Voydeya (Danicopan)
Complement Factor D Inhibitor for Paroxysmal Nocturnal Hemoglobinuria (PNH)
Voydeya (danicopan) is a first-in-class oral complement factor D inhibitor approved for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) who have residual hemolytic anemia despite ongoing treatment with a C5 inhibitor. It is the first add-on oral therapy designed to address the extravascular hemolysis that can persist with C5 inhibitor monotherapy.
Quick Facts
Key Takeaways
- Voydeya is the first oral complement factor D inhibitor approved as add-on therapy for PNH patients on C5 inhibitors with residual anemia.
- Meningococcal vaccination is mandatory at least 2 weeks before starting treatment due to increased risk of serious meningococcal infection.
- The standard starting dose is 150 mg three times daily (every 8 hours), taken with food, which may be increased to 200 mg three times daily.
- The most common side effects are fever, headache, and elevated liver enzymes; liver function should be monitored before and during treatment.
- Voydeya must not be used as a standalone therapy — patients must continue their C5 inhibitor (ravulizumab or eculizumab) alongside it.
What Is Voydeya and What Is It Used For?
Quick Answer: Voydeya (danicopan) is an oral medication that blocks complement factor D, a protein involved in the immune-mediated destruction of red blood cells. It is used as add-on therapy in adults with paroxysmal nocturnal hemoglobinuria (PNH) who still have anemia despite treatment with a C5 inhibitor.
Voydeya contains the active substance danicopan, a selective inhibitor of complement factor D. Factor D is a serine protease that plays a central role in the alternative pathway of the complement system — a part of the body's innate immune defense. In patients with PNH, the complement system mistakenly targets and destroys their own red blood cells, a process known as hemolysis.
While C5 inhibitors such as ravulizumab (Ultomiris) and eculizumab (Soliris) effectively block intravascular hemolysis (destruction of red blood cells within blood vessels), many patients continue to experience extravascular hemolysis. This occurs when complement proteins called C3 fragments coat (opsonize) the red blood cells, marking them for removal by the spleen and liver. By blocking factor D upstream in the complement cascade, danicopan prevents this C3 opsonization and reduces extravascular hemolysis.
In the pivotal Phase III ALPHA trial, patients receiving Voydeya as add-on therapy to a C5 inhibitor showed significant improvements in hemoglobin levels compared to placebo, with a least-squares mean difference of approximately 1.8 g/dL at 12 weeks. Many patients achieved clinically meaningful reductions in fatigue and improved quality of life. The benefit was sustained throughout the 24-week treatment period and in long-term extension studies.
Voydeya received marketing authorization from the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) for adult patients with PNH who have an inadequate response to a C5 inhibitor. It is the first approved oral add-on therapy for this indication, representing a significant advance in PNH management by addressing an unmet medical need for patients with persistent hemolytic anemia.
What Should You Know Before Taking Voydeya?
Quick Answer: You must be vaccinated against meningococcal disease before starting Voydeya. Do not take this medicine if you have an active meningococcal infection or are allergic to danicopan. Inform your doctor about kidney problems, low body weight, pregnancy, or other medications you are taking.
Contraindications
Do not take Voydeya if any of the following apply to you:
- You are allergic to danicopan or any of the other ingredients in this medicine (see Ingredients section).
- You have not been vaccinated against meningococcal infection caused by Neisseria meningitidis.
- You currently have a meningococcal infection.
Warnings and Precautions
Talk to your doctor or pharmacist before taking Voydeya if you have any infections. Because Voydeya targets the complement system, which is part of your body's defense against infections, taking this medicine increases your risk of certain serious infections.
Voydeya increases the risk of meningococcal infection caused by Neisseria meningitidis. These are severe infections affecting the membranes surrounding the brain and spinal cord (meningitis), which can cause brain inflammation (encephalitis) and may spread through the bloodstream (sepsis). Meningococcal infection can be life-threatening if not treated promptly.
Before starting Voydeya, you must have received meningococcal vaccinations at least 2 weeks prior to your first dose. If vaccination cannot be given 2 weeks in advance, your doctor will prescribe prophylactic antibiotics to reduce the risk of infection until 2 weeks after vaccination. Even if you have been previously vaccinated, you may need booster doses before starting Voydeya. Be aware that vaccination does not eliminate all risk of meningococcal infection.
Seek emergency medical attention immediately if you experience any of these symptoms of meningococcal infection:
- Headache with nausea or vomiting
- Headache with fever
- Headache with stiff neck or stiff back
- Fever with skin rash
- Confusion or disorientation
- Muscle pain with flu-like symptoms
- Sensitivity to light (photophobia)
If you plan to travel to a region where you cannot contact your doctor or access medical care, your doctor may prescribe a course of antibiotics against Neisseria meningitidis for you to carry. If you develop any meningococcal symptoms while traveling, take the antibiotics as prescribed and seek medical attention as soon as possible, even if you feel better after taking them.
Kidney problems: Tell your doctor if you have severe kidney impairment. Your doctor may adjust your dose and monitor you more closely during treatment because danicopan levels in the blood may be higher in patients with kidney disease. Voydeya is not recommended for patients with severe renal impairment.
Low body weight: If you weigh less than 60 kg (132 lb), tell your doctor. You may need closer monitoring during treatment due to potentially higher blood levels of danicopan.
Liver function: Voydeya can increase certain liver enzymes in the blood. Your doctor will perform blood tests to check your liver function before you start treatment and may monitor liver enzymes during therapy.
Pregnancy and Breastfeeding
If you are pregnant, think you may be pregnant, or are planning to have a baby, ask your doctor or pharmacist for advice before taking this medicine. The effects of Voydeya on an unborn baby are not known. As a precaution, you should not take Voydeya if you are pregnant.
Danicopan may pass into breast milk. Do not breastfeed while taking Voydeya. Breastfeeding should not be started until 3 days after you stop taking Voydeya.
Children and Adolescents
Voydeya must not be given to children or adolescents under 18 years of age. There are no data on the safety and efficacy of danicopan in this age group.
Driving and Using Machines
Voydeya has no or negligible effect on the ability to drive and use machines.
How Does Voydeya Interact with Other Drugs?
Quick Answer: Voydeya can interact with several medications including blood thinners (dabigatran, edoxaban), heart medications (digoxin), immunosuppressants (tacrolimus), and cholesterol-lowering drugs (rosuvastatin). Always inform your doctor about all medications you are currently taking.
Tell your doctor or pharmacist about all medicines you are taking, have recently taken, or might take. Danicopan may affect the blood levels or effectiveness of other drugs, and some medications may affect how Voydeya works in your body. The following are medications that require special attention:
| Medication | Therapeutic Use | Interaction Type | Clinical Significance |
|---|---|---|---|
| Dabigatran | Anticoagulant (blood thinner) | Increased dabigatran levels | Increased bleeding risk; dose adjustment may be needed |
| Edoxaban | Anticoagulant (blood thinner) | Increased edoxaban levels | Increased bleeding risk; dose adjustment may be needed |
| Digoxin | Heart rhythm medication | Increased digoxin levels | Risk of digoxin toxicity; monitoring recommended |
| Fexofenadine | Antihistamine (allergy treatment) | Altered fexofenadine levels | May affect antihistamine efficacy |
| Tacrolimus | Immunosuppressant | Increased tacrolimus levels | Risk of tacrolimus toxicity; close monitoring required |
| Rosuvastatin | Cholesterol-lowering (statin) | Increased rosuvastatin levels | Increased risk of statin side effects (myopathy) |
| Sulfasalazine | Inflammatory bowel disease / rheumatoid arthritis | Altered sulfasalazine levels | May affect therapeutic efficacy |
Danicopan is a substrate and inhibitor of several drug transporters, which explains many of its pharmacokinetic interactions. It inhibits P-glycoprotein (P-gp), breast cancer resistance protein (BCRP), and organic anion transporting polypeptides (OATP1B1 and OATP1B3). These transporters are responsible for the absorption, distribution, and elimination of many commonly used drugs.
It is important that your doctor is aware of all medications you are taking so they can assess whether any dose adjustments or additional monitoring are necessary. This includes prescription medicines, over-the-counter drugs, herbal products, and dietary supplements. Do not start or stop any medication without first consulting your doctor.
What Is the Correct Dosage of Voydeya?
Quick Answer: The recommended starting dose is 150 mg three times daily (one 50 mg tablet plus one 100 mg tablet), taken approximately every 8 hours with food. Your doctor may increase the dose to 200 mg three times daily based on your response.
Always take Voydeya exactly as your doctor or pharmacist has told you. Do not change your dose without consulting your healthcare provider. The tablets should be taken with food (a meal or a snack) to ensure proper absorption.
Adults with Normal Kidney Function
Standard Dosing
Starting dose: 150 mg three times daily (approximately every 8 hours, ±2 hours)
Maximum dose: 200 mg three times daily, based on treatment response
- 100 mg dose: one 100 mg tablet
- 150 mg dose: one 50 mg tablet + one 100 mg tablet
- 200 mg dose: two 100 mg tablets
Patients with Severe Kidney Impairment
Adjusted Dosing for Severe Renal Impairment
Starting dose: 100 mg three times daily (approximately every 8 hours, ±2 hours)
Maximum dose: 150 mg three times daily, based on treatment response
| Patient Group | Starting Dose | Maximum Dose | Frequency |
|---|---|---|---|
| Adults (normal renal function) | 150 mg | 200 mg | Three times daily (~every 8 hours) |
| Severe renal impairment | 100 mg | 150 mg | Three times daily (~every 8 hours) |
| Children (<18 years) | Not approved | Not approved | N/A |
Missed Dose
If you miss a dose, take it as soon as you remember. However, if it is almost time for your next dose, skip the missed dose and take the next one at its scheduled time. Do not take a double dose to make up for a forgotten dose.
Overdose
If you have taken too much Voydeya, contact your doctor immediately. Bring the medicine packaging with you so that you can easily describe what you have taken. There is no specific antidote for danicopan overdose; treatment is supportive and symptom-based.
Stopping Treatment
Do not stop taking Voydeya unless your doctor tells you to. If you stop taking this medicine, symptoms of residual hemolytic anemia may return. If discontinuation is necessary, your doctor will gradually reduce your dose to minimize the risk of a hemolytic flare.
What Are the Side Effects of Voydeya?
Quick Answer: The most common side effects of Voydeya include fever (pyrexia), headache, and elevated liver enzymes. Common side effects include limb pain, vomiting, and high blood pressure. Serious but rare side effects include meningococcal infection, which requires immediate medical attention.
Like all medicines, Voydeya can cause side effects, although not everybody gets them. Side effects are classified by how frequently they occur. The following frequency grid provides an overview of the reported adverse reactions from clinical trials and post-marketing surveillance.
Very Common
Affects more than 1 in 10 patients
- Fever or high temperature (pyrexia)
- Headache
- Elevated liver enzymes (detected in blood tests)
Common
Affects up to 1 in 10 patients
- Pain in the arms and legs (extremity pain)
- Vomiting
- High blood pressure (hypertension)
Contact your doctor or go to the emergency department immediately if you experience any symptoms of meningococcal infection, including headache with nausea, vomiting, or fever; stiff neck or back; skin rash with fever; confusion; severe muscle pain with flu-like symptoms; or sensitivity to light. Meningococcal infection can progress rapidly and be fatal if untreated.
Your doctor will monitor your liver enzyme levels through blood tests during treatment. If you notice any signs of liver problems such as yellowing of the skin or eyes (jaundice), dark urine, unusual fatigue, or upper right abdominal pain, contact your doctor promptly.
If you experience any side effects, including any not listed above, talk to your doctor or pharmacist. You can also report suspected side effects to your national pharmacovigilance authority. Reporting helps the medical community continuously monitor the benefit-risk profile of medications.
How Should You Store Voydeya?
Quick Answer: Store Voydeya out of the sight and reach of children. No special storage conditions are required. After first opening the bottle, use the medicine within 48 days. Do not use after the expiry date on the packaging.
Keep this medicine out of the sight and reach of children. Do not use Voydeya after the expiry date (EXP) printed on the carton, bottle, or blister packaging. The expiry date refers to the last day of that month.
There are no special storage conditions for Voydeya tablets. After first opening the bottle, the medicine should be used within 48 days. Store the tablets in the original packaging to protect from moisture.
Do not dispose of medicines in wastewater or household waste. Ask your pharmacist how to dispose of medicines you no longer use. These measures help protect the environment.
What Does Voydeya Contain?
Quick Answer: Each Voydeya tablet contains either 50 mg or 100 mg of the active substance danicopan. Inactive ingredients include lactose monohydrate, microcrystalline cellulose, croscarmellose sodium, and other standard tablet excipients.
Active substance: Danicopan. Each film-coated tablet contains either 50 mg or 100 mg of danicopan.
Tablet core excipients: Lactose monohydrate, microcrystalline cellulose, croscarmellose sodium, sodium lauryl sulfate, magnesium stearate, colloidal silicon dioxide (hydrophobic), hypromellose acetate succinate.
Film coating: Polyvinyl alcohol, titanium dioxide (E171), macrogol 4000, talc.
Lactose: Voydeya contains lactose monohydrate. If you have been told by your doctor that you have an intolerance to some sugars, contact your doctor before taking this medicine.
Sodium: This medicine contains less than 1 mmol (23 mg) sodium per tablet, meaning it is essentially sodium-free.
Appearance: Voydeya tablets are white to off-white, round, film-coated tablets. The 50 mg tablets are debossed with “DCN” above “50” on one side and plain on the other. The 100 mg tablets are debossed with “DCN” above “100” on one side and plain on the other.
Pack sizes: Voydeya is available as bottles (90 tablets per bottle) or in wallet blister packs (21 tablets per blister card). The 150 mg dose pack contains 90 × 50 mg tablets and 90 × 100 mg tablets. The 200 mg dose pack contains 180 × 100 mg tablets. Not all pack sizes may be marketed in all countries.
Frequently Asked Questions About Voydeya
Voydeya (danicopan) is used to treat adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who have residual hemolytic anemia despite ongoing treatment with a C5 inhibitor such as ravulizumab (Ultomiris) or eculizumab (Soliris). It is given as an add-on therapy — meaning you continue your C5 inhibitor while also taking Voydeya — to further reduce the destruction of red blood cells.
C5 inhibitors like ravulizumab and eculizumab block the terminal complement pathway by targeting complement component C5, preventing intravascular hemolysis (direct destruction of red blood cells in the bloodstream). However, C5 inhibitors do not prevent upstream C3 opsonization, where complement proteins coat red blood cells and mark them for removal by the spleen and liver (extravascular hemolysis). Voydeya blocks factor D further upstream in the complement cascade, preventing this C3 opsonization and thereby addressing the extravascular hemolysis that C5 inhibitors cannot fully control.
The most common side effects (affecting more than 1 in 10 patients) include fever (pyrexia), headache, and elevated liver enzymes detected on blood tests. Common side effects (affecting up to 1 in 10 patients) include pain in the arms and legs, vomiting, and high blood pressure. Most side effects are mild to moderate in severity. Your doctor will monitor liver enzymes before and during treatment.
Voydeya inhibits complement factor D, which is part of the alternative complement pathway — a crucial component of the immune system's defense against certain bacteria. Blocking this pathway increases the risk of infection with Neisseria meningitidis, which can cause life-threatening meningitis and sepsis. Vaccination at least 2 weeks before starting treatment significantly reduces this risk. If vaccination cannot be completed in time, your doctor will prescribe prophylactic antibiotics until 2 weeks after vaccination.
No. Voydeya is currently approved only as add-on therapy to a C5 inhibitor. It is not authorized for use as monotherapy for PNH. Patients must continue taking their C5 inhibitor (either ravulizumab or eculizumab) alongside Voydeya. Stopping the C5 inhibitor while on Voydeya alone could lead to uncontrolled intravascular hemolysis and serious complications. Always follow your hematologist's treatment plan.
If you miss a dose, take it as soon as you remember. If it is almost time for your next scheduled dose, skip the missed dose and resume your regular dosing schedule. Do not take two doses at once to compensate for a missed dose. If you are unsure what to do, contact your doctor or pharmacist for advice. Maintaining a consistent dosing schedule (approximately every 8 hours) is important for optimal treatment effect.
References
- European Medicines Agency (EMA). Voydeya (danicopan) — Summary of Product Characteristics. Available at: EMA Voydeya EPAR. Accessed January 2026.
- Kulasekararaj AG, et al. “Danicopan as add-on therapy to a complement C5 inhibitor in paroxysmal nocturnal haemoglobinuria (ALPHA): a randomised, double-blind, placebo-controlled, phase 3 trial.” The Lancet Haematology. 2024.
- Brodsky RA. “Paroxysmal nocturnal hemoglobinuria.” Blood. 2014;124(18):2804-2811.
- Hill A, et al. “Complement inhibition in paroxysmal nocturnal hemoglobinuria: current and emerging therapies.” Hematology/Oncology Clinics of North America. 2023.
- U.S. Food and Drug Administration (FDA). Voydeya Prescribing Information. 2024.
- Risitano AM, et al. “Anti-complement treatment for paroxysmal nocturnal hemoglobinuria: time for proximal complement inhibition?” Haematologica. 2014;99(4):616-624.
- World Health Organization (WHO). Model List of Essential Medicines — 23rd List, 2023.
Editorial Team
This article was written by the iMedic Medical Editorial Team, a group of licensed physicians and pharmacologists with expertise in hematology, clinical pharmacology, and evidence-based medicine. All content follows the iMedic Editorial Standards and is reviewed according to the GRADE evidence framework.
All medical information is based on approved prescribing information (EMA SmPC, FDA label), peer-reviewed clinical trial data, and international guidelines. iMedic has no commercial funding and accepts no pharmaceutical advertising. Read more about our Medical Review Board.