Raxone: Uses, Dosage & Side Effects
A synthetic coenzyme Q10 analogue for the treatment of visual impairment in Leber’s hereditary optic neuropathy (LHON) in adults and adolescents
Raxone (idebenone) is a prescription medication used to treat visual impairment in adults and adolescents aged 12 years and older with Leber’s hereditary optic neuropathy (LHON), a rare inherited mitochondrial disorder that causes progressive vision loss. Idebenone is a short-chain synthetic analogue of coenzyme Q10 (ubiquinone) that acts as an electron carrier in the mitochondrial electron transport chain. By bypassing the dysfunctional complex I found in LHON patients, idebenone helps restore cellular energy (ATP) production in retinal ganglion cells, potentially reactivating dormant cells and improving visual function. Raxone is taken as two tablets three times daily (900 mg total daily dose) with food, and has been approved by the European Medicines Agency under exceptional circumstances as an orphan drug for this ultra-rare condition.
Quick Facts: Raxone
Key Takeaways
- Raxone (idebenone) is the first and only approved treatment specifically indicated for visual impairment caused by Leber’s hereditary optic neuropathy (LHON), a rare mitochondrial disease affecting the optic nerve.
- The recommended dose is 900 mg per day (two 150 mg tablets, three times daily), always taken with food to significantly increase absorption and bioavailability.
- Idebenone works by bypassing dysfunctional mitochondrial complex I, restoring energy production in retinal ganglion cells and potentially recovering lost vision, particularly in patients with early-stage disease.
- Raxone has been approved under exceptional circumstances by the EMA as an orphan drug, meaning its long-term data is limited due to the extreme rarity of LHON, with annual reviews of new evidence.
- Common side effects include nasopharyngitis and cough; the medication may cause harmless reddish-brown discoloration of urine, which should be reported to your doctor to rule out other causes.
What Is Raxone and What Is It Used For?
Raxone contains the active substance idebenone, a short-chain synthetic benzoquinone that is structurally related to ubiquinone (coenzyme Q10), a naturally occurring molecule found in virtually every cell in the human body. Coenzyme Q10 plays a critical role in the mitochondrial electron transport chain, the cellular machinery responsible for producing adenosine triphosphate (ATP), the primary energy currency of the cell. Idebenone was specifically developed to mimic and enhance certain functions of coenzyme Q10, particularly its ability to transfer electrons within the mitochondrial respiratory chain.
Raxone is indicated for the treatment of visual impairment in adults and adolescents aged 12 years and older with Leber’s hereditary optic neuropathy (LHON). LHON is a rare inherited mitochondrial disorder that primarily affects the retinal ganglion cells in the eye, the specialized neurons responsible for transmitting visual information from the retina to the brain via the optic nerve. LHON is classified as a rare disease, with an estimated prevalence of approximately 1 in 30,000 to 1 in 50,000 people worldwide. The condition predominantly affects young men, with the typical age of onset between 15 and 35 years, although it can occur at any age in both sexes.
LHON is caused by point mutations in mitochondrial DNA (mtDNA) that disrupt the function of complex I (NADH:ubiquinone oxidoreductase), the first and largest enzyme complex in the mitochondrial electron transport chain. Three primary mutations account for approximately 90–95% of all LHON cases worldwide: m.11778G>A (affecting the ND4 subunit, responsible for approximately 70% of cases), m.3460G>A (affecting ND1, approximately 13% of cases), and m.14484T>C (affecting ND6, approximately 14% of cases). These mutations impair the ability of complex I to efficiently transfer electrons from NADH to ubiquinone, reducing the electrochemical gradient that drives ATP synthesis and increasing the production of reactive oxygen species (ROS) that damage cellular components.
The retinal ganglion cells are particularly vulnerable to mitochondrial dysfunction because of their exceptionally high energy demands. The unmyelinated portion of their axons within the retinal nerve fiber layer requires enormous amounts of ATP to maintain the ionic gradients necessary for electrical signal conduction. When complex I function is compromised, these cells cannot generate sufficient energy, leading to cellular dysfunction, axonal swelling, and eventually cell death. Clinically, this manifests as painless, subacute bilateral central vision loss that typically begins in one eye and involves the second eye within weeks to months. Patients often experience a dense central scotoma (blind spot) with relatively preserved peripheral vision.
Idebenone addresses this pathology by functioning as an alternative electron carrier in the mitochondrial respiratory chain. Unlike natural coenzyme Q10, idebenone has a shorter, more polar side chain that enhances its ability to shuttle electrons directly from cytosolic NADH (via the enzyme NQO1, cytosolic NAD(P)H:quinone oxidoreductase 1) to complex III, effectively bypassing the dysfunctional complex I. This bypass mechanism restores electron flow through the remainder of the respiratory chain (complexes III and IV), regenerating the proton gradient across the inner mitochondrial membrane and enabling ATP synthase (complex V) to resume ATP production. Additionally, idebenone possesses antioxidant properties that help neutralize the excess reactive oxygen species generated by the defective complex I, providing further protection to vulnerable retinal ganglion cells.
The pivotal clinical evidence for Raxone in LHON comes from the RHODOS trial (Rescue of Hereditary Optic Disease Outpatient Study), a randomized, double-blind, placebo-controlled study that evaluated idebenone 900 mg/day in 85 patients with LHON. While the primary endpoint (change in best visual acuity in the best-seeing eye) did not reach statistical significance in the overall population, post-hoc analyses and subgroup analyses demonstrated clinically meaningful benefits. Patients with discordant visual acuity (those whose two eyes were at different stages of disease) showed significant improvement, and a higher proportion of idebenone-treated patients experienced clinically relevant recovery of visual acuity compared with placebo. An expanded access program and real-world registry data have further supported the efficacy of idebenone, particularly when treatment is initiated early in the disease course, before irreversible retinal ganglion cell death has occurred.
Raxone has been approved by the European Medicines Agency (EMA) under “exceptional circumstances.” This means that because LHON is an extremely rare disease, it has not been possible to obtain comprehensive clinical data under normal conditions. The EMA reviews any new information that becomes available each year and updates the product information as necessary. This designation does not affect the quality, safety, or efficacy standards required for approval but reflects the inherent challenges of conducting large-scale clinical trials in ultra-rare diseases.
What Should You Know Before Taking Raxone?
Contraindications
The primary contraindication to Raxone use is hypersensitivity (allergy) to idebenone or to any of the other ingredients in the formulation. The excipients in Raxone include lactose monohydrate, microcrystalline cellulose, croscarmellose sodium, povidone K25, magnesium stearate, and colloidal anhydrous silica in the tablet core, plus macrogol, poly(vinyl alcohol), talc, titanium dioxide, and sunset yellow FCF (E110) in the film coating. Patients with known allergies to any of these substances should not take Raxone.
Patients with known lactose intolerance should consult their doctor before taking Raxone, as the tablets contain lactose monohydrate. Additionally, the film coating contains sunset yellow FCF (also known as E110), an azo dye that may cause allergic reactions in susceptible individuals, particularly those with a history of aspirin sensitivity or asthma. If you have experienced allergic reactions to food colorings or azo dyes in the past, inform your healthcare provider before starting Raxone.
Warnings and Precautions
Raxone can cause your urine to turn reddish-brown. This color change is harmless and does not mean your treatment needs to be changed. However, it could potentially mask other urinary conditions. Always tell your doctor if your urine changes color so they can perform appropriate tests to ensure the discoloration is not hiding a kidney or bladder problem.
Before starting Raxone, discuss the following with your healthcare provider:
- Blood disorders: If you have any existing blood conditions, inform your doctor. Idebenone has been associated with changes in blood test results, including low levels of white blood cells (leukopenia), low levels of red blood cells (anemia), and low levels of platelets (thrombocytopenia) in post-marketing reports. Your doctor may wish to monitor your blood counts periodically during treatment.
- Liver problems: If you have a history of liver disease or currently have liver problems, tell your doctor. Elevated liver enzymes and bilirubin levels have been reported with idebenone use. In rare cases, hepatitis has been observed. Your doctor may perform liver function tests before and during treatment to monitor for any liver-related effects.
- Kidney problems: If you have kidney disease, consult your doctor. The reddish-brown urine discoloration caused by idebenone can potentially mask signs of kidney or urinary tract problems. Your doctor may need to perform urine tests to ensure proper kidney function.
Your doctor will check your vision before you start taking Raxone and at regular follow-up visits during treatment. These eye examinations are essential for monitoring the response to treatment and assessing the progression of LHON. Visual acuity testing, visual field assessment, and optical coherence tomography (OCT) of the retinal nerve fiber layer may be performed as part of routine monitoring.
