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ALXN2060
ALXN2060 is a Complement C3 inhibitor Small molecule drug developed by Alexion Pharmaceuticals, Inc.. It is currently in Phase 3 development for Paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy. Also known as: AG10, Acoramidis.
ALXN2060 is a complement C3 inhibitor that blocks the central complement pathway to reduce inflammation and tissue damage.
ALXN2060 is a complement C3 inhibitor that blocks the central complement pathway to reduce inflammation and tissue damage. Used for Paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy.
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Baseline phase 3 → approval rate
+58.3pp
Industry-wide phase 3 drugs reach approval ~58.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas). -
Immunology slight uplift
+1.0pp
Mature endpoint landscape (ACR, DAS28, PASI) makes immunology approvals slightly more predictable.
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2028–2030 | — |
| EMA | EU | 2029–2031 | +0.7 yr |
| MHRA | GB | 2029–2031 | +0.7 yr |
| Health Canada | CA | 2029–2032 | +0.9 yr |
| TGA | AU | 2029–2032 | +1.2 yr |
| PMDA | JP | 2029–2032 | +1.5 yr |
| NMPA | CN | 2030–2033 | +2.3 yr |
| MFDS | KR | 2029–2032 | +1.4 yr |
| CDSCO | IN | 2029–2033 | +1.8 yr |
| ANVISA | BR | 2030–2033 | +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 | ALXN2060 |
|---|---|
| Also known as | AG10, Acoramidis |
| Sponsor | Alexion Pharmaceuticals, Inc. |
| Drug class | Complement C3 inhibitor |
| Target | Complement C3 |
| Modality | Small molecule |
| Therapeutic area | Immunology / Hematology |
| Phase | Phase 3 |
Mechanism of action
ALXN2060 selectively inhibits complement component C3, preventing its cleavage and activation of both the classical and alternative complement pathways. By targeting C3, the drug reduces downstream complement-mediated inflammation, cell lysis, and tissue injury. This mechanism is intended to treat complement-driven diseases including paroxysmal nocturnal hemoglobinuria (PNH) and other rare blood disorders.
Approved indications
- Paroxysmal nocturnal hemoglobinuria (PNH)
- C3 glomerulopathy
Common side effects
- Meningococcal infection
- Headache
- Fatigue
- Infusion-related reactions
Key clinical trials
- Study to Assess the Effect of Food on a Single Dose of Acoramidis in Healthy Adult Participants (PHASE1)
- Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant (PHASE3)
- A Phase 3 Study of ALXN2060 in Japanese Participants With Symptomatic ATTR-CM (PHASE3)
- Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants (PHASE3)
- To Evaluate the Long-term Safety and Tolerability of Acoramidis in Participants With Newly Diagnosed ATTR-CM (ACT-EARLY OLE) (PHASE3)
- Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy (PHASE3)
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:
- ALXN2060 CI brief — competitive landscape report
- ALXN2060 updates RSS · CI watch RSS
- Alexion Pharmaceuticals, Inc. portfolio CI
Frequently asked questions about ALXN2060
What is ALXN2060?
How does ALXN2060 work?
What is ALXN2060 used for?
Who makes ALXN2060?
Is ALXN2060 also known as anything else?
What drug class is ALXN2060 in?
What development phase is ALXN2060 in?
What are the side effects of ALXN2060?
What does ALXN2060 target?
Related
- Drug class: All Complement C3 inhibitor drugs
- Target: All drugs targeting Complement C3
- Manufacturer: Alexion Pharmaceuticals, Inc. — full pipeline
- Therapeutic area: All drugs in Immunology / Hematology
- Indication: Drugs for Paroxysmal nocturnal hemoglobinuria (PNH)
- Indication: Drugs for C3 glomerulopathy
- Also known as: AG10, Acoramidis
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing