Drugs / Danicopan
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 25
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 114 trials | ||||||
| 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 · 1 met primary · 2 missed | ||||||
| Phase 2 | NCT05019521 | Aug 2021 → Jul 2024 | diffuse secondary choroid atrophy | Alexion Pharmaceuticals, Inc. | Terminated | Missed primary |
| Phase 2 | NCT04988035 | Aug 2021 → Apr 2022 | COVID-19 | National Institute of Allergy and Infectious Diseases (NIAID) | Completed | Missed primary |
| 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 | Met primary |
| Phase 33 trials · 1 met primary | ||||||
| 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 | Met primary |
| Phase not stated1 trial | ||||||
| — | NCT05982938 | — | anemia, nonspherocytic hemolytic, paroxysmal nocturnal hemoglobinuria | Alexion Pharmaceuticals, Inc. | Available | No outcome recorded |
| Comparator or background therapy1 trial | ||||||
| Phase 2 | NCT03369236 Comparator | Jun 2018 → Dec 2019 | C3 glomerulonephritis, dense deposit disease | Alexion Pharmaceuticals, Inc. | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 11
| Date | Issuer | Release |
|---|---|---|
| 2025-12-04 | AstraZeneca | Results AstraZeneca advances haematology and cell therapy ambition with largest-ever presence at ASH astrazeneca.com ↗
Danicopan add-on therapy demonstrates positive efficacy and safety outcomes in advanced age adults with paroxysmal nocturnal hemoglobinuria and clinically significant extravascular hemolysis: A sub-analysis of the phase 3 ALPHA trial |
| 2024-11-07 | AstraZeneca | Results AstraZeneca showcases strength of haematology portfolio and pipeline at ASH 2024 astrazeneca.com ↗
New, long-term data for Voydeya as add-on to Ultomiris or Soliris will show low rate of breakthrough haemolysis events and sustained improvements in quality-of-life measures in PNH with extravascular haemolysis |
| 2024-04-23 | AstraZeneca | Regulatory 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 ↗
Voydeya (danicopan) has been approved in the European Union (EU) as an add-on to ravulizumab or eculizumab for the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have residual haemolytic anaemia. |
| 2024-04-01 | AstraZeneca | Regulatory 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 ↗
Voydeya (danicopan) has been approved in the US as add-on therapy to ravulizumab or eculizumab for the treatment of extravascular haemolysis (EVH) in adults with paroxysmal nocturnal haemoglobinuria (PNH). |
| 2024-02-26 | AstraZeneca | Regulatory 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 ↗
Voydeya (danicopan) has been recommended for marketing authorisation in the European Union (EU) as an add-on to ravulizumab or eculizumab for the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have residual haemolytic anaemia. |
| 2024-01-19 | AstraZeneca | Regulatory Voydeya (danicopan) granted first-ever regulatory approval in Japan for adults with PNH to be used in combination with C5 inhibitor therapy astrazeneca.com ↗
Voydeya (danicopan) has been approved in Japan for the treatment of paroxysmal nocturnal haemoglobinuria (PNH). |
| 2023-12-11 | AstraZeneca | Results 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 ↗
Data showed that improvements in mean haemoglobin levels and absolute reticulocyte count (ARC) levels, which were demonstrated at 12 weeks, were maintained through 48 weeks. |
| 2023-11-29 | AstraZeneca | Results AstraZeneca advances scientific leadership in haematology at ASH 2023 astrazeneca.com ↗
Further, patient-reported outcomes from the ALPHA trial will suggest danicopan as an add-on to Ultomiris or Soliris improved quality of life compared to C5 inhibitor therapy alone in patients with PNH who experience clinically significant EVH. |
| 2023-06-09 | AstraZeneca | Results Danicopan as add-on to Ultomiris or Soliris improved haemoglobin levels and maintained disease control in patients with PNH experiencing signs or symptoms of clinically significant extravascular haemolysis astrazeneca.com ↗
All key secondary endpoints met statistical superiority, showing danicopan reduced fatigue and anaemia as well as the need for transfusions |
| 2023-05-16 | AstraZeneca | Results AstraZeneca showcases scientific advances across rare and malignant haematological conditions at EHA 2023 astrazeneca.com ↗
Pivotal ALPHA Phase III trial results will show benefit of danicopan, Alexion's first-in- class Factor D inhibitor, in improving clinically significant extravascular haemolysis while allowing patients with PNH to remain on standard of care treatment |
| 2022-09-16 | AstraZeneca | Results Danicopan (ALXN2040) add-on to Ultomiris or Soliris met primary endpoint in ALPHA Phase III trial for patients with paroxysmal nocturnal haemoglobinuria who experience clinically significant extravascular haemolysis astrazeneca.com ↗
The trial met its primary endpoint of change in haemoglobin from baseline at 12 weeks and key secondary endpoints, including transfusion avoidance and change in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue score. |
All press releases naming this drug 11 releases
Evidence & citations 9 cited values
Every value below carries the sentence it was read from. 29 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Danicopan | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03108274 ↗ |
| 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 ↗ |
| 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 ↗ |
| 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 |