Danicopan

Danicopan binds reversibly to complement Factor D and selectively inhibits the alternative complement pathway. Danicopan prevents the cleavage of complement Factor B into the Ba and Bb fragments which are required for the formation of the alternative pathway (AP) complement component C3 convertase (C3bBb), the generation of downstream effectors including C3 fragment opsonization, and the amplification of the terminal pathway.

• VOYDEYA is indicated as add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH). VOYDEYA is a complement factor D inhibitor indicated as add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH) ( 1 ).

• VOYDEYA is contraindicated for initiation in patients with unresolved serious infection caused by encapsulated bacteria, including Neisseria meningitidis , Streptococcus pneumoniae , or Haemophilus influenzae type B [see Warnings and Precautions (5.1) ] . Initiation in patients with unresolved serious infection caused by encapsulated bacteria ( 4 ).contraindicated

Start 150 mg three times a day orally, with or without food. Depending on clinical response, can increase to 200 mg three times a day. See Full Prescribing Information for instructions on dosage and administration ( 2.1 , 2.2 ). 2.1 Recommended Vaccination and Prophylaxis for Encapsulated Bacterial Infections Vaccinate patients against encapsulated bacteria, including Neisseria meningitidis (serogroups A, C, W, Y, and B) and Streptococcus pneumoniae according to current ACIP recommendations at least 2 weeks prior to initiation of VOYDEYA. If urgent VOYDEYA therapy is indicated in a patient who is not up to date with vaccines for Neisseria meningitidis and Streptococcus pneumoniae according to ACIP recommendations, provide the patient with antibacterial drug prophylaxis and administer these vaccines as soon as possible [see Warnings and Precautions (5.1) ] . Healthcare professionals who prescribe VOYDEYA must enroll in the VOYDEYA REMS [see Warnings and Precautions (5.2) ] . 2.2 Recommended Dosage Starting Dose: The recommended dosage of VOYDEYA is 150 mg three times a day administered orally. VOYDEYA can be taken with or without food. VOYDEYA doses should be taken around the same time every day. Dose Adjustment: The dose can be increased to 200 mg three times a day if the patient's hemoglobin (Hgb) level has not increased by greater than 2 g/dL after 4 weeks of therapy, if the patient required a transfusion during the previous 4 weeks, or to achieve an appropriate Hgb response based on clinical judgement.

Hepatic Enzyme Increases: Assess liver enzymes before treatment initiation and periodically during treatment. Consider treatment interruption or discontinuation if elevations are clinically significant or if the patient becomes symptomatic ( 5.3 ). Hyperlipidemia: Monitor serum lipids periodically during treatment and initiate cholesterol-lowering medication if indicated ( 5.4 ). 5.1 Serious Infections Caused by Encapsulated Bacteria VOYDEYA, a complement inhibitor, increases a patient's susceptibility to serious, life-threatening, or fatal infections caused by encapsulated bacteria including Neisseria meningitidis (caused by any serogroup, including non-groupable strains), Streptococcus pneumoniae , and Haemophilus influenzae type B. Life-threatening and fatal infections with encapsulated bacteria have occurred in both vaccinated and unvaccinated patients treated with complement inhibitors. The initiation of VOYDEYA treatment is contraindicated in patients with unresolved serious infections caused by encapsulated bacteria. Complete or update vaccination against encapsulated bacteria, specifically Neisseria meningitidis and Streptococcus pneumoniae at least 2 weeks prior to administration of the first dose of VOYDEYA, according to the current ACIP recommendations for patients receiving a complement inhibitor. Revaccinate patients in accordance with ACIP recommendations considering the duration of therapy with VOYDEYA.

The following clinically significant adverse reactions are discussed in greater detail in other sections of the labeling: Serious Infections Caused by Encapsulated Bacteria [see Warnings and Precautions (5.1) ] Hepatic Enzyme Increases [see Warnings and Precautions (5.3) ] Hyperlipidemia [see Warnings and Precautions (5.4) ] Most frequent adverse reaction (incidence ≥10%) was headache ( 6 ). To report SUSPECTED ADVERSE REACTIONS, contact Alexion Pharmaceuticals, Inc. at 1-844-259-6783 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The safety of VOYDEYA was evaluated in 86 adults with PNH in Study ALXN2040-PNH-301 [see Clinical Studies (14) ] . Study ALXN2040-PNH-301 enrolled adults with PNH with clinically significant EVH who had been treated with a stable dose of ravulizumab or eculizumab for at least the previous 6 months. Patients were randomly assigned 2:1 to receive double-blind VOYDEYA 150 mg (n=57) or placebo (n=29) orally three times a day in addition to ravulizumab or eculizumab for 12 weeks. Patients received either US-approved or non-US-approved ravulizumab or eculizumab in the trial. Among patients who were randomized to receive VOYDEYA, 84% were exposed for at least 12 weeks.

