Danicopan

also known as ACH-0144471 · ALXN2040 · ACH-4471 · Voydeya

Small molecule targets CFD via inhibition

Developed for: paroxysmal nocturnal hemoglobinuria · anemia, nonspherocytic hemolytic · C3 glomerulonephritis · dense deposit disease · immunoglobulin-mediated membranoproliferative glomerulonephritis · COVID-19 · diffuse secondary choroid atrophy · complement 3 glomerulopathy
Investigated by: Alexion Pharmaceuticals, Inc. · Achillion, a wholly owned subsidiary of Alexion · Celerion · AstraZeneca · National Institute of Allergy and Infectious Diseases (NIAID)

Regulatory milestones approvals, filings & regulatory actions · 2 recorded

Milestone	Jurisdiction	Brand	Indication	Date	Sentence it was read from
Approved	EU (EMA)	Voydeya	—	2024-04-19	europa.eu ↗
Approved	US (FDA)	VOYDEYA	—	2024-03-29	fda.gov ↗

Trials 26 · a red edge is where a trial was stopped

today

US approved 2024

EU approved 2024

NCT06449001 · Ph 3 NCT05708573 · Ph 1 NCT05389449 · Ph 3 NCT05019521 · Ph 2 NCT04988035 · Ph 2 NCT04469465 · Ph 3 NCT04609696 · Ph 1 NCT04451434 · Ph 1 NCT04551599 · Ph 1 NCT04709094 · Ph 1 NCT05109390 · Ph 1 NCT05016206 · Ph 1 NCT03459443 · Ph 2 NCT03472885 · Ph 2 NCT03555539 · Ph 1 NCT04935294 · Ph 1 NCT03384186 · Ph 1 NCT04889391 · Ph 1 NCT03124368 · Ph 2 NCT03181633 · Ph 2 NCT03108274 · Ph 1 NCT03053102 · Ph 2 NCT04940559 · Ph 1 NCT04889690 · Ph 1 NCT04889677 · Ph 1

201620172018201920202021202220232024202520262027

Phase	Registry id	Dates	Indication	Sponsor	Status	Outcome
Phase 115 trials
Phase 1	NCT05708573	Feb → Mar 2023	—	Alexion Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 1	NCT04609696	Oct → Dec 2020	—	Alexion Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 1	NCT04451434	Aug → Sep 2020	—	Alexion Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 1	NCT04551599	Feb 2020 → Feb 2021	—	Alexion Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 1	NCT04709094	Jul → Oct 2019	—	Alexion Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 1	NCT05109390	Jul → Oct 2018	—	Alexion Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 1	NCT05016206	Jul → Oct 2018	—	Alexion Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 1	NCT03555539	May → Sep 2018	—	Alexion Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 1	NCT04935294	Jan → May 2018	impaired renal function disease	Alexion Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 1	NCT03384186	Dec 2017 → Mar 2018	—	Alexion Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 1	NCT04889391	Sep → Oct 2017	—	Alexion Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 1	NCT03108274	Apr → Jun 2017	—	Alexion Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 1	NCT04940559	Aug → Oct 2016	—	Alexion Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 1	NCT04889690	May 2016 → Jan 2017	—	Alexion Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 1	NCT04889677	Feb → Jun 2016	—	Alexion Pharmaceuticals, Inc.	Completed	No outcome recorded

Phase 27 trials
Phase 2	NCT05019521	Aug 2021 → Jul 2024	diffuse secondary choroid atrophy	Alexion Pharmaceuticals, Inc.	Terminated	No outcome recorded
Phase 2	NCT04988035	Aug 2021 → Apr 2022	COVID-19	National Institute of Allergy and Infectious Diseases (NIAID)	Completed	No outcome recorded
Phase 2	NCT03459443	Jun 2018 → Mar 2021	C3 glomerulonephritis, dense deposit disease, immunoglobulin-mediated membranoproliferative glomerulonephritis	Alexion Pharmaceuticals, Inc.	Terminated	No outcome recorded
Phase 2	NCT03472885	May 2018 → Sep 2019	paroxysmal nocturnal hemoglobinuria	Alexion Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 2	NCT03124368	Aug 2017 → Dec 2018	C3 glomerulonephritis, dense deposit disease, immunoglobulin-mediated membranoproliferative glomerulonephritis, membranoproliferative glomerulonephritis	Alexion Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 2	NCT03181633	Jun 2017 → Jan 2022	paroxysmal nocturnal hemoglobinuria	Alexion Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 2	NCT03053102	Mar 2017 → Nov 2018	paroxysmal nocturnal hemoglobinuria	Alexion Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 33 trials
Phase 3	NCT06449001	Aug 2025 → May 2027 expected	anemia, nonspherocytic hemolytic, paroxysmal nocturnal hemoglobinuria	Alexion Pharmaceuticals, Inc.	Recruiting	No outcome recorded
Phase 3	NCT05389449	Oct 2022 → Aug 2026 overdue	paroxysmal nocturnal hemoglobinuria	Alexion Pharmaceuticals, Inc.	Active not recruiting	No outcome recorded
Phase 3	NCT04469465	Dec 2020 → Jun 2022	paroxysmal nocturnal hemoglobinuria	Alexion Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase not stated1 trial
—	NCT05982938	—	anemia, nonspherocytic hemolytic, paroxysmal nocturnal hemoglobinuria	Alexion Pharmaceuticals, Inc.	Available	No outcome recorded

Also used as a comparator or background therapy in 1 trial

Phase	Registry id	Dates	Indication	Sponsor	Status	Outcome
Phase 2	NCT03369236 Comparator	Jun 2018 → Dec 2019	C3 glomerulonephritis, dense deposit disease	Alexion Pharmaceuticals, Inc.	Completed	No outcome recorded

Press releases naming this drug 5 releases

Date	Issuer	Release
2024-04-23	AstraZeneca	Voydeya approved in the EU as add-on treatment to ravulizumab or eculizumab for adults with the rare disease PNH who have residual haemolytic anaemia astrazeneca.com ↗
2024-04-01	AstraZeneca	Voydeya approved in the US as add-on therapy to ravulizumab or eculizumab for treatment of extravascular haemolysis in adults with the rare disease PNH astrazeneca.com ↗
2024-02-26	AstraZeneca	Voydeya recommended for approval in the EU by CHMP as add-on treatment to ravulizumab or eculizumab for adults with PNH who have residual haemolytic anaemia astrazeneca.com ↗
2024-01-19	AstraZeneca	Voydeya (danicopan) granted first-ever regulatory approval in Japan for adults with PNH to be used in combination with C5 inhibitor therapy astrazeneca.com ↗
2023-12-11	AstraZeneca	Long-term ALPHA Phase III trial data showed danicopan as add-on to Ultomiris or Soliris sustained clinical improvements in subset of patients with PNH experiencing clinically significant extravascular haemolysis astrazeneca.com ↗

Evidence & citations 9 cited values

Every value below carries the sentence it was read from. 30 sources stand behind the page.

Field	Value	Cited text
Known as	Danicopan	ClinicalTrials.gov intervention name — accepted as the source's own label NCT03555539 ↗ 24 NCT04609696 ↗ NCT05019521 ↗ NCT04469465 ↗ NCT06449001 ↗ NCT04940559 ↗ NCT04889391 ↗ NCT03108274 ↗ NCT04889677 ↗ NCT05982938 ↗ NCT04709094 ↗ NCT03369236 ↗ NCT04988035 ↗ NCT05109390 ↗ NCT05016206 ↗ NCT03459443 ↗ NCT03472885 ↗ NCT04889690 ↗ NCT03124368 ↗ NCT03384186 ↗ NCT05389449 ↗ NCT04451434 ↗ NCT04935294 ↗ NCT03053102 ↗ NCT04551599 ↗
Known as	ACH-0144471	“The primary purpose of this study was to evaluate the efficacy of 12 months of oral ACH-0144471 (also known as danicopan and ALXN2040) in participants with C3G or IC-MPGN based...” NCT03459443 ↗ 2 “To determine the effectiveness of ACH-0144471 (also known as danicopan and ALXN2040) in improving anemia when given with eculizumab for 24 weeks in participants with PNH.” NCT03472885 ↗ NCT03181633 ↗
Known as	ACH-4471	ChEMBL registry synonym — accepted as the source's own label CHEMBL4250860 ↗
Known as	ALXN2040	“To determine the effectiveness of ACH-0144471 (also known as danicopan and ALXN2040) in improving anemia when given with eculizumab for 24 weeks in participants with PNH.” NCT03472885 ↗ 1 NCT05708573 ↗
Known as	Voydeya	ChEMBL registry synonym — accepted as the source's own label CHEMBL4250860 ↗
Action	Inhibit	“Danicopan is a first-in-class oral proximal, complement alternative pathway factor D (FD) inhibitor.” PMID 33121236 ↗ Dec 2021
Modality	Small molecule	“The reversible small-molecule inhibitor danicopan.” PMID 31573880 ↗ 2020
Route	Oral	“Danicopan was administered as multiple oral doses over a period of at least 28 days.” NCT03053102 ↗
Target	CFD	“Danicopan is a first-in-class oral proximal, complement alternative pathway factor D (FD) inhibitor.” PMID 33121236 ↗ Dec 2021