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ALXN1210
ALXN1210 is a Complement inhibitor Small molecule drug developed by Alexion Pharmaceuticals, Inc.. It is currently in Phase 1 development for Paroxysmal Nocturnal Hemoglobinuria (PNH). Also known as: Ultomiris, Ravulizumab.
Complement inhibitor
ALXN1210 is a complement C5 inhibitor, classified as an antibody inhibitor. It is being studied in clinical trials for various conditions, including COVID-19 severe pneumonia, acute lung injury, and acute respiratory distress syndrome.
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Baseline phase 1 → approval rate
+9.6pp
Industry-wide phase 1 drugs reach approval ~9.6% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas). -
Rare-disease pathway favourability
+5.0pp
Rare-disease drugs benefit from FDA Orphan Drug Act, smaller pivotal trials, and more flexible endpoints. Approval rates run ~5pp above baseline.
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2033–2036 | — |
| EMA | EU | 2034–2037 | +0.7 yr |
| MHRA | GB | 2034–2037 | +0.7 yr |
| Health Canada | CA | 2034–2038 | +0.9 yr |
| TGA | AU | 2034–2038 | +1.2 yr |
| PMDA | JP | 2034–2038 | +1.5 yr |
| NMPA | CN | 2035–2039 | +2.3 yr |
| MFDS | KR | 2034–2038 | +1.4 yr |
| CDSCO | IN | 2034–2039 | +1.8 yr |
| ANVISA | BR | 2035–2039 | +2.3 yr |
Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).
Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.
At a glance
| Generic name | ALXN1210 |
|---|---|
| Also known as | Ultomiris, Ravulizumab |
| Sponsor | Alexion Pharmaceuticals, Inc. |
| Drug class | Complement inhibitor |
| Target | C5 |
| Modality | Small molecule |
| Therapeutic area | Rare Disease |
| Phase | Phase 1 |
Mechanism of action
ALXN1210 is a recombinant form of the human C5 protein, which inhibits the complement system by binding to C5 and preventing its cleavage into C5a and C5b.
Approved indications
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
Common side effects
- Anemia
- Thrombocytopenia
- Neutropenia
Key clinical trials
- 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 (PHASE3)
- Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant (PHASE3)
- Study of Ravulizumab in Immunoglobulin A Nephropathy (IgAN) (PHASE3)
- A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works (PHASE3)
- ARTEMIS: Ravulizumab to Protect Patients With CKD From CSA-AKI and MAKE (PHASE3)
- Study of Ravulizumab in Pediatric Participants With Primary IgAN (PHASE3)
- Rituximab Versus Ravulizumab, Inebilizumab, Satralizumab, and Eculizumab in NMOSD (PHASE4)
- Ravulizumab Treatment Outcomes in Patients With Generalized Myasthenia Gravis
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 |
|---|---|
| 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:
- ALXN1210 CI brief — competitive landscape report
- ALXN1210 updates RSS · CI watch RSS
- Alexion Pharmaceuticals, Inc. portfolio CI
Frequently asked questions about ALXN1210
What is ALXN1210?
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Is ALXN1210 also known as anything else?
What drug class is ALXN1210 in?
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What does ALXN1210 target?
Related
- Drug class: All Complement inhibitor drugs
- Target: All drugs targeting C5
- Manufacturer: Alexion Pharmaceuticals, Inc. — full pipeline
- Therapeutic area: All drugs in Rare Disease
- Indication: Drugs for Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Also known as: Ultomiris, Ravulizumab
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing