Last reviewed · How we verify
DANICOPAN
Danicopan is a selective inhibitor of complement Factor D, preventing the cleavage of Factor B and the formation of the C3 convertase in the alternative complement pathway.
Danicopan (VOYDEYA) is a complement factor D inhibitor developed by Alexion Pharmaceuticals for the treatment of extravascular hemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH). It is used as an add-on therapy to ravulizumab or eculizumab. The drug targets the alternative complement pathway to control C3 fragment-mediated EVH. Key safety concerns include serious infections caused by encapsulated bacteria, hepatic enzyme increases, and hyperlipidemia. Common side effects include headache. Danicopan has specific drug interactions with BCRP and P-gp substrates, requiring careful monitoring and potential dose adjustments.
At a glance
| Generic name | DANICOPAN |
|---|---|
| Drug class | Complement Factor D Inhibitor |
| Target | Complement Factor D |
| Modality | Small molecule |
| Therapeutic area | Other |
| Phase | FDA-approved |
| First approval | 2024 |
Mechanism of action
Danicopan binds to complement Factor D, inhibiting its activity and thus blocking the alternative complement pathway. This prevents the formation of C3 convertase, reducing the generation of C3 fragments and the amplification of the terminal pathway.
Approved indications
Boxed warnings
- SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA: VOYDEYA increases the risk of serious infections, especially those caused by encapsulated bacteria, such as Neisseria meningitidis, Streptococcus pneumoniae, and Haemophilus influenzae type B.
Common side effects
- Headache
Drug interactions
- undefined
- undefined
Key clinical trials
- A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH (PHASE3)
- Danicopan PMS in Korea
- Danicopan Early Access Program
- Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data
- Long-term Safety of Danicopan: IPIG Registry-based Cohort Study
- Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis (PHASE3)
- A Study of Danicopan in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration (PHASE2)
- Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA) (PHASE3)
Patents
| Patent | Expiry | Type |
|---|---|---|
| 9796741 | 2035-02-25 | Compound |
| 12076319 | 2038-08-02 | Method of Use |
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- DANICOPAN CI brief — competitive landscape report
- DANICOPAN updates RSS · CI watch RSS