ULTOMIRIS (ravulizumab-cwvz)
ULTOMIRIS is indicated for the treatment of Paroxysmal nocturnal hemoglobinuria (PNH); Atypical hemolytic uremic syndrome (aHUS); Anti-acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG); Anti-aquaporin-4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD).
How ULTOMIRIS Works
Ravulizumab-cwvz is a terminal complement inhibitor that binds with high affinity to the complement protein C5. This binding prevents the cleavage of C5 into C5a and C5b, which stops the formation of the membrane attack complex (MAC). By blocking MAC formation, the drug inhibits terminal complement-mediated intravascular hemolysis in PNH and thrombotic microangiopathy in aHUS. In gMG and NMOSD, the therapeutic effect is presumed to involve the reduction or inhibition of terminal complement complex deposition at the neuromuscular junction or other targeted tissues.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2018-12-21
- Patent Cliff
- 2028
- Routes
- INJECTION
- Dosage Forms
- INJECTABLE
ULTOMIRIS Approval History
What ULTOMIRIS Treats
4 indicationsULTOMIRIS is approved for 4 conditions since its original approval in 2018. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Paroxysmal nocturnal hemoglobinuria (PNH)
- Atypical hemolytic uremic syndrome (aHUS)
- Anti-acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG)
- Anti-aquaporin-4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD)
ULTOMIRIS Boxed Warning
SERIOUS MENINGOCOCCAL INFECTIONS ULTOMIRIS, a complement inhibitor, increases the risk of serious infections caused by Neisseria meningitidis [see Warnings and Precautions (5.1) ] . Life-threatening and fatal meningococcal infections have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if not recognized and treated early . Complete or update vaccination for meningococcal bacteria (for serogroups A, C, W, Y, and B) at least 2 ...
WARNING: SERIOUS MENINGOCOCCAL INFECTIONS ULTOMIRIS, a complement inhibitor, increases the risk of serious infections caused by Neisseria meningitidis [see Warnings and Precautions (5.1) ] . Life-threatening and fatal meningococcal infections have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if not recognized and treated early . Complete or update vaccination for meningococcal bacteria (for serogroups A, C, W, Y, and B) at least 2 weeks prior to the first dose of ULTOMIRIS, unless the risks of delaying therapy with ULTOMIRIS outweigh the risk of developing a serious infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against meningococcal bacteria in patients receiving a complement inhibitor. See Warnings and Precautions (5.1) for additional guidance on the management of the risk of serious infections caused by meningococcal bacteria. Patients receiving ULTOMIRIS are at increased risk for invasive disease caused by Neisseria meningitidis , even if they develop antibodies following vaccination. Monitor patients for early signs and symptoms of serious meningococcal infections and evaluate immediately if infection is suspected. Because of the risk of serious meningococcal infections, ULTOMIRIS is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called ULTOMIRIS and SOLIRIS REMS [see Warnings and Precautions (5.2) ]. WARNING: SERIOUS MENINGOCOCCAL INFECTIONS See full prescribing information for complete boxed warning. ULTOMIRIS increases the risk of serious and life-threatening infections caused by Neisseria meningitidis . Complete or update meningococcal vaccination at least 2 weeks prior to the first dose of ULTOMIRIS, unless the risks of delaying ULTOMIRIS outweigh the risks of developing a serious infection. Comply with the most current Advisory Committee on Immunization P
ULTOMIRIS Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Direct competitors
Same target(s) AND same indication — head-to-head.
MoA expansion candidates
Same target(s), different indications — where else is this mechanism being explored?
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
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Clinical Trial Registry
36 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT05133531 ACCESS-1 | R3918-PNH-2021 2020-004486-40, 2023-509657-31-00 | Ph 3 | active not recruiting | A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment |
| NCT07557420 | D9282C00006 ALXN1210-NMO-324 | Ph 3 | not yet recruiting | Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity Study of Ravulizumab in Chinese Adults With Neuromyelitis Optica Spectrum Disorder (NMOSD) |
| NCT06333652 | 22-009239 | Ph 2 | recruiting | Ravulizumab in Pregnancies Complicated by Severe Hypertensive Disorders |
| NCT06830798 AWAKE | D928EC00001 2024-517568-48 | Ph 3 | recruiting | Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab Administered Intravenously in Adult Participants at High Risk of Delayed Graft Function After Kidney Transplantation |
| NCT07308574 | D928BL00001 NEPH-ULT-501 | Ph 4 | recruiting | Post-Marketing Clinical Study of Ravulizumab in Participants With Clinical aHUS |
| NCT05644561 | ALXN1210-MG-319 | Ph 3 | active not recruiting | Evaluation of PK, PD, Efficacy, Safety, and Immunogenicity of IV Ravulizumab in Pediatric Participants With Generalized Myasthenia Gravis |
| NCT06291376 I CAN | D928FC00001 | Ph 3 | recruiting | Study of Ravulizumab in Immunoglobulin A Nephropathy (IgAN) |
| NCT05746559 ARTEMIS | D928DC00001 ALXN1210-CSA-AKI-318, 2022-501802-36 | Ph 3 | active not recruiting | ARTEMIS: Ravulizumab to Protect Patients With CKD From CSA-AKI and MAKE |
| NCT07221838 OCTAGON | D9281R00005 ALX-MG-502 | Ph 4 | recruiting | A Study to Investigate OCS Tapering in Adult Participants With Generalized Myasthenia Gravis Treated With Ravulizumab |
| NCT04543591 | ALXN1210-TMA-313 | Ph 3 | completed | Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant |
| NCT07024563 | D928FC00002 2024-520167-13-00 | Ph 3 | recruiting | Study of Ravulizumab in Pediatric Participants With Primary IgAN |
| NCT07010302 BEST-NMOSD | 40200 | Ph 4 | not yet recruiting | Rituximab Versus Ravulizumab, Inebilizumab, Satralizumab, and Eculizumab in NMOSD |
| NCT04564339 SANCTUARY results posted | ALXN1210-NEPH-202 2020-001537-13 | Ph 2 | terminated | Study of Ravulizumab in Proliferative Lupus Nephritis (LN) or Immunoglobulin A Nephropathy (IgAN) |
| NCT06578949 | D9289C00008 ALXN1210-PNH-323 | Ph 3 | completed | Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) |
| NCT04557735 results posted | ALXN1210-TMA-314 2020-000761-16 | Ph 3 | completed | Study of Ravulizumab in Pediatric Participants With HSCT-TMA |
| NCT05346354 | ALXN1210-NMO-317 | Ph 2, Ph 3 | recruiting | Efficacy and Safety Study of Ravulizumab IV in Pediatric Participants With NMOSD |
| NCT04201262 results posted | ALXN1210-NMO-307 CHAMPION-NMO-307, 2019-003352-37 | Ph 3 | completed | An Efficacy and Safety Study of Ravulizumab in Adult Participants With NMOSD |
| NCT04999020 results posted | ALXN1210-DM-310 2021-001200-15 | Ph 2, Ph 3 | terminated | Ravulizumab Versus Placebo in Adult Participants With Dermatomyositis |
| NCT04702568 results posted | BCX9930-201 2020-000501-93 | Ph 2 | terminated | A Long-Term Safety Study of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) |
| NCT05131204 ACCESS 2 results posted | R3918-PNH-2022 2020-002761-33 | Ph 3 | terminated | Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria |
| NCT03056040 results posted | ALXN1210-PNH-302 2016-002026-36 | Ph 3 | completed | ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab |
| NCT05116774 REDEEM-1 results posted | BCX9930-202 2020-004438-39 | Ph 2 | terminated | BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants With Inadequate Response to C5 Inhibitor Therapy |
| NCT04558918 APPLY-PNH results posted | CLNP023C12302 2019-004665-40 | Ph 3 | completed | Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment |
| NCT04743804 results posted | ALXN1210-TMA-315 2020-005328-13 | Ph 3 | terminated | Ravulizumab in Thrombotic Microangiopathy Associated With a Trigger |
| NCT03748823 results posted | ALXN1210-PNH-303 2017-002370-39 | Ph 3 | completed | Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab |
| NCT04320602 results posted | ALXN1210-PNH-401 2019-003440-74 | Ph 4 | completed | Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab |
| NCT03920293 results posted | ALXN1210-MG-306 2018-003243-39 | Ph 3 | completed | Safety and Efficacy Study of Ravulizumab in Adults With Generalized Myasthenia Gravis |
| NCT02946463 results posted | ALXN1210-PNH-301 2016-002025-11 | Ph 3 | completed | ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) |
| NCT03131219 results posted | ALXN1210-aHUS-312 2016-002499-29 | Ph 3 | completed | Study of Ravulizumab in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS) |
| NCT02949128 results posted | ALXN1210-aHUS-311 2016-002027-29 | Ph 3 | completed | Study of ALXN1210 in Complement Inhibitor Treatment-Naïve Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS) |
| NCT05396742 results posted | ALXN1210-HV-105 2017-004931-35 | Ph 1 | completed | Pharmacokinetic Study of Ravulizumab Administered Subcutaneously With Recombinant Human Hyaluronidase PH20 (rHuPH20) in Healthy Adult Volunteers |
| NCT03406507 results posted | ALXN1210-PNH-304 2017-002820-26 | Ph 3 | completed | A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria |
| NCT04248465 results posted | ALXN1210-ALS-308 2019-004619-30 | Ph 3 | terminated | An Efficacy and Safety Study of Ravulizumab in ALS Participants |
| NCT02605993 results posted | ALXN1210-PNH-201 2015-002674-20 | Ph 2 | completed | Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) |
| NCT04369469 results posted | ALXN1210-COV-305 | Ph 3 | terminated | Efficacy and Safety Study of IV Ravulizumab in Patients With COVID-19 Severe Pneumonia |
| NCT07596784 | D9281C00003 ALXN1210-MG-326 | Ph 3 | not yet recruiting | Efficacy and Safety of Ravulizumab in Chinese Adults Participants With Generalized Myasthenia Gravis (gMG) |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
ULTOMIRIS FDA Label Details
Indications & Usage
FDA Label (PDF)ULTOMIRIS is indicated for the treatment of Paroxysmal nocturnal hemoglobinuria (PNH); Atypical hemolytic uremic syndrome (aHUS); Anti-acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG); Anti-aquaporin-4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD).
WARNING: SERIOUS MENINGOCOCCAL INFECTIONS ULTOMIRIS, a complement inhibitor, increases the risk of serious infections caused by Neisseria meningitidis [see Warnings and Precautions (5.1) ] . Life-threatening and fatal meningococcal infections have occurred in patients treated with complement inhibit...
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment