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CAEL-101
CAEL-101 is a transthyretin (TTR) stabilizer that binds to and stabilizes the transthyretin protein, preventing its misfolding and amyloid fibril formation.
CAEL-101 is a transthyretin (TTR) stabilizer that binds to and stabilizes the transthyretin protein, preventing its misfolding and amyloid fibril formation. Used for Transthyretin cardiac amyloidosis (ATTR-CM), Hereditary transthyretin amyloidosis (hATTR).
At a glance
| Generic name | CAEL-101 |
|---|---|
| Also known as | Anselamimab |
| Sponsor | Alexion Pharmaceuticals, Inc. |
| Drug class | Transthyretin (TTR) stabilizer |
| Target | Transthyretin (TTR) |
| Modality | Small molecule |
| Therapeutic area | Rare Disease / Amyloidosis |
| Phase | Phase 3 |
Mechanism of action
CAEL-101 works by directly binding to transthyretin tetramers and stabilizing their native conformation, which prevents dissociation into monomers that would otherwise misfold and aggregate into amyloid fibrils. This mechanism halts the progression of transthyretin amyloidosis by reducing the formation of toxic amyloid deposits that damage tissues, particularly the heart and peripheral nerves. By stabilizing the protein rather than clearing existing amyloid