Drugs / Ravulizumab
Regulatory milestones approvals, filings & regulatory actions · 8 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed generalized myasthenia gravis 1 milestone | |||||
| Filed | US (FDA) | Ultomiris | generalized myasthenia gravis | 2021-12-21 | “for Ultomiris (ravulizumab-cwvz) in adults with generalised myasthenia gravis (gMG) has been accepted for Priority Review” astrazeneca.com ↗ |
| Filed IgA glomerulonephritis 1 milestone | |||||
| Filed | US (FDA) | Ultomiris | IgA glomerulonephritis | 2026-06-15 | “supplemental Biologics License Application (sBLA) for Ultomiris (ravulizumab) has been accepted and granted Priority Review by the US Food and Drug Administration (FDA)” astrazeneca.com ↗ |
| Filed neuromyelitis optica spectrum disorder with anti-AQP4 antibodies 3 milestones | |||||
| Filed | EU (EMA) | Ultomiris | neuromyelitis optica spectrum disorder with anti-AQP4 antibodies | — | “submissions for Ultomiris for the treatment of NMOSD are currently under review with multiple health authorities, including in the United States (US), European Union (EU)” astrazeneca.com ↗ |
| Filed | Japan (PMDA) | Ultomiris | neuromyelitis optica spectrum disorder with anti-AQP4 antibodies | — | “submissions for Ultomiris for the treatment of NMOSD are also currently under review with multiple health authorities, including in the United States (US) and Japan.” astrazeneca.com ↗ “Ultomiris for the treatment of NMOSD are currently under review with multiple health authorities, including in the United States (US), European Union (EU) and Japan” astrazeneca.com ↗ |
| Filed | US (FDA) | Ultomiris | neuromyelitis optica spectrum disorder with anti-AQP4 antibodies | — | “submissions for Ultomiris for the treatment of NMOSD are also currently under review with multiple health authorities, including in the United States (US) and Japan.” astrazeneca.com ↗ “Regulatory submissions for Ultomiris for the treatment of NMOSD are currently under review with multiple health authorities, including in the United States (US)” astrazeneca.com ↗ |
| Filed paroxysmal nocturnal hemoglobinuria 2 milestones | |||||
| Filed | EU (EMA) | — | paroxysmal nocturnal hemoglobinuria | — | “submissions have been filed in the U.S. and EU for Alexion's ravulizumab (formerly ALXN1210) for the treatment of patients with paroxysmal nocturnal hemoglobinuria” xencor.com ↗ |
| Filed | US (FDA) | — | paroxysmal nocturnal hemoglobinuria | — | “submissions have been filed in the U.S. and EU for Alexion's ravulizumab (formerly ALXN1210) for the treatment of patients with paroxysmal nocturnal hemoglobinuria” xencor.com ↗ |
| Approved Indication not stated 1 milestone | |||||
| Approved | EU (EMA) | Ultomiris | — | 2019-07-02 | europa.eu ↗ |
Trials 39
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 15 trials | ||||||
| Phase 1 | NCT05396742 | Aug 2018 → May 2019 | — | Alexion Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 1 | NCT05288829 | Aug 2016 → Jul 2017 | — | Alexion Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 1 | NCT05288816 | Apr 2016 → Jul 2017 | — | Alexion Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 1 | NCT05288673 | Jul 2015 → Sep 2016 | — | Alexion Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 1 | NCT05288660 | Aug 2014 → Mar 2015 | — | Alexion Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT02598583 | Nov 2015 → Jul 2016 | paroxysmal nocturnal hemoglobinuria | Alexion Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 23 trials | ||||||
| Phase 2 | NCT06333652 | Sep 2026 → Dec 2028 expected | preeclampsia | Mayo Clinic | Recruiting | No outcome recorded |
| Phase 2 | NCT04564339 | Jan 2021 → Mar 2025 | IgA glomerulonephritis, lupus nephritis | Alexion Pharmaceuticals, Inc. | Terminated | Mixed Stop: Business |
| Phase 2 | NCT02605993 | Jan 2016 → Feb 2017 | paroxysmal nocturnal hemoglobinuria | Alexion Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 2/32 trials · 1 missed | ||||||
| Phase 2/3 | NCT05346354 | Jun 2022 → Jun 2026 overdue | neuromyelitis optica spectrum disorder with anti-AQP4 antibodies | Alexion Pharmaceuticals, Inc. | Active not recruiting | No outcome recorded |
| Phase 2/3 | NCT04999020 | Nov 2021 → Oct 2023 | dermatomyositis | Alexion Pharmaceuticals, Inc. | Terminated | Missed primary Stop: Business |
| Phase 324 trials · 6 met primary · 3 missed | ||||||
| Phase 3 | NCT07596784 | Jul 2026 → Aug 2027 expected | generalized myasthenia gravis | Alexion Pharmaceuticals, Inc. | Recruiting | No outcome recorded |
| Phase 3 | NCT07557420 | Jul 2026 → Sep 2028 expected | neuromyelitis optica spectrum disorder with anti-AQP4 antibodies | Alexion Pharmaceuticals, Inc. | Recruiting | No outcome recorded |
| Phase 3 | NCT07024563 | Jun 2025 → Jul 2028 expected | IgA glomerulonephritis, immunoglobulin A vasculitis | Alexion Pharmaceuticals, Inc. | Recruiting | No outcome recorded |
| Phase 3 | NCT06830798 | May 2025 → Nov 2027 expected | — | Alexion Pharmaceuticals, Inc. | Recruiting | No outcome recorded |
| Phase 3 | NCT06578949 | Oct 2024 → May 2025 | paroxysmal nocturnal hemoglobinuria | Alexion Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 3 | NCT06291376 | Mar 2024 → Jan 2026 overdue | IgA glomerulonephritis | Alexion Pharmaceuticals, Inc. | Active not recruiting | Met primary |
| Phase 3 | NCT05644561 | Jun 2023 → Jul 2026 overdue | generalized myasthenia gravis | Alexion Pharmaceuticals, Inc. | Active not recruiting | No outcome recorded |
| Phase 3 | NCT05746559 | Apr 2023 → Oct 2025 | chronic kidney disease, heart disorder | Alexion Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 3 | NCT05131204 | Oct 2022 → Jul 2023 | paroxysmal nocturnal hemoglobinuria | Regeneron Pharmaceuticals | Terminated | No outcome recorded Stop: Business |
| Phase 3 | NCT05133531 | Aug 2022 → Oct 2026 expected | paroxysmal nocturnal hemoglobinuria | Regeneron Pharmaceuticals | Active not recruiting | No outcome recorded |
| Phase 3 | NCT04743804 | Jun 2021 → Dec 2022 | thrombotic microangiopathy | Alexion Pharmaceuticals, Inc. | Terminated | No outcome recorded Stop: Enrollment |
| Phase 3 | NCT04570397 | Dec 2020 → May 2023 | COVID-19, acute kidney injury, thrombotic microangiopathy | Brigham and Women's Hospital | Unknown | No outcome recorded |
| Phase 3 | NCT04543591 | Dec 2020 → Sep 2025 | thrombotic microangiopathy | Alexion Pharmaceuticals, Inc. | Completed | Missed primary |
| Phase 3 | NCT04557735 | Dec 2020 → Nov 2024 | thrombotic microangiopathy | Alexion Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 3 | NCT04369469 | May 2020 → Feb 2021 | COVID-19, acute lung injury, adult acute respiratory distress syndrome, viral pneumonia | Alexion Pharmaceuticals, Inc. | Terminated | Missed primary |
| Phase 3 | NCT04248465 | Mar 2020 → Oct 2021 | amyotrophic lateral sclerosis | Alexion Pharmaceuticals, Inc. | Terminated | Missed primary |
| Phase 3 | NCT04201262 | Dec 2019 → Mar 2022 | neuromyelitis optica | Alexion Pharmaceuticals, Inc. | Completed | Met primary |
| Phase 3 | NCT03920293 | Mar 2019 → May 2021 | generalized myasthenia gravis | Alexion Pharmaceuticals, Inc. | Completed | Met primary |
| Phase 3 | NCT03748823 | Feb 2019 → Feb 2021 | paroxysmal nocturnal hemoglobinuria | Alexion Pharmaceuticals, Inc. | Completed | Met primary |
| Phase 3 | NCT03406507 | Feb 2018 → Mar 2020 | paroxysmal nocturnal hemoglobinuria | Alexion Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 3 | NCT03131219 | Aug 2017 → Dec 2022 | atypical hemolytic-uremic syndrome | Alexion Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 3 | NCT03056040 | May 2017 → Feb 2022 | paroxysmal nocturnal hemoglobinuria | Alexion Pharmaceuticals, Inc. | Completed | Met primary |
| Phase 3 | NCT02949128 | Jan 2017 → Jan 2023 | atypical hemolytic-uremic syndrome | Alexion Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 3 | NCT02946463 | Dec 2016 → Feb 2023 | paroxysmal nocturnal hemoglobinuria | Alexion Pharmaceuticals, Inc. | Completed | Met primary |
| Phase 44 trials · 1 missed | ||||||
| Phase 4 | NCT07010302 | Sep 2026 → Jan 2030 expected | neuromyelitis optica spectrum disorder with anti-AQP4 antibodies | Massachusetts General Hospital | Recruiting | No outcome recorded |
| Phase 4 | NCT07308574 | Dec 2025 → Jun 2027 expected | atypical hemolytic-uremic syndrome | Alexion Pharmaceuticals, Inc. | Recruiting | No outcome recorded |
| Phase 4 | NCT04320602 | Apr 2021 → Dec 2022 | paroxysmal nocturnal hemoglobinuria | Alexion Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 4 | NCT04390464 | May 2020 → May 2021 | COVID-19 | Cambridge University Hospitals NHS Foundation Trust | Unknown | Missed primary |
| Comparator or background therapy5 trials | ||||||
| Phase 2 | NCT05116774 Comparator | Dec 2021 → Sep 2023 | paroxysmal nocturnal hemoglobinuria | BioCryst Pharmaceuticals | Terminated | No outcome recorded Stop: Business |
| Phase 2 | NCT04702568 Background | Dec 2020 → Oct 2023 | paroxysmal nocturnal hemoglobinuria | BioCryst Pharmaceuticals | Terminated | No outcome recorded Stop: Business |
| Phase 3 | NCT04558918 Comparator | Jan 2021 → Sep 2022 | paroxysmal nocturnal hemoglobinuria | Novartis Pharmaceuticals | Completed | Met primary |
| Phase 3 | NCT04469465 Background | Dec 2020 → Jun 2022 | paroxysmal nocturnal hemoglobinuria | Alexion Pharmaceuticals, Inc. | Completed | Met primary |
| Phase 4 | NCT07221838 Background | Mar 2026 → Aug 2027 expected | generalized myasthenia gravis | Alexion Pharmaceuticals, Inc. | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 28
| Date | Issuer | Release |
|---|---|---|
| 2026-07-27 | AstraZeneca | Results Update on Phase III trial of Ultomiris in adults and adolescents with thrombotic microangiopathy after haematopoietic stem cell transplant astrazeneca.com ↗
High-level results from the ALXN1210-TMA-313 Phase III clinical trial showed that Ultomiris (ravulizumab) did not achieve statistical significance for the primary endpoint of event-free survival through 26 weeks compared to placebo in adults and adolescents (aged 12 years or older) with thrombotic microangiopathy after haematopoietic stem cell transplant (HSCT-TMA). |
| 2026-06-15 | AstraZeneca | Regulatory Ultomiris granted Priority Review in the US as treatment for adults with immunoglobulin A nephropathy astrazeneca.com ↗
Alexion, AstraZeneca Rare Disease’s supplemental Biologics License Application (sBLA) for Ultomiris (ravulizumab) has been accepted and granted Priority Review by the US Food and Drug Administration (FDA) for the treatment of adults with immunoglobulin A nephropathy (IgAN). |
| 2026-06-06 | AstraZeneca | Results Ultomiris demonstrated 43.4% reduction in proteinuria vs placebo in adults with immunoglobulin A nephropathy at 34 weeks in I CAN Phase III trial astrazeneca.com ↗
The reduction in proteinuria was rapid, observed as early as week 10 with Ultomiris (36.7% [95% CI: 30.2%, 42.6%]) and sustained through 34 weeks, compared to placebo (8.5% [95% CI: 0.5%, 15.8%]). |
| 2026-04-21 | AstraZeneca | Results Ultomiris demonstrated statistically significant and clinically meaningful reduction of proteinuria in adults with immunoglobulin A nephropathy in I CAN Phase III trial astrazeneca.com ↗
Ultomiris delivered rapid reduction in proteinuria as early as week 10 |
| 2025-09-19 | AstraZeneca | Results Alexion data presented at ECTRIMS 2025 reinforces Ultomiris leadership in transforming care for patients with NMOSD astrazeneca.com ↗
Final results of CHAMPION-NMOSD Phase III trial long-term extension will show zero relapses in patients on Ultomiris through a median follow-up of 170.3 weeks |
| 2025-06-21 | AstraZeneca | Results Alexion data presented at EAN 2025 highlights its leadership and commitment to improving patient outcomes in rare neurological diseases astrazeneca.com ↗
Data show reduction in steroid burden and event-related hospitalisations in adult patients with AChR-Ab+ gMG treated with Ultomiris (ravulizumab) |
| 2024-12-07 | Regeneron Pharmaceuticals | Results Novel Combination of Pozelimab and Cemdisiran (Poze-Cemdi) Achieved Greater Control of Intravascular Hemolysis in Patients with Paroxysmal Nocturnal Hemoglobinuria Compared to Ravulizumab regeneron.com ↗
Head-to-head exploratory cohort of a Phase 3 trial showed first-in-class poze-cemdi combination treatment helped patients achieve and maintain greater disease control, as measured by lactate dehydrogenase (LDH) levels, compared to standard-of-care ravulizumab |
| 2024-03-25 | AstraZeneca | Regulatory Ultomiris approved in the US for the treatment of adults with neuromyelitis optica spectrum disorder (NMOSD) astrazeneca.com ↗
Ultomiris (ravulizumab-cwvz) has been approved in the United States (US) as the first and only long-acting C5 complement inhibitor for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD). |
| 2023-11-29 | AstraZeneca | Results AstraZeneca advances scientific leadership in haematology at ASH 2023 astrazeneca.com ↗
Results will suggest Ultomiris improved survival and maintained effective long-term control of IVH, the most significant contributor to morbidity and premature mortality in PNH. |
| 2023-09-06 | AstraZeneca | Regulatory Update on US regulatory review of Ultomiris in NMOSD astrazeneca.com ↗
The FDA requested modifications to enhance the Ultomiris Risk Evaluation and Mitigation Strategy (REMS) to further validate patients’ meningococcal vaccination status or prophylactic administration of antibiotics prior to treatment. |
| 2023-05-26 | AstraZeneca | Regulatory Ultomiris approved in Japan for the prevention of relapses in patients with neuromyelitis optica spectrum disorder (NMOSD) astrazeneca.com ↗
Ultomiris (ravulizumab) has been approved in Japan as the first and only long-acting C5 complement inhibitor for the prevention of relapses in patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD), including neuromyelitis optica. |
| 2023-05-10 | AstraZeneca | Regulatory Ultomiris approved in the EU for adults with neuromyelitis optica spectrum disorder (NMOSD) astrazeneca.com ↗
Ultomiris (ravulizumab) has been approved in the European Union (EU) as the first and only long-acting C5 complement inhibitor for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD). |
| 2023-04-03 | AstraZeneca | Regulatory Ultomiris recommended for approval in the EU by CHMP for the treatment of adults with neuromyelitis optica spectrum disorder (NMOSD) astrazeneca.com ↗
Ultomiris (ravulizumab) has been recommended for marketing authorisation in the European Union (EU) for the treatment of adult patients with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive (Ab+). |
| 2022-10-27 | AstraZeneca | Results Ultomiris showed zero relapses in adults with neuromyelitis optica spectrum disorder (NMOSD) with median treatment duration of 73 weeks astrazeneca.com ↗
Results from CHAMPION-NMOSD trial demonstrated Ultomiris reduced the risk of relapse in AQP4 Ab+ NMOSD by 98.6% compared to placebo |
| 2022-10-24 | Novartis Pharmaceuticals | Results Novartis investigational oral monotherapy iptacopan demonstrates clinically meaningful superiority over anti-C5 treatment in Phase III APPLY-PNH study novartis.com ↗ |
| 2022-10-12 | AstraZeneca | Results Alexion aims to advance NMOSD treatment landscape with exceptional Ultomiris efficacy data at ECTRIMS 2022 astrazeneca.com ↗
Results from CHAMPION-NMOSD trial demonstrated zero relapses with a median treatment duration of 73 weeks |
| 2022-09-23 | AstraZeneca | Regulatory Ultomiris approved in Europe for the treatment of adults with generalised myasthenia gravis astrazeneca.com ↗
Ultomiris (ravulizumab) has been approved in Europe as an add-on to standard therapy for the treatment of adult patients with generalised myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive. |
| 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 ↗
Danicopan plus Ultomiris or Soliris demonstrated superiority compared to placebo plus Ultomiris or Soliris for this specific patient population, with statistically significant and clinically meaningful improvements in haemoglobin levels, transfusion avoidance and FACIT Fatigue scores from baseline. |
| 2022-08-25 | AstraZeneca | Regulatory Ultomiris approved in Japan for the treatment of adults with generalised myasthenia gravis astrazeneca.com ↗
Ultomiris (ravulizumab) has been approved in Japan for the treatment of adult patients with generalised myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive and whose symptoms are difficult to control with high-dose intravenous immunoglobulin therapy (IVIg) or plasmaphaeresis. |
| 2022-07-25 | AstraZeneca | Regulatory Ultomiris recommended for approval in the EU by CHMP for the treatment of adults with generalised myasthenia gravis astrazeneca.com ↗
Ultomiris (ravulizumab) has been recommended for marketing authorisation in the European Union (EU) as an add-on to standard therapy for the treatment of adult patients with generalised myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive. |
| 2022-06-10 | Sobi, Inc. | Results New data reinforce robust efficacy and safety profile of Aspaveli/Empaveli (pegcetacoplan) for PNH at the EHA2022 Congress sobi.com ↗
Patients treated with Aspaveli/Empaveli showed significant improvements compared to C5 inhibitors across all key disease measures evaluated, including lactate dehydrogenase normalisation, haemoglobin stabilisation and transfusion avoidance at week 26. |
| 2022-05-05 | AstraZeneca | Results Ultomiris met primary endpoint in CHAMPION-NMOSD Phase III trial in adults with neuromyelitis optica spectrum disorder astrazeneca.com ↗
Ultomiris , the first and only long-acting C5 complement inhibitor, met the primary endpoint of time to first on-trial relapse, as confirmed by an independent adjudication committee. |
| 2022-04-28 | AstraZeneca | Regulatory Ultomiris approved in the US for adults with generalised myasthenia gravis astrazeneca.com ↗
Ultomiris (ravulizumab-cwvz) has been approved in the US for the treatment of adult patients with generalised myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive, which represents 80% of people living with the disease. |
| 2022-04-06 | AstraZeneca | Results Ultomiris demonstrated sustained improvements in functional activities and quality of life in adults with generalised myasthenia gravis through 60 weeks astrazeneca.com ↗ |
| 2021-12-21 | AstraZeneca | Regulatory Ultomiris regulatory submission accepted under FDA Priority Review in the US for adults with generalised myasthenia gravis astrazeneca.com ↗
The supplemental Biologics License Application (sBLA) for Ultomiris (ravulizumab-cwvz) in adults with generalised myasthenia gravis (gMG) has been accepted for Priority Review by the US Food and Drug Administration (FDA). |
| 2021-09-03 | AstraZeneca | Regulatory Ultomiris approved in the EU for children and adolescents with paroxysmal nocturnal haemoglobinuria astrazeneca.com ↗
Ultomiris (ravulizumab) has been approved in the European Union for expanded use to include children (with a body weight of 10 kg or above) and adolescents with paroxysmal nocturnal haemoglobinuria (PNH), an ultra-rare and severe blood disorder characterised by the destruction of red blood cells that can cause thrombosis (blood clots) and result in organ damage and potentially premature death. |
| 2021-08-20 | AstraZeneca | Results Update on CHAMPION-ALS Phase III trial of Ultomiris in amyotrophic lateral sclerosis astrazeneca.com ↗
The IDMC recommended that the trial be discontinued due to lack of efficacy. |
| 2021-07-26 | AstraZeneca | Regulatory Ultomiris recommended for approval in the EU by CHMP for children and adolescents with paroxysmal nocturnal haemoglobinuria astrazeneca.com ↗
Alexion’s Ultomiris (ravulizumab) has been recommended for marketing authorisation in the European Union (EU) for expanded use to include children and adolescents with paroxysmal nocturnal haemoglobinuria (PNH). |
All press releases naming this drug 38 releases
Evidence & citations 8 cited values
Every value below carries the sentence it was read from. 46 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Ravulizumab | “Participants will continue to receive their ongoing C5 inhibitor (eculizumab or ravulizumab) therapy according to their usual dose and schedule.” NCT04469465 ↗ |
| Known as | ALXN1210 | “ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)” NCT02946463 ↗ |
| Known as | Ravulizumab cwvz | ChEMBL registry synonym — accepted as the source's own label CHEMBL3989986 ↗ |
| Known as | Ultomiris | “Ultomiris (Ravulizumab), is a monoclonal antibody that specifically targets terminal complement products” NCT04570397 ↗ |
| Action | Inhibit | “complement inhibitor” NCT02598583 ↗ |
| Modality | Monoclonal antibody | “a humanized monoclonal antibody to complement component C5” PMID 30171081 ↗ Sep 2018 |
| Route | Intravenous | “All treatments were given as IV infusions.” NCT02605993 ↗ |
| Target | C5 | “complement component C5” PMID 30171081 ↗ Sep 2018 |