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H5G1.1 (ECULIZUMAB)
Eculizumab-aeeb binds to complement protein C5, inhibiting its cleavage and preventing terminal complement complex formation.
Eculizumab (H5G1.1) is a complement inhibitor developed by Alexion Pharmaceuticals, targeting the C5 protein to prevent complement-mediated damage. It is a small molecule drug approved by the FDA in 2007 for various indications, including atypical hemolytic uremic syndrome, congenital hemolytic uremic syndrome, and paroxysmal nocturnal hemoglobinuria. Eculizumab is still owned by Alexion Pharmaceuticals and is not off-patent. It is used to treat conditions characterized by excessive complement activation, which can lead to tissue damage and organ failure. Key safety considerations include the risk of meningococcal infections and the potential for increased susceptibility to infections.
At a glance
| Generic name | ECULIZUMAB |
|---|---|
| Sponsor | AstraZeneca |
| Drug class | Complement Inhibitor [EPC] |
| Target | C5 |
| Modality | Monoclonal antibody |
| Therapeutic area | Neuroscience |
| Phase | FDA-approved |
| First approval | 2007 |
Mechanism of action
Eculizumab-aeeb is a monoclonal antibody that targets the complement protein C5. By binding to C5, it prevents the protein from being split into C5a and C5b, which stops the formation of the terminal complement complex C5b-9. This inhibition helps prevent conditions like intravascular hemolysis in PNH and TMA in aHUS. In gMG, the exact mechanism is unclear but likely involves reducing C5b-9 deposition at the neuromuscular junction.
Approved indications
- Atypical hemolytic uremic syndrome
- Congenital hemolytic uremic syndrome
- Neuromyelitis optica
- Paroxysmal nocturnal hemoglobinuria
- Refractory generalized myasthenia gravis
Boxed warnings
- WARNING: SERIOUS MENINGOCOCCAL INFECTIONS Eculizumab products, complement inhibitors, increase 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 BKEMV, unless the risks of delaying therapy with BKEMV 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 eculizumab products 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, BKEMV is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called BKEMV REMS [ see Warnings and Precautions (5.2) ]. WARNING: SERIOUS MENINGOCOCCAL INFECTIONS See full prescribing information for complete boxed warning Eculizumab products increase 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 BKEMV, unless the risks of delaying BKEMV outweigh the risk of developing a serious infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for meningococcal vaccination in patients receiving a complement inhibitor. ( 5.1 ) Patients receiving eculizumab products 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 meningococcal infections and evaluate immediately if infection is suspected. ( 5.1 ) BKEMV is available only through a restricted program called BKEMV REMS. ( 5.2 )
Common side effects
- Headache
- Nasopharyngitis
- Back pain
- Nausea
- Fatigue
- Cough
- Herpes simplex infections
- Sinusitis
- Respiratory tract infection
- Constipation
- Myalgia
- Pain in extremity
Key clinical trials
- Study of Safety in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated With Eculizumab (PHASE3)
- Open Label Controlled Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Resistant aHUS (PHASE2)
- An Open-Label, Multi-Center Clinical Trial of Eculizumab in Pediatric Patients With Atypical Hemolytic-Uremic Syndrome (PHASE2)
- Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-sensitive Atypical Hemolytic Uremic Syndrome (aHUS) (PHASE2)
- Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-Resistant aHUS (PHASE2)
- Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients (PHASE3)
- Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) (PHASE3)
- An Open-label, Multi-center Clinical Trial of Eculizumab in Adult Patients With Atypical Hemolytic-uremic Syndrome (PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- H5G1.1 CI brief — competitive landscape report
- H5G1.1 updates RSS · CI watch RSS
- AstraZeneca portfolio CI