Children and Adolescents
Raxone is approved for use in adults and adolescents aged 12 years and older. The medication should not be given to children under 12 years of age because the safety and efficacy of idebenone have not been established in this younger age group. There is insufficient clinical data to support dosing recommendations or to characterize the safety profile in children under 12. If a child under 12 has been diagnosed with LHON, the treating physician should seek specialist advice from a mitochondrial disease center and consider alternative management strategies, including genetic counseling and supportive care.
Pregnancy and Breastfeeding
If you are pregnant, think you may be pregnant, or are planning to become pregnant, consult your doctor before using Raxone. Your doctor will only prescribe Raxone if the benefit of treatment outweighs the potential risk to the unborn baby. The effects of idebenone on human pregnancy have not been adequately studied, and as a precaution, Raxone should be avoided during pregnancy unless clearly necessary. Women of childbearing potential should discuss contraception with their doctor during treatment.
Idebenone may pass into breast milk. If you are breastfeeding, your doctor will discuss with you whether you should stop breastfeeding or stop taking the medication. This decision should weigh the benefit of breastfeeding for the infant against the benefit of Raxone treatment for the mother. Until more data are available, a cautious approach is recommended.
Driving and Operating Machinery
Raxone is not expected to affect your ability to drive or operate machinery based on its pharmacological properties and known side effect profile. However, it is important to remember that patients taking Raxone have LHON, which itself causes significant visual impairment that may affect the ability to drive safely. Your fitness to drive should be assessed by your doctor based on your visual function, regardless of medication use.
How Does Raxone Interact with Other Drugs?
Unlike monoclonal antibody therapies which generally have few drug interactions, idebenone is a small molecule that is subject to cytochrome P450 (CYP)-mediated metabolism and can therefore interact with other drugs that are substrates, inhibitors, or inducers of these enzymes. Because idebenone undergoes extensive hepatic metabolism, the potential for pharmacokinetic drug-drug interactions is a clinically important consideration. Patients taking Raxone should inform their doctor about all medications, herbal supplements, and over-the-counter products they are currently using.
The following table summarizes the key drug interactions identified for idebenone:
| Drug Category | Examples | Interaction Type | Clinical Significance |
|---|---|---|---|
| Antihistamines | Astemizole, terfenadine | CYP enzyme interaction | Potential for cardiac arrhythmias; avoid co-administration |
| Prokinetic agents | Cisapride | CYP enzyme interaction | Risk of QT prolongation; avoid co-administration |
| Antipsychotics | Pimozide | CYP enzyme interaction | Risk of cardiac arrhythmias; avoid co-administration |
| Antiarrhythmics | Quinidine | CYP enzyme interaction | Altered drug levels; monitor closely |
| Ergot alkaloids | Dihydroergotamine, ergotamine | CYP enzyme interaction | Risk of ergot toxicity (vasospasm); avoid co-administration |
| Anesthetic agents | Alfentanil | CYP3A4 interaction | Altered sedation; inform anesthesiologist |
| Immunosuppressants | Ciclosporin, sirolimus, tacrolimus | CYP3A4 interaction | Altered immunosuppressant levels; monitor drug levels |
| Opioid analgesics | Fentanyl | CYP3A4 interaction | Altered opioid levels; dose adjustment may be needed |
Many of the identified interactions involve drugs that are substrates of cytochrome P450 3A4 (CYP3A4), one of the most important drug-metabolizing enzymes in the human body. Idebenone may affect the metabolism of CYP3A4 substrates, potentially leading to increased or decreased blood levels of these medications. This is particularly concerning for drugs with narrow therapeutic indices, where small changes in blood concentration can lead to either toxicity or loss of efficacy.
The interactions with astemizole, terfenadine, cisapride, and pimozide are especially important because elevated blood levels of these drugs can cause prolongation of the QT interval on the electrocardiogram, a condition that predisposes to potentially life-threatening cardiac arrhythmias, including torsades de pointes. Although astemizole, terfenadine, and cisapride have been withdrawn from the market in many countries due to cardiac safety concerns, they may still be available in some regions.
If you are scheduled for surgery or a medical procedure requiring anesthesia, inform your anesthesiologist that you are taking Raxone, as the interaction with alfentanil and potentially other opioid anesthetics could affect dosing requirements. Similarly, if you are taking immunosuppressant medications (such as ciclosporin, sirolimus, or tacrolimus following organ transplantation or for autoimmune conditions), your doctor may need to monitor drug levels more frequently and adjust doses accordingly.
Always tell your doctor, pharmacist, or other healthcare provider about all medications you are taking, including prescription drugs, over-the-counter medications, herbal supplements, and vitamins. Drug interactions can change how medications work and may increase the risk of serious side effects. Never start or stop a medication without consulting your doctor first.
What Is the Correct Dosage of Raxone?
Raxone should always be used exactly as your doctor or pharmacist has instructed. The medication is supplied as orange, round, film-coated tablets, each containing 150 mg of idebenone, marked with “150” on one side. The tablets are packaged in white plastic bottles, each containing 180 tablets. If you are unsure about how to take your medication, ask your doctor or pharmacist for clarification.
Adults and Adolescents (12 Years and Older)
The recommended dose of Raxone is the same for all eligible patients:
| Time of Day | Dose | Number of Tablets | Instructions |
|---|---|---|---|
| Morning (with breakfast) | 300 mg | 2 tablets | Take with food |
| Midday (with lunch) | 300 mg | 2 tablets | Take with food |
| Evening (with dinner) | 300 mg | 2 tablets | Take with food |
| Total Daily Dose | 900 mg | 6 tablets | Divided into 3 doses |
Taking Raxone with food is critically important. Food significantly increases the absorption of idebenone from the gastrointestinal tract, leading to substantially higher blood levels compared with taking the medication on an empty stomach. This enhanced absorption ensures that sufficient idebenone reaches the target tissues (particularly the retinal ganglion cells) to exert its therapeutic effect. A meal containing some fat is particularly effective at improving absorption.
Swallow the tablets whole with a glass of liquid. Do not crush, break, or chew the tablets, as this may alter the release characteristics of the medication and could affect its taste (idebenone has a bitter taste). Taking the tablets at consistent times each day helps maintain steady blood levels and makes it easier to remember your doses.
Children Under 12 Years
Raxone is not recommended for children under 12 years of age. The safety and efficacy of idebenone have not been established in this age group, and there are currently no clinical data to support dosing recommendations for younger children. If a child under 12 has LHON, treatment decisions should be made by a specialist with experience in mitochondrial diseases.
Elderly Patients
There is limited clinical experience with Raxone in elderly patients, as LHON typically manifests in younger individuals. No specific dose adjustment has been established for the elderly. However, as liver and kidney function may decline with age, elderly patients should be monitored carefully during treatment, and dose adjustments may be considered on a case-by-case basis by the treating physician.
Missed Dose
If you forget to take a dose, do not take the missed dose. Simply take your next dose at the usual time. Do not take a double dose to make up for a missed one. Taking double doses could increase the risk of side effects without providing additional therapeutic benefit. If you frequently forget to take your medication, consider using a pill organizer, setting reminders on your phone, or associating each dose with a specific mealtime to help establish a consistent routine.
Overdose
If you have taken too much Raxone, contact your doctor immediately or go to the nearest emergency department. Bring the medication container with you so that healthcare professionals can identify what you have taken. While specific data on idebenone overdose in humans are limited, treatment would be supportive, focusing on monitoring vital signs and managing any symptoms that may arise. There is no specific antidote for idebenone overdose.
Stopping Treatment
Do not stop taking Raxone without first consulting your doctor. Discontinuation of treatment may lead to a recurrence or worsening of visual impairment. Your doctor will advise you on the appropriate duration of treatment based on your individual response and the progression of your condition. In general, treatment with Raxone is intended to be long-term, as ongoing mitochondrial support may be needed to maintain any visual benefits achieved.
The bioavailability of idebenone is dramatically increased when taken with food. In pharmacokinetic studies, food enhanced the absorption of idebenone by up to 5–7 fold compared with the fasted state. Always take your Raxone tablets during or immediately after a meal to ensure you get the full benefit of the medication. A meal containing some dietary fat further optimizes absorption.
What Are the Side Effects of Raxone?
Like all medicines, Raxone can cause side effects, although not everyone who takes it will experience them. The side effect profile of idebenone has been characterized through clinical trials, expanded access programs, and post-marketing surveillance. Because LHON is an extremely rare disease, the clinical trial database is relatively small compared with medications for more common conditions, and some side effects have been identified only through post-marketing reports.
The following side effects have been reported with Raxone, organized by frequency:
Very Common
May affect more than 1 in 10 people
- Nasopharyngitis (common cold symptoms)
- Cough
Common
May affect up to 1 in 10 people
- Diarrhea (usually mild to moderate; typically does not require discontinuation)
- Back pain
Frequency Not Known
Cannot be estimated from available data
- Bronchitis
- Changes in blood test results: low white blood cell count (leukopenia), low red blood cell count (anemia), or low platelet count (thrombocytopenia)
- Elevated cholesterol or lipid levels (shown in blood tests)
- Seizures, confusion, hallucinations (seeing or hearing things that are not real), agitation, uncontrolled movements, tendency to wander, dizziness, headache, restlessness
- Nausea, vomiting, loss of appetite, indigestion
- Elevated liver enzymes (shown in blood tests), indicating potential liver problems
- Elevated bilirubin levels, which may cause yellowing of the skin and whites of the eyes (jaundice)
- Hepatitis (inflammation of the liver)
- Skin rash, itching
- Pain in the arms or legs
- Elevated blood nitrogen levels (shown in blood tests)
- Change in urine color (reddish-brown; harmless)
- General feeling of being unwell (malaise)
The most frequently reported side effects in clinical trials were upper respiratory tract infections (nasopharyngitis) and cough. These are common in the general population and may not necessarily be directly caused by the medication. The diarrhea observed with Raxone is typically mild to moderate in severity and rarely requires discontinuation of treatment. It may improve over time as the body adjusts to the medication.
The reddish-brown discoloration of urine is a characteristic effect of idebenone and its metabolites being excreted through the kidneys. This color change is entirely harmless and is not an indication of blood in the urine or kidney damage. However, because the discoloration could theoretically mask the detection of hematuria (blood in the urine) or other urinary abnormalities, patients should inform their doctor about this effect so that appropriate laboratory tests can be performed if needed.
The neurological side effects listed in the “frequency not known” category (seizures, confusion, hallucinations, agitation) have been reported in post-marketing surveillance. These events are uncommon and may occur more frequently in patients with pre-existing neurological conditions or in those taking concomitant medications that affect the central nervous system. If you experience any neurological symptoms while taking Raxone, contact your doctor immediately.
Liver-related side effects, including elevated liver enzymes, elevated bilirubin, and hepatitis, have also been reported. Your doctor may recommend periodic liver function tests during treatment, particularly if you have a history of liver disease or are taking other medications that can affect the liver. Signs of liver problems include unexplained tiredness, loss of appetite, nausea, yellowing of the skin or eyes, dark urine, or abdominal pain. If you notice any of these symptoms, contact your doctor promptly.
Contact your doctor immediately if you experience: severe diarrhea that does not improve, signs of a severe allergic reaction (difficulty breathing, swelling of face or throat, severe rash), yellowing of the skin or eyes, seizures, confusion, or hallucinations. Report any unusual or concerning symptoms to your healthcare provider, even if they are not listed above.
How Should You Store Raxone?
Proper storage of Raxone is essential to maintain the quality, safety, and efficacy of the medication throughout its shelf life. Unlike some biological medications that require refrigeration, Raxone tablets are stable at standard room temperature conditions, making storage relatively straightforward.
Follow these storage guidelines:
- Keep out of reach and sight of children: Store Raxone in a safe location where children cannot access it. The orange tablets could be mistaken for candy by young children.
- Check expiration date: Do not use Raxone after the expiration date printed on the bottle and outer carton after “EXP.” The expiration date refers to the last day of the stated month. Using expired medication may result in reduced efficacy or altered safety.
- Room temperature storage: No special storage conditions are required. Store at room temperature, away from excessive heat, moisture, and direct sunlight.
- Keep in original container: Keep the tablets in the original white plastic bottle to protect them from light and moisture.
- Proper disposal: Do not dispose of Raxone in household waste or pour unused medication down the drain. Ask your pharmacist about proper disposal methods for medications that are no longer needed. Proper disposal helps protect the environment.
When traveling with Raxone, keep the medication in your hand luggage in its original container. Carry a copy of your prescription or a letter from your doctor to facilitate customs and security checks. Avoid leaving the medication in a car where temperatures can become extreme, or in checked luggage where it may be subject to very low temperatures in the aircraft cargo hold.
What Does Raxone Contain?
Understanding the complete composition of your medication is important, particularly if you have known allergies or sensitivities to specific pharmaceutical ingredients. Below is a detailed breakdown of the ingredients in Raxone.
Active Ingredient
The active substance is idebenone (2-(10-hydroxydecyl)-5,6-dimethoxy-3-methyl-2,5-cyclohexadiene-1,4-dione), a synthetic short-chain benzoquinone structurally related to coenzyme Q10. Each film-coated tablet contains exactly 150 mg of idebenone.
Inactive Ingredients (Excipients)
| Ingredient | Component | Notes |
|---|---|---|
| Idebenone | Active substance | 150 mg per tablet |
| Lactose monohydrate | Tablet core (filler) | Caution in lactose intolerance |
| Microcrystalline cellulose | Tablet core (filler/binder) | Provides tablet structure |
| Croscarmellose sodium | Tablet core (disintegrant) | Aids tablet dissolution |
| Povidone K25 | Tablet core (binder) | Holds tablet together |
| Magnesium stearate | Tablet core (lubricant) | Prevents sticking during manufacturing |
| Colloidal anhydrous silica | Tablet core (glidant) | Improves powder flow |
| Macrogol | Film coating | Coating agent |
| Poly(vinyl alcohol) | Film coating | Film-forming agent |
| Talc | Film coating | Anti-tacking agent |
| Titanium dioxide | Film coating (opacifier) | Provides white base color |
| Sunset yellow FCF (E110) | Film coating (colorant) | May cause allergic reactions |
Appearance and Pack Sizes
Raxone film-coated tablets are orange, round, 10 mm in diameter, and marked with “150” on one side. The tablets are supplied in white plastic bottles, each containing 180 tablets. Each bottle provides a 30-day supply at the recommended dose of 6 tablets per day.
Marketing Authorization Holder and Manufacturer
Raxone is manufactured by Excella GmbH & Co. KG (Nürnberger Strasse 12, 90537 Feucht, Germany) and marketed by Chiesi Farmaceutici S.p.A. (Via Palermo 26/A, 43122 Parma, Italy). The marketing authorization holder for the European Union is Chiesi Farmaceutici S.p.A. Further information about Raxone is available on the European Medicines Agency (EMA) website, which also provides links to information about rare diseases and their treatments.
Frequently Asked Questions About Raxone
Leber’s hereditary optic neuropathy (LHON) is a rare inherited mitochondrial disorder that causes progressive, painless vision loss. It is caused by mutations in mitochondrial DNA that impair the function of complex I in the mitochondrial electron transport chain. This leads to energy failure in retinal ganglion cells, the nerve cells in the eye responsible for transmitting visual information to the brain. LHON typically affects young men between ages 15 and 35, though it can occur at any age in both sexes. Vision loss usually starts in one eye and involves the second eye within weeks to months, resulting in a central blind spot (scotoma) with relatively preserved peripheral vision. The condition is inherited maternally, meaning it is passed from mother to children.
The duration of Raxone treatment should be determined by your doctor based on your individual response and disease progression. In general, treatment is intended to be long-term, as ongoing mitochondrial support may be necessary to maintain any visual improvements achieved. In clinical trials, patients were treated for 6 months, and real-world data suggest that longer treatment durations may be associated with better outcomes. Do not stop taking Raxone without consulting your doctor, as this could lead to worsening of your visual condition. Your doctor will schedule regular eye examinations to monitor your response and determine the optimal treatment duration.
Taking Raxone with food dramatically increases the absorption of idebenone from your digestive tract into your bloodstream. Studies have shown that food can increase the bioavailability of idebenone by up to 5–7 times compared with taking it on an empty stomach. This means that significantly more of the medication reaches your retinal ganglion cells where it is needed. A meal containing some fat is particularly effective. Without adequate absorption, the medication may not achieve sufficient concentrations to provide therapeutic benefit. This is why it is so important to take each of your three daily doses with a meal.
No, Raxone (idebenone) is not the same as over-the-counter coenzyme Q10 (CoQ10) supplements. While idebenone is structurally related to CoQ10 and shares some functional properties, there are important differences. Idebenone has a shorter, more polar side chain that gives it distinct pharmacological properties, including better ability to shuttle electrons from cytosolic NADH to complex III via the NQO1 enzyme, effectively bypassing the defective complex I in LHON patients. CoQ10 supplements have not been shown to be effective in treating LHON, and they should not be used as a substitute for Raxone. Raxone is a rigorously tested pharmaceutical product manufactured to strict quality standards, with defined purity and potency that cannot be guaranteed with dietary supplements.
Evidence suggests that starting Raxone treatment as early as possible after the onset of LHON symptoms provides the best chance of visual recovery. This is because idebenone works by restoring energy production in retinal ganglion cells that have become dysfunctional but have not yet died. Once these cells undergo irreversible death, the opportunity for recovery is lost. Real-world data from the LHON registry indicate that patients treated within the first year of symptom onset are more likely to experience clinically relevant improvements in visual acuity. If you or a family member is diagnosed with LHON, consult a specialist as soon as possible to discuss treatment options including Raxone.
Gene therapy is an emerging treatment approach for LHON, with some products (such as lenadogene nolparvovec) receiving approval in certain regions. The question of whether Raxone can be used alongside gene therapy is an area of active clinical interest. Since the two treatments work through different mechanisms – idebenone provides a biochemical bypass of complex I dysfunction while gene therapy aims to introduce a functional copy of the affected gene – there is a theoretical rationale for combination therapy. However, the clinical evidence for combined use is still very limited. Decisions about using Raxone together with gene therapy should be made by a specialist in mitochondrial diseases, taking into account the individual patient’s situation, the timing of treatments, and available evidence.
References
- European Medicines Agency (EMA). Raxone (idebenone) – Summary of Product Characteristics. Last updated 2025. Available at: EMA Raxone EPAR.
- Klopstock T, Yu-Wai-Man P, Dimitriadis K, et al. A Randomized Placebo-Controlled Trial of Idebenone in Leber’s Hereditary Optic Neuropathy (RHODOS). Brain. 2011;134(Pt 9):2677–2686. doi:10.1093/brain/awr170.
- Carelli V, Carbonelli M, de Coo IF, et al. International Consensus Statement on the Clinical and Therapeutic Management of Leber Hereditary Optic Neuropathy. J Neuroophthalmol. 2017;37(4):371–381. doi:10.1097/WNO.0000000000000570.
- Yu-Wai-Man P, Votruba M, Burté F, et al. A Neurodegenerative Perspective on Mitochondrial Optic Neuropathies. Acta Neuropathol. 2016;132(6):789–806. doi:10.1007/s00401-016-1625-2.
- Catarino CB, von Livonius B, Priglinger C, et al. Real-World Clinical Experience With Idebenone in the Treatment of Leber Hereditary Optic Neuropathy. J Neuroophthalmol. 2020;40(4):558–565. doi:10.1097/WNO.0000000000001023.
- Newman NJ, Yu-Wai-Man P, Carelli V, et al. Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A ND4 Mutation. Ophthalmology. 2023;130(5):513–521.
- European Medicines Agency (EMA). Committee for Orphan Medicinal Products – Public Summary of Opinion on Orphan Designation: Idebenone for the Treatment of LHON. 2007.
- World Health Organization (WHO). Genes and Human Disease: Mitochondrial Diseases. 2023. Available at: WHO Genetic Diseases.
- British National Formulary (BNF). Idebenone. National Institute for Health and Care Excellence (NICE). 2025.
- Rudolph G, Dimitriadis K, Büchner B, et al. Effects of Idebenone on Color Vision in Patients With Leber Hereditary Optic Neuropathy. J Neuroophthalmol. 2013;33(1):30–36. doi:10.1097/WNO.0b013e318272c643.
About Our Medical Team
All content on iMedic is created and reviewed by qualified medical professionals following international evidence-based guidelines. Our editorial process ensures accuracy, currency, and clinical relevance.
Medical Content Team
Specialists in ophthalmology, neuro-ophthalmology, and clinical pharmacology with experience in mitochondrial diseases and rare disease management.
Medical Review Board
Independent panel of board-certified physicians who verify all medical claims against current evidence and international guidelines (WHO, EMA, EFNS).
Editorial Standards
All content follows the GRADE evidence framework and is based on Level 1A evidence from systematic reviews and randomized controlled trials where available.
Independence
iMedic receives no pharmaceutical company funding or sponsorship. All content is editorially independent, ensuring unbiased medical information for patients and caregivers.