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Drug

Danicopan

Approved · EMA
Emerging researchLate-stage development

Also known as (2S,4R)-1-(2-(3-acetyl-5-(2-methylpyrimidin-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromopyridin-2-yl)-4-fluoropyrrolidine-2-carboxamide, (2S,4R)-1-(2-(3-acetyl-5-(2-methylpyrimidine-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromopyridine-2-yl)-4-fluoropyrrolidine-2-carboxamide, Voydeya.

RxNorm2678952ChEMBLCHEMBL4250860UNIIJM8C1SFX0U
2
Research papers
2
Active clinical trials
1
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

A Long-term Extension (LTE) Study to Characterize the Safety and Efficacy of Danicopan as an Add-on Therapy to a Complement Component 5 Inhibitor (C5i) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Previously Treated With Danicopan in an Alexion-sponsored Clinical Study

Clinical trial2026-06-18Results expected Q3 2026 · ClinicalTrials.gov

A Phase 3 Open-Label Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Significant Extravascular Hemolysis

Clinical trial2025-12-23Results expected Q2 2027 · ClinicalTrials.gov

A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)

Clinical trial2025-05-04Results posted · ClinicalTrials.gov

A Phase 2, Double-Masked, Placebo-Controlled, Dose Range Finding Study of Danicopan (ALXN2040) in Patients With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)

Clinical trial2025-01-21Terminated · ClinicalTrials.gov

Approval: Voydeya (EMA)

Regulatory2024-04-19EMA

A Phase 2 Open-label Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have an Inadequate Response to Eculizumab Monotherapy

Clinical trial2023-12-12Results posted · ClinicalTrials.gov

Addition of danicopan to ravulizumab or eculizumab in patients with paroxysmal nocturnal haemoglobinuria and clinically significant extravascular haemolysis (ALPHA): a double-blind, randomised, phase 3 trial.

Research2023-12-01The Lancet. Haematology

A Multicenter Platform Trial of Putative Therapeutics for the Treatment of COVID-19 in Hospitalized Adults

Clinical trial2023-06-08Results posted · ClinicalTrials.gov

A Phase 2, Proof-of-Concept, Randomized, Double-Blinded, Placebo-Controlled Study of ACH-0144471 Treatment for 6 Months in Patients With C3 Glomerulopathy (C3G), With an Open-label Extension

Clinical trial2022-10-14Results posted · ClinicalTrials.gov

A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of ACH-0144471 in Untreated Patients With Paroxysmal Nocturnal Hemoglobinuria

Clinical trial2022-06-23Results posted · ClinicalTrials.gov

Danicopan: an oral complement factor D inhibitor for paroxysmal nocturnal hemoglobinuria

Research2021-12-01Haematologica

A Phase 2a Proof-of-Mechanism, Open-Label Study to Determine the Effect of ACH-0144471 on C3 Levels in Participants With Low C3 Levels Due to Either C3 Glomerulopathy (C3G) or Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN)

Clinical trial2021-11-04Results posted · ClinicalTrials.gov

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Danicopan
Aliases & brands
(2S,4R)-1-(2-(3-acetyl-5-(2-methylpyrimidin-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromopyridin-2-yl)-4-fluoropyrrolidine-2-carboxamide(2S,4R)-1-(2-(3-acetyl-5-(2-methylpyrimidine-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromopyridine-2-yl)-4-fluoropyrrolidine-2-carboxamideVoydeya
RxNorm CUI
2678952
ChEMBL ID
CHEMBL4250860
UNII
JM8C1SFX0U
Regulatory jurisdictions
ema

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2024-04-19
Latest approval
2024-04-19
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2024-04-19Approval
Approval: Voydeya (EMA)
Indication: Voydeya is indicated as an add-on to ravulizumab or eculizumab for the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have residual haemolytic… Show full indication

Voydeya is indicated as an add-on to ravulizumab or eculizumab for the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have residual haemolytic anaemia 

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

23 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
23
registered trials across all phases
LATEST COMPLETION 2024
2
Active studies
1
Recruiting
3
Late-stage (III+)
19
Completed
2
Discontinued
PHASE DISTRIBUTIONn = 23
Phase 113Phase 27Phase 33

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

A Phase 3 Open-Label Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Significant Extravascular Hemolysis

RecruitingPH IIIAlexion Pharmaceuticals, Inc.n=62028

A Long-term Extension (LTE) Study to Characterize the Safety and Efficacy of Danicopan as an Add-on Therapy to a Complement Component 5 Inhibitor (C5i) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Previously Treated With Danicopan in an Alexion-sponsored Clinical Study

Active, not recruitingPH IIIAlexion Pharmaceuticals, Inc.n=802026

A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)

CompletedPH IIIAlexion Pharmaceuticals, Inc.n=862024

Recent completions

Trials that read out recently, adding to the completed evidence base.

A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)

CompletedPH IIIAlexion Pharmaceuticals, Inc.n=862024

A Phase 2 Open-label Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have an Inadequate Response to Eculizumab Monotherapy

CompletedPH IIAlexion Pharmaceuticals, Inc.n=122023

A Multicenter Platform Trial of Putative Therapeutics for the Treatment of COVID-19 in Hospitalized Adults

CompletedPH IINational Institute of Allergy and Infectious Diseases (NIAID)n=2012022

A Two-Part, Open-Label Phase 1 Study to Evaluate the Effect of Food and Age on the Pharmacokinetics of Danicopan

CompletedPH IAlexion Pharmaceuticals, Inc.n=272021

A Phase 2, Proof-of-Concept, Randomized, Double-Blinded, Placebo-Controlled Study of ACH-0144471 Treatment for 6 Months in Patients With C3 Glomerulopathy (C3G), With an Open-label Extension

CompletedPH IIAlexion Pharmaceuticals, Inc.n=132020

A 2-Part, Open-Label, Randomized, Single-Dose, 3-Period Crossover, Phase 1, Relative Bioavailability, and Food Effect Study, Comparing Different Formulations of Danicopan in Healthy Adult Subjects

CompletedPH IAlexion Pharmaceuticals, Inc.n=242020

Research activity

2 papers

Key research shaping understanding of this drug, combining the latest publications with the most influential evidence.

Publications over time
20212023
Major research themes4
Hemoglobinuria, Paroxysmal2Cholecystitis1Complement Factor D1COVID-191
Journals, researchers & institutions
Top journals
  • Haematologica1
  • The Lancet. Haematology1
Leading researchers
  • Kulasekararaj AG2
  • Lee JW2
  • Brodsky R1
  • Browett P1
  • Carrillo Infante C1
  • Filippov G1
  • Geffner M1
  • Griffin M1
Leading institutions
free-text, unnormalised
  • Federico II University of Naples2
  • Seoul St. Mary's Hospital2
  • Centre de Référence Aplasie Médullaire1
  • Cleveland Clinic1
  • Core Research Laboratory1
  • Johns Hopkins University School of Medicine1
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • Europe PMC — research literature
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.