Hepatic Impairment: Avoid use in patients with severe hepatic impairment (Child-Pugh C) ( 8.6 ). 8.1 Pregnancy Risk Summary There are no available data on VOYDEYA use in pregnant individuals to evaluate for a drug-associated risk of major birth defects, miscarriage or adverse maternal or fetal outcomes. There are risks to the mother and fetus associated with untreated PNH in pregnancy (see Clinical Considerations ) . The use of VOYDEYA in pregnant women or women planning to become pregnant may be considered following an assessment of the risks and benefits. In animal reproduction studies, oral administration of danicopan to pregnant New Zealand White (NZW) rabbits and Wistar Hans (WH) rats during organogenesis at exposures 18 or 25-times, respectively, above the human exposure at the maximum recommended human dose (MRHD) of 200 mg three times a day (based on AUC) resulted in no adverse developmental effects (see Data ) . The estimated background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, respectively.

Metabolism

At the recommended dosages of 150 or 200 mg three times a day, the median systemic exposure of danicopan at steady state has a maximum plasma concentration (C max,ss ) of 535 or 665 ng/mL, respectively, and has an area under the plasma drug concentration time curve (AUC 24,ss ) of 8180 or 10200 ng × h/mL, respectively. Danicopan exposures at steady state generally increase in a dose-proportional manner from 150 mg three times a day to 200 mg three times a day.

Serum ALT elevations occurred after treatment cessation without a taper in 2 healthy subjects who received danicopan 500 mg and 800 mg twice a day. These abnormal ALT findings were transient, with no evidence of hepatic function abnormality and resolved spontaneously. In case of overdose, elevations in liver enzymes may occur. General supportive measures are recommended. It is not known if VOYDEYA can be removed by dialysis.

• Mar 29, 2024NDANDA218037Alexion Pharms Inc

1. 1Haemoglobin Decreased2913%
2. 2Haemolysis2310.0%
3. 3Fatigue229.5%
4. 4Extravascular Haemolysis156.5%
5. 5Anaemia146.1%
6. 6Headache135.6%
7. 7Breakthrough Haemolysis125.2%
8. 8Drug Ineffective114.8%
9. 9Pneumonia104.3%
10. 10Asthenia93.9%
11. 11Blood Bilirubin Increased93.9%
12. 12Covid-1993.9%
13. 13Off Label Use93.9%
14. 14Jaundice83.5%
15. 15Nausea83.5%

The 10 most recently updated of 30 ClinicalTrials.gov registrations naming Danicopan as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Danicopan Early Access Program
  Available · Expanded access · Alexion Pharmaceuticals, Inc.
  NCT05982938updated 2026-06-04
• Danicopan PMS in Korea
  Recruiting · Observational · 8 enrolled · AstraZeneca
  NCT07457151updated 2026-05-22
• A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH
  Active not recruiting · Phase 3 · Interventional · 80 enrolled · Alexion Pharmaceuticals, Inc.
  NCT05389449updated 2026-04-16
• Long-term Safety of Danicopan: IPIG Registry-based Cohort Study
  Active not recruiting · Observational · 50 enrolled · Alexion Pharmaceuticals, Inc.
  NCT07413679updated 2026-02-17
• Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data
  Active not recruiting · Observational · 50 enrolled · Alexion Pharmaceuticals, Inc.
  NCT07413250updated 2026-02-17
• Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis
  Recruiting · Phase 3 · Interventional · 6 enrolled · Alexion Pharmaceuticals, Inc.
  NCT06449001updated 2025-12-23
• A Study of Danicopan in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration
  Terminated · Phase 2 · Interventional · 365 enrolled · Alexion Pharmaceuticals, Inc.
  NCT05019521updated 2025-09-05
• Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)
  Completed · Phase 3 · Interventional · 86 enrolled · Alexion Pharmaceuticals, Inc.
  NCT04469465updated 2025-05-04
• Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy
  Terminated · Phase 2 · Interventional · 29 enrolled · Alexion Pharmaceuticals, Inc.
  NCT04170023updated 2024-11-20
• Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab
  Completed · Phase 2 · Interventional · 12 enrolled · Alexion Pharmaceuticals, Inc.
  NCT03472885updated 2023-12-12

Frequently asked questions

How does Danicopan work?

Danicopan binds reversibly to complement Factor D and selectively inhibits the alternative complement pathway. Danicopan prevents the cleavage of complement Factor B into the Ba and Bb fragments which are required for the formation of the alternative pathway (AP) complement component C3 convertase (C3bBb), the generation of downstream effectors including C3 fragment opsonization, and the amplification of the terminal pathway.

What is Danicopan used for?

According to FDA labeling, Danicopan carries indications including: VOYDEYA is indicated as add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH). VOYDEYA is a complement factor D inhibitor indicated as add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH) ( 1 ).. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Danicopan?

Danicopan is classified as Complement inhibitors, Complement Factor D Inhibitor, Breast Cancer Resistance Protein Inhibitors, Complement Factor D Inhibitors, Complement Inhibitors, P-Glycoprotein Inhibitors, Decreased Complement Activity.

What are the brand names for Danicopan?

Danicopan is marketed under brand names including Voydeya.

What are the contraindications for Danicopan?

Danicopan labeling lists contraindications including: VOYDEYA is contraindicated for initiation in patients with unresolved serious infection caused by encapsulated bacteria, including Neisseria meningitidis , Streptococcus pneumoniae , or Haemophilus influenzae type B [see Warnings and Precautions (5.1) ] . Initiation in patients with unresolved serious infection caused by encapsulated bacteria ( 4 ).. Always consult the full prescribing information and a clinician.