idebenone / Generic mfg. - LARVOL DELTA

Real-world Study of Idebenone in the Treatment of Leber's Hereditary Optic Neuropathy (ChiCTR) - P4 | N=50 | Recruiting | Sponsor: Peking Union Medical College Hospital; Peking Union Medical College Hospital

New P4 trial • Real-world evidence • Inherited Retinal Dystrophy • Leber Hereditary Optic Neuropathy • Ocular Inflammation • Ophthalmology • Optic Neuritis

Physicochemical and safety evaluation of an antioxidant ophthalmic hydrogel for corneal injuries. (PubMed, Int J Pharm) - "This work describes the development of antioxidant ophthalmic hydrogels containing Idebenone (IDB) and hydroxocobalamin (HCB) as a complement treatment for corneal injuries, using hydroxypropyl- β-cyclodextrin (HPβCD) to improve IDB aqueous solubility. Additionally, C. Elegans survival assay confirmed the treatment́s safety. Overall, the developed hydrogel, composed of P407 and HPβCD, shows potential as a safe topical platform to increase ocular residence time and provide localized antioxidant support during corneal injuries management."

Journal • Corneal Abrasion • Infectious Disease • Inflammation • Ocular Inflammation • Ophthalmology • Pain

Risk Factors for Amisulpride-Associated Hyperprolactinemia: Gender-Specific Plasma Concentration Cutoffs Derived from ROC Analysis. (PubMed, Drug Des Devel Ther) - "Daily amisulpride dose was positively correlated with plasma drug concentration (p < 0.001); multivariable analysis identified sex, plasma amisulpride concentration, and concomitant idebenone or lorazepam use as independent risk factors for amisulpride-induced prolactin elevation (p < 0.05). Plasma amisulpride concentration cutoffs were higher in women (317.8 ng/mL) than in men (294.2 ng/mL), suggesting greater tolerance to higher plasma concentrations among women and confirming significant sex differences in susceptibility to amisulpride-induced prolactin elevation. Clinically, 28 ng/mL can be adopted as the screening cutoff value for elevated prolactin levels in patients treated with amisulpride, we advocate for sex-dependent plasma concentration management for personalized therapeutic drug monitoring to effectively prevent and control amisulpride-associated HPRL."

Journal • Retrospective data

Idebenone improves post-thaw motility and mitochondrial function of buck sperm. (PubMed, Anim Biosci) - "These results suggest that freezing extender supplemented with optimum level of ID (5 µM) can improve the post-thaw quality of buck sperm. The beneficial effects of ID5 appear to be associated with improved motility, acrosomal status, and mitochondrial function, indicating its potential as a useful additive for buck sperm cryoprocessing."

Journal • SOD2

Could NQO1 genotypes impact the results of clinical trials with idebenone? (PubMed, Brain) - No abstract available

Journal • NQO1

2000-2025: Emerging therapies in primary mitochondrial disease: A structured review, horizon scan, and roadmap for the future. (PubMed, Ther Adv Rare Dis) - "The European Medicines Agency (EMA) approved idebenone for Leber Hereditary Optic Neuropathy (LHON) in 2015 but only recently, in 2025 did the Food and Drug Administration (FDA) in the US approve a treatment for Barth syndrome and thymidine kinase 2 deficiency (TK2d)...Recent accelerated and full FDA approvals validate the feasibility of tailored evidence packages, but sustaining this momentum will require more rigorous alignment of trial design with molecular biology, strengthening of natural history infrastructure, deployment of sensitive biomarkers, and adoption of innovative statistical approaches. Early regulatory engagement and robust patient-community partnerships will be key."

Journal • Review • Ataxia • Friedreich ataxia • Gene Therapies • Inherited Retinal Dystrophy • Leber Hereditary Optic Neuropathy • Metabolic Disorders • Movement Disorders • Ocular Inflammation • Ophthalmology • Optic Neuritis • Rare Diseases

Late-onset Leber Hereditary Optic Neuropathy: disease triggers and prognostic factors in a large Italian cohort. (PubMed, Ophthalmology) - "Late-onset LHON represents a clinically relevant subset in which environmental and hormonal factors may contribute to disease conversion. In this retrospective cohort, high-dose idebenone treatment was associated with a higher probability of visual recovery, particularly among patients with a J or T haplogroup background. These exploratory findings should be considered hypothesis-generating and warrant confirmation in prospective studies."

Biomarker • Journal • Glaucoma • Inherited Retinal Dystrophy • Leber Hereditary Optic Neuropathy • Ocular Inflammation • Ophthalmology • Optic Neuritis • NQO1

Leber's hereditary optic nerve atrophy (PubMed, Vestn Oftalmol) - "The only drug currently approved for the treatment of LHON is the antioxidant, synthetic coenzyme Q10 analogue idebenone. Among alternative therapeutic strategies, gene therapy is at the most advanced stage of development."

Journal • Review • Gene Therapies • Inherited Retinal Dystrophy • Leber Hereditary Optic Neuropathy • Metabolic Disorders • Ocular Inflammation • Ophthalmology • Optic Neuritis • Pain

Development of Arginine Stearate-Based Solid Lipid Nanoparticles for Enhanced Idebenone Delivery: In Vitro Evaluation on Glioblastoma Model. (PubMed, Molecules) - "The rationale behind the development of arginine stearate-based SLNSs is to optimize the performance of pharmaceutical active ingredients, such as idebenone, versus non-tumorigenic cells. Overall, these findings support the potential of the developed SLNs as a promising delivery system for further in vitro and in vivo investigations."

Journal • Preclinical • Brain Cancer • Glioblastoma • Oncology • Solid Tumor

Discovery of a novel idebenone-rhein hybrid 4c with enhanced NQO1 expression for neuroprotection against cerebral ischemia-reperfusion injury. (PubMed, Eur J Med Chem) - "In addition, 4c showed favorable in vitro PAMPA-BBB permeability, and H&E staining of major organs showed no obvious short-term histopathological abnormalities under the present experimental conditions. Collectively, these findings identify 4c as a promising idebenone-rhein conjugate with neuroprotective activity, which may involve NQO1 expression modulation and additional mitochondrial protective mechanisms, highlighting its potential value for further investigation in ischemic stroke and other oxidative stress-related neurological disorders."

Journal • Cardiovascular • CNS Disorders • Ischemic stroke • Reperfusion Injury • NQO1

CLRN1 Variants in Müller Cells Cause Mitochondrial Dysfunction in USH3A Retinal Organoids. (PubMed, CNS Neurosci Ther) - "CLRN1-related mitochondrial impairment in Müller cells contributes to the pathogenesis of retinitis pigmentosa in USH3A. These findings identify Müller cell mitochondrial dysfunction as a key disease mechanism and highlight potential therapeutic targets."

Journal • Genetic Disorders • Inherited Retinal Dystrophy • Metabolic Disorders • Ocular Inflammation • Ophthalmology • Otorhinolaryngology • Retinal Disorders • Retinitis Pigmentosa

Systematic review of Leber's hereditary optic neuropathy - Clinical diagnosis, genetics overview and current concepts of treatment. (PubMed, Indian J Ophthalmol) - "Idebenone, a synthetic CoQ10 analog, is the first pharmacologic agent approved in Europe, demonstrating partial visual recovery in patients treated early by improving mitochondrial electron transport and reducing oxidative stress. Gene therapy using allotopic expression of ND4 via adeno-associated viral vectors (rAAV2/2-ND4) has shown improvement in both eyes even after unilateral injection...High-throughput genetic testing, including whole-genome sequencing and clinical exome analysis, enables precise identification of nuclear modifiers that influence LHON phenotypes, facilitating early diagnosis and intervention. Current clinical trials, including RESTORE and REFLECT, emphasize the importance of prompt treatment to optimize visual outcomes."

Journal • Review • Gene Therapies • Inherited Retinal Dystrophy • Leber Hereditary Optic Neuropathy • Metabolic Disorders • Ocular Inflammation • Ophthalmology • Optic Neuritis • Pain

Mitochondrial Genetic Diseases and Ophthalmic Manifestations: Molecular Pathophysiology, Genetics, and Clinical Management. (PubMed, Int J Mol Sci) - "Recurring themes are heteroplasmy, the threshold effect, and the selective vulnerability of RGCs and extraocular muscle across genetically distinct disorders. Treatment remains largely supportive, but idebenone, gene therapy, mitophagy modulation, and targeted antioxidants now offer mechanism-based intervention for several ophthalmic manifestations."

Journal • Review • Gene Therapies • Genetic Disorders • Metabolic Disorders • Ophthalmology • Retinal Disorders

A Single-Arm Study on the Efficacy and Safety of Idebenone in the Treatment of Multiple System Atrophy (ChiCTR) - P=N/A | N=50 | Not yet recruiting | Sponsor: Qilu Hospital of Shandong University; Qilu Hospital of Shandong University

New trial • CNS Disorders • Movement Disorders • Multiple System Atrophy • Parkinson's Disease

When LHON Mimics Demyelination: Area Postrema Syndrome in Biallelic DNAJC30 Variants. (PubMed, J Clin Med) - "After initiation of idebenone therapy, the patient showed significant improvement in visual function, normalization of lactate levels, and complete resolution of the brainstem lesions on follow-up MRI. This case further expands the neuro-ophthalmic spectrum associated with DNAJC30 variants and suggests that DNAJC30-related disease may closely mimic seronegative NMOSD. We highlight that early genetic diagnosis is essential, as recognition of this mitochondrial etiology enables targeted therapy and may substantially improve clinical outcomes."

Journal • CNS Disorders • Inherited Retinal Dystrophy • Leber Hereditary Optic Neuropathy • Metabolic Disorders • Neuromyelitis Optica Spectrum Disorder • Ocular Inflammation • Ophthalmology • Optic Neuritis • Otorhinolaryngology • Rare Diseases • Vertigo

Idebenone reduces oxidative stress and differentially improves chromatin integrity in dog and cat epididymal spermatozoa after slow freezing and vitrification. (PubMed, Vet Res Commun) - "However, improved chromatin integrity in cats was only observed in the SF + IDB treatment compared with SF-Control (P < 0.05). In conclusion, idebenone reduced oxidative stress in dog and cat epididymal spermatozoa, while improved chromatin integrity was observed in dogs after both cryopreservation methods and in cats only after slow freezing."

Journal

Idebenone attenuates sciatic nerve ligation-induced neuropathic pain through modulating antioxidant and anti-inflammatory mechanisms in rats. (PubMed, Inflammopharmacology) - "This study aimed to investigate the therapeutic potential of idebenone, a compound with potent antioxidant and anti-inflammatory properties, in a rat model of neuropathic pain induced by chronic constriction injury (CCI) of the sciatic nerve, and to elucidate its underlying mechanisms through biochemical, electrophysiological, and histopathological analyses.A total of 42 male Sprague-Dawley rats were randomly allocated into six groups: Sham, CCI, idebenone 50 mg/kg, idebenone 100 mg/kg, idebenone 200 mg/kg, and pregabalin (10 mg/kg)...Electrophysiologically, idebenone enhanced compound muscle action potential amplitude without significantly affecting latency. Histopathological findings indicated that idebenone did not significantly alter the number of myelinated axons but was unable to prevent the CCI-induced reduction in myelinated axon area. Overall, idebenone exhibited antioxidant, anti-inflammatory, and neuroprotective effects, as evidenced by the..."

Journal • Preclinical • Inflammation • Neuralgia • Oncology • Pain • IL10 • TNFA

When LHON Mimics Demyelination: Area Postrema Syndrome in Biallelic DNAJC30 Variant (EAN 2026) - "DNAJC30-associated disease may mimic neuromyelitis optica spectrum disorder (NMOSD). We highlight the need of early genetic diagnosis in patients with optic neuropathy and APS who do not respond to immunotherapy, as targeted treatment with idebenone may lead to significant clinical and radiological improvement."

CNS Disorders • Inherited Retinal Dystrophy • Leber Hereditary Optic Neuropathy • Neuromyelitis Optica Spectrum Disorder • Ocular Inflammation • Ophthalmology • Optic Neuritis • Otorhinolaryngology • Rare Diseases • Vertigo

A Multicenter Randomized Trial Of Idebenone Augmentation For Post-stroke Depression And Cognitive Recovery (AAIC 2026) - No abstract available

Clinical • Cardiovascular • CNS Disorders • Depression • Mood Disorders • Psychiatry

Idebenone Loaded Plga Based Nanoparticles As Antioxidant In AD (AAIC 2026) - No abstract available

Clinical development in primary mitochondrial diseases at a translational inflection point: Lessons, mechanisms, and emerging therapeutic strategies. (PubMed, Biomed Pharmacother) - "Four therapies have achieved regulatory authorization, including idebenone for Leber hereditary optic neuropathy, taurine for MELAS, and recent FDA approvals of doxecitine and doxribtimine (Kygevvi) for thymidine kinase 2 deficiency and elamipretide (FORZINITY) for Barth syndrome. Emerging strategies include NAD⁺ augmentation, soluble guanylate cyclase stimulation, mTOR modulation, gene therapies, and heteroplasmy-targeting approaches. Collectively, these advances mark an emerging inflection point and suggest a path toward greater regulatory success in the coming decade."

Journal • Review • Gene Therapies • Inherited Retinal Dystrophy • Leber Hereditary Optic Neuropathy • Metabolic Disorders • Ocular Inflammation • Ophthalmology • Optic Neuritis

Design, synthesis, and biological evaluation of a multi-layer-linked idebenone derivative targeting mitochondrial dysfunction and neuroinflammation for ischemic stroke. (PubMed, Bioorg Chem) - "Multi-omics analyses further revealed that compound 8 attenuates neuroinflammation, in part, by inhibiting the SerpinA3N/NF-κB signaling axis and suppressing astrocyte activation. These findings demonstrate that multi-layer molecular engineering of idebenone enhances its pharmacological profile and represents a promising strategy for developing neuroprotective agents targeting mitochondrial dysfunction and neuroinflammation."

Journal • Cardiovascular • Inflammation • Ischemic stroke • Metabolic Disorders • Reperfusion Injury • SERPINA3

Idebenone in Rapid Eye Movement Sleep Behavioral Disorder for Parkinson's Disease Prevention. (PubMed, Mov Disord) - No abstract available

Journal • CNS Disorders • Mental Retardation • Movement Disorders • Parkinson's Disease • Psychiatry • Sleep Disorder

Reply to: "Idebenone in Rapid Eye Movement Sleep Behavioral Disorder for Parkinson's Disease Prevention". (PubMed, Mov Disord) - No abstract available

Journal • CNS Disorders • Mental Retardation • Movement Disorders • Parkinson's Disease • Psychiatry

Leber hereditary optic neuropathy (LHON) in a 6-year-old boy with a transient spinal cord lesion. (PubMed, Turk J Pediatr) - "Idebenone therapy was initiated, and follow-up was scheduled...The presence of a transient and asymptomatic spinal cord lesion in this patient expands the recognized spectrum of central nervous system involvement in LHON. Therefore, LHON-plus syndrome should be considered when spinal cord involvement accompanies optic neuropathy after excluding other demyelinating diseases."

Journal • Inherited Retinal Dystrophy • Leber Hereditary Optic Neuropathy • Metabolic Disorders • Ocular Inflammation • Ophthalmology • Optic Neuritis • Pediatrics