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Avicel

(Idebenone)

Phase 3Small molecule

SSanthera Pharmaceuticals

Idebenone is used to treat a rare genetic eye disease called Leber hereditary optic neuropathy (LHON), which causes vision loss. It works by helping cells produce energy more efficiently.[1] Avicel is currently being investigated across 7 indications[3], with its most advanced program in Phase 3.[1]

Avicel is one of 143 small molecule programs targeting Eye Disease. It is currently in Phase 3.

7

Indications

29

Total Trials

2

Active Trials

142

Competitors

Pipeline

Status by Indication[3][1]

Indication

Eye DiseaseActive, not recruiting

Leber Hereditary Optic NeuropathyUnknown

Attention Deficit Hyperactivity DisorderUnknown

Cognitive ImpairmentUnknown

Friedreich AtaxiaUnknown

MigraineUnknown status

SynucleinopathiesActive, not recruiting

Eye DiseasePreclinical

Active, not recruiting

Leber Hereditary Optic NeuropathyPreclinical

Unknown

Attention Deficit Hyperactivity DisorderPreclinical

Unknown

Cognitive ImpairmentPreclinical

Unknown

Friedreich AtaxiaPreclinical

Unknown

MigrainePhase 3

Unknown status

SynucleinopathiesPhase 2

Active, not recruiting

Competition

Competitive Landscape

105 competitors in Migraine

#DrugCompanyPhaseTrials

Zonegran

AAzurity Pharmaceuticals

Avicel

SSanthera Pharmaceuticals

Magnesium

SSpherium Biomed

Aspirin /d

TThe First Affiliated Hospital with Nanjing Medical University

Flunarizine

AAspartes Pharmaceuticals, Inc.

BAT7104

Tizanidine Hydrochloride

EElan Pharmaceuticals

Bayer Aspirin

Cervidil

Cymbalta

Jazz Pharmaceuticals

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2026

Phase 2Active, not recruiting

Synucleinopathies

NCT04534023

Clinical

Trials by Indication[1]

Ataxia8 trials

Phase 1 Trial of Idebenone to Treat Patients With Friedreich's Ataxia

Completed·NNational Institute of Neurological Disorders and Stroke (NINDS)·Phase 1·NCT00078481·16 enrolled

A Study of Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (FRDA) Patients

Completed·SSanthera Pharmaceuticals·Phase 3·NCT00905268·232 enrolled

Safety Study of Idebenone to Treat Friedreich's Ataxia

Completed·NNational Institute of Neurological Disorders and Stroke (NINDS)·Phase 1·NCT00015808·100 enrolled

Show 5 more trials

Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia

Completed·SSanthera Pharmaceuticals·Phase 3·NCT00537680·70 enrolled

Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients

Completed·SSanthera Pharmaceuticals·Phase 3·NCT00697073·68 enrolled

Long-Term Safety and Tolerability of Idebenone in Friedreich's Ataxia Patients (MICONOS Extension)

Completed·SSanthera Pharmaceuticals·Phase 3·NCT00993967·200 enrolled

Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone (PROTI)

Completed·SSanthera Pharmaceuticals·Phase 3·NCT01303406·29 enrolled

Idebenone to Treat Friedreich's Ataxia

Completed·NNational Institute of Neurological Disorders and Stroke (NINDS)·Phase 2·NCT00229632·51 enrolled

Duchenne Muscular Dystrophy6 trials

Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)

Completed·SSanthera Pharmaceuticals·Phase 3·NCT01027884·65 enrolled

Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD)

No longer available·SSanthera Pharmaceuticals··NCT03433807

Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)

Completed·SSanthera Pharmaceuticals·Phase 2·NCT00758225·21 enrolled

Show 3 more trials

A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids

Terminated·SSanthera Pharmaceuticals·Phase 3·NCT02814019·255 enrolled

Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)

Terminated·SSanthera Pharmaceuticals·Phase 3·NCT03603288·161 enrolled

Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy

Completed·SSanthera Pharmaceuticals·Phase 2·NCT00654784·21 enrolled

Neuropathy3 trials

Study to Assess Efficacy,Safety and Tolerability of Idebenone in the Treatment of Leber's Hereditary Optic Neuropathy

Completed·SSanthera Pharmaceuticals·Phase 2·NCT00747487·85 enrolled

Study With Idebenone in Patients With Chronic Vision Loss Due to Leber's Hereditary Optic Neuropathy (LHON)

Withdrawn·SSanthera Pharmaceuticals·Phase 3·NCT01495715

Expanded Access Program for Idebenone in Patients With Leber's Hereditary Optic Neuropathy Who Completed the LEROS Study

No longer available·SSanthera Pharmaceuticals··NCT04381091

Migraine2 trials

Idebenone for the Preventive Treatment of Migraine

Recruiting·SSecond Affiliated Hospital, School of Medicine, Zhejiang University·Phase 3·NCT04151472·180 enrolled

Safety and Efficacy of Oral Idebenone for Preventive Treatment of Migraine in Adult Migraine Patients

Unknown status·—·Phase 4·NCT05411978·900 enrolled

Primary Progressive Multiple Sclerosis2 trials

Idebenone for Primary Progressive Multiple Sclerosis

Completed·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 1/2·NCT01854359·61 enrolled

Clinical Trial of Idebenone in Primary Progressive Multiple Sclerosis (IPPoMS)

Completed·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 1/2·NCT00950248·85 enrolled

8 more indications

Parkinson disease1 trial

A Study of Efficacy and Safety of Idebenone Vs. Placebo in Prodromal Parkinson Disease

Withdrawn·SSecond Affiliated Hospital, School of Medicine, Zhejiang University·Phase 2/3·NCT04152655

Cognitive impairment1 trial

Study of the Therapeutic Effects of Naohuan Dan and Idebenone in Treating Mild Cognitive Impairment With Kidney Deficiency and Phlegm Stasis

Completed·SSun Yat-Sen Memorial Hospital of Sun Yat-Sen University··NCT05931029·64 enrolled

Efficacy and Safety1 trial

Study to Assess the Efficacy and Safety of Raxone in LHON Patients

Completed·SSanthera Pharmaceuticals·Phase 4·NCT02774005·199 enrolled

Lactic Acidosis1 trial

Study of Idebenone in the Treatment of Mitochondrial Encephalopathy Lactic Acidosis & Stroke-like Episodes

Completed·MMichio Hirano·Phase 2·NCT00887562·27 enrolled

Huntington's Disease1 trial

Symptomatic Therapy for Patients With Huntington's Disease

Unknown status·SSecond Affiliated Hospital, School of Medicine, Zhejiang University·Phase 1·NCT04071639·60 enrolled

Synucleinopathies1 trial

A Clinical Study of the Efficacy of Idebenone in the Treatment of iRBD Into Synucleinopathies

Active, not recruiting·RRuijin Hospital·Phase 2·NCT04534023·28 enrolled

Non-Alcoholic Steatohepatitis1 trial

Study of Oral Idebenone to Treat Non-Alcoholic Steatohepatitis

Completed·SStanford University·Phase 1/2·NCT04669158·45 enrolled

Unspecified1 trial

Post Authorisation Safety Study With Raxone in LHON Patients

Completed·SSanthera Pharmaceuticals··NCT02771379·229 enrolled

Sources

References

[1]
ClinicalTrials.gov — 29 trial records for Idebenone. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Idebenone compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Idebenone. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Santhera Pharmaceuticals company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 3[1]
Company: SSanthera Pharmaceuticals

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