VOYDEYA (danicopan)
VOYDEYA (danicopan) is a complement factor D inhibitor indicated as an add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH). It is specifically intended for patients who require additional control over EVH while maintaining their existing treatment for intravascular hemolysis. The medication has not demonstrated effectiveness as a monotherapy and must be prescribed in combination with the specified terminal complement inhibitors.
How VOYDEYA Works
Danicopan functions by reversibly binding to complement Factor D to selectively inhibit the alternative complement pathway. This inhibition prevents the cleavage of complement Factor B, which is a necessary step in forming the C3 convertase enzyme and generating downstream effectors. By blocking this proximal part of the complement cascade, the drug controls the C3 fragment opsonization that facilitates extravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria. This mechanism allows for the targeted management of extravascular hemolysis while co-administered treatments maintain control over terminal pathway-mediated intravascular hemolysis.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2024-03-29
- Patent Cliff
- 2038
- Routes
- ORAL
- Dosage Forms
- TABLET
VOYDEYA Approval History
What VOYDEYA Treats
2 indicationsVOYDEYA is approved for 2 conditions since its original approval in 2024. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Extravascular Hemolysis
- Paroxysmal Nocturnal Hemoglobinuria
VOYDEYA Boxed Warning
SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA VOYDEYA, a complement inhibitor, increases the risk of serious infections, especially those caused by encapsulated bacteria, such as Neisseria meningitidis , Streptococcus pneumoniae , and Haemophilus influenzae type B [ see Warnings and Precautions (5.1) ]. Life-threatening and fatal infections with encapsulated bacteria have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal i...
WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA VOYDEYA, a complement inhibitor, increases the risk of serious infections, especially those caused by encapsulated bacteria, such as Neisseria meningitidis , Streptococcus pneumoniae , and Haemophilus influenzae type B [ see Warnings and Precautions (5.1) ]. Life-threatening and fatal infections with encapsulated bacteria have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if not recognized and treated early. Complete or update vaccination for encapsulated bacteria specifically, Neisseria meningitidis and Streptococcus pneumoniae at least 2 weeks prior to the first dose of VOYDEYA, unless the risks of delaying therapy with VOYDEYA outweigh the risk of developing a serious infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against encapsulated bacteria in patients receiving a complement inhibitor. See Warnings and Precautions (5.1) for additional guidance on the management of the risk of serious infections caused by encapsulated bacteria. Patients receiving VOYDEYA are at increased risk for invasive disease caused by encapsulated bacteria, even if they develop antibodies following vaccination. Monitor patients for early signs and symptoms of serious infections and evaluate immediately if infection is suspected. Because of the risk of serious infections caused by encapsulated bacteria, VOYDEYA is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the VOYDEYA REMS [see Warnings and Precautions (5.2) ] . WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA See full prescribing information for complete boxed warning. VOYDEYA increases the risk of serious and life-threatening infections, caused by encapsulated bacteria, including Neisseria meningitidis , Streptococcus pneumoniae , and Haemophilus influenzae t
VOYDEYA Competitive Set
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Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
Drugs Similar to VOYDEYA
3 of 5FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
20 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT05389449 | ALXN2040-PNH-303 | Ph 3 | active not recruiting | A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH |
| NCT06449001 | D7332C00006 ALXN2040-PNH-302 | Ph 3 | recruiting | Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis |
| NCT05019521 results posted | ALXN2040-GA-201 2021-001198-22 | Ph 2 | terminated | A Study of Danicopan in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration |
| NCT04469465 results posted | ALXN2040-PNH-301 2019-003829-18 | Ph 3 | completed | Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA) |
| NCT03472885 PNH results posted | ACH471-101 2016-003526-16, U1111-1209-4655 | Ph 2 | completed | Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab |
| NCT03459443 results posted | ACH471-205 2017-002674-39 | Ph 2 | terminated | A Proof of Concept Study for a 12 Month Treatment in Patients With C3G or IC-MPGN Treated With ACH-0144471 |
| NCT04988035 results posted | 20-0013C | Ph 2 | completed | ACTIV-5 / Big Effect Trial (BET-C) for the Treatment of COVID-19 |
| NCT04551599 | ACH471-016 | Ph 1 | completed | A Study of the Effects of Food and Age on Danicopan |
| NCT03369236 results posted | ACH471-204 2017-000663-33 | Ph 2 | completed | A Proof-of-Concept Study of Danicopan for 6 Months of Treatment in Participants With C3 Glomerulopathy (C3G) |
| NCT03053102 results posted | ACH471-100 2016-002652-25, U1111-1190-3490 | Ph 2 | completed | Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) |
| NCT05109390 | ACH471-014 | Ph 1 | completed | A Study of the Drug Interactions Between Danicopan and Cyclosporine, Tacrolimus, Antacids, and Omeprazole in Healthy Adults |
| NCT03124368 results posted | ACH471-201 2016-003525-42 | Ph 2 | completed | A Proof-of-Mechanism Study to Determine the Effect of Danicopan on C3 Levels in Participants With C3G or IC-MPGN |
| NCT05016206 | ACH471-013 | Ph 1 | completed | A Study of the Cardiac Effects of Danicopan in Healthy Adults |
| NCT03555539 | ACH471-012 | Ph 1 | completed | Study of Danicopan in Participants With Hepatic Impairment |
| NCT03108274 | ACH471-010 U1111-1193-2774 | Ph 1 | completed | A Drug Interaction Study Between Danicopan and Midazolam, Fexofenadine, and Mycophenolate Mofetil in Healthy Participants |
| NCT04935294 | ACH471-009 | Ph 1 | completed | Study of Danicopan in Participants With Normal Kidney Function and Participants With Kidney Dysfunction |
| NCT04889690 | ACH471-002 | Ph 1 | completed | Study of Multiple Doses of Danicopan in Healthy Participants |
| NCT04889677 | ACH471-001 | Ph 1 | completed | Study of a Single Dose of Danicopan in Healthy Participants |
| NCT04451434 | ALXN2040-HV-101 | Ph 1 | completed | Study of Danicopan in Participants of Japanese Descent |
| NCT04709094 | ACH471-017 CA27858 | Ph 1 | completed | A Drug Interaction Study of Danicopan |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
VOYDEYA FDA Label Details
Indications & Usage
VOYDEYA is indicated for the treatment of Extravascular Hemolysis; Paroxysmal Nocturnal Hemoglobinuria.
WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA VOYDEYA, a complement inhibitor, increases the risk of serious infections, especially those caused by encapsulated bacteria, such as Neisseria meningitidis , Streptococcus pneumoniae , and Haemophilus influenzae type B [ see Warnings and Pr...
VOYDEYA Patents & Exclusivity
Patents (2 active)
Exclusivity
Pro Intelligence Preview
Deep insights for VOYDEYA
Revenue Insights
- • Quarterly revenue tracking
- • Historical trend analysis
Patent Timeline
- • Cliff: 2038
- • 8 active patents
Trial Analysis
- • 20 total trials
- • Stage: Declining
Competitive Landscape
- • 5 similar drugs
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment