ULTOMIRIS
Ultomiris (ravulizumab-cwvz) is a complement inhibitor indicated for the treatment of adult and pediatric patients aged one month and older with paroxysmal nocturnal hemoglobinuria (PNH) or atypical hemolytic uremic syndrome (aHUS). It is also approved for adult patients with anti-acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG) and anti-aquaporin-4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD). The therapy is used to inhibit complement-mediated processes, though it is specifically not indicated for Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).
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.
Details
- Status
- Prescription
- First Approved
- 2018-12-21
- 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
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Trial Timeline
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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
ProIndications & Usage
FDA Label (PDF)ULTOMIRIS is a complement inhibitor indicated for: the treatment of adult and pediatric patients one month of age and older with paroxysmal nocturnal hemoglobinuria (PNH). the treatment of adult and pediatric patients one month of age and older with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA). Limitations of Use: ULTOMIRIS is not indicated for the treatment of patients with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS). the treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcho...
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.