TherapeuticsDT-216P2

A Single Ascending Dose Study of DT-216P2 in Normal Healthy Participants

Start: Feb 2025Est. completion: Dec 202536 patients
Phase 1Not Yet Recruiting
NCT06681766Larimar TherapeuticsNomlabofusp

A Study to Assess Nomlabofusp in Adolescents and Children With Friedreich's Ataxia

Start: Dec 2024Est. completion: Apr 202518 patients
Phase 1Terminated
NCT06483802AstellasASP2016

A Study of ASP2016 in Adults Who Have Heart Disease Associated With Friedreich Ataxia

Start: Nov 2024Est. completion: Oct 20250
Phase 1Withdrawn
NCT05573698Design TherapeuticsDT-216

Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia

Start: Sep 2022Est. completion: Aug 202332 patients
Phase 1Completed
NCT05285540Design TherapeuticsDT-216

Study to Evaluate DT-216 in Adult Patients With Friedreich Ataxia

Start: Mar 2022Est. completion: Dec 202239 patients
Phase 1Completed
NCT04519567Larimar TherapeuticsCTI-1601

Multiple Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia

Start: Jul 2020Est. completion: Mar 202127 patients
Phase 1Completed
NCT04234672TakedaTAK-831

A Study to Assess Absolute Bioavailability (ABA) of TAK-831 and to Characterize Mass Balance, Pharmacokinetics (PK), Metabolism, and Excretion of [14C]TAK-831 in Male Healthy Participants

Start: Feb 2020Est. completion: Apr 20206 patients
Phase 1Completed
NCT04176991Larimar TherapeuticsCTI-1601

Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia

Start: Dec 2019Est. completion: Oct 202028 patients
Phase 1Completed
NCT03706469TakedaTAK-831

A Study to Evaluate the Relative Bioavailability (BA) and Effect of Food on TAK-831 Tablet Formulations in Healthy Participants

Start: Oct 2018Est. completion: Dec 201816 patients
Phase 1Completed
NCT03101293TakedaTAK-831

A Study to Evaluate the Effect of Food on the Pharmacokinetics of TAK-831 Tablet Formulation

Start: Apr 2017Est. completion: May 201715 patients
Phase 1Completed
NCT02705547Alliance PharmaceuticalsRosuvastatin

Rosuvastatin (Crestor) in Friedreich Ataxia

Start: May 2016Est. completion: Aug 201712 patients
Phase 1Completed
NCT02424435Alliance PharmaceuticalsMethylprednisolone

Methylprednisolone Treatment of Friedreich Ataxia

Start: Jun 2015Est. completion: Apr 201811 patients
Phase 1Completed
NCT06628687BiogenOmaveloxolone

A Study to Learn How BIIB141 (Omaveloxolone) Affects the Health of Participants With Friedrich's Ataxia Who Took it During Pregnancy and/or During Breastfeeding and About the Health of Their Babies

Start: Oct 2026Est. completion: Apr 203520 patients
N/ARecruiting
NCT06865482Lexeo TherapeuticsClinical Course Of Disease In Participants With FA-CM

Clinical Course Of Disease In Participants With FA-CM

Start: Sep 2025Est. completion: Sep 202765 patients
N/ARecruiting
NCT06560346Alliance PharmaceuticalsGeneticlly confirmed disease causing FXN mutatuion

Understanding the Natural History Early in the Course or Presentation of Friedreich Ataxia

Start: May 2025Est. completion: Dec 20280
N/AWithdrawn
NCT06623890BiogenOmaveloxolone

A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors

Start: Dec 2024Est. completion: Oct 2029300 patients
N/ARecruiting
NCT06016946Alliance PharmaceuticalsFriedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study

Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study

Start: Jun 2023Est. completion: Jan 20483,000 patients
N/ARecruiting
NCT04649866Alliance Pharmaceuticalsplethysmography

Coronary Artery Disease in Patients With Friedreich's Ataxia

Start: Feb 2021Est. completion: Sep 20237 patients
N/ACompleted
NCT04349514Alliance PharmaceuticalsNatural history

A Natural History Study to TRACK Brain and Spinal Cord Changes in Individuals with Friedreich Ataxia (TRACK-FA)

Start: Feb 2021Est. completion: Oct 2025300 patients
N/AActive Not Recruiting
NCT04255680Larimar TherapeuticsBuccal Swabs and Blood Draws

A Study to Assess Variation in Potential Biomarkers in Friedreich Ataxia

Start: Jan 2020Est. completion: Jun 202020 patients
N/ACompleted
NCT03418740Alliance PharmaceuticalsNeurology Measures in FA Children

Neurology Measures in FA Children

Start: Nov 2017Est. completion: Feb 2023108 patients
N/ACompleted
NCT03090789Alliance PharmaceuticalsFA Clinical Outcome Measures

FA Clinical Outcome Measures

Start: Jan 2001Est. completion: Jan 20302,000 patients
N/AActive Not Recruiting

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Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

1 late-stage (Phase 3) programs — potential near-term approvals
5 actively recruiting trials targeting 6,676 patients
11 companies competing in this space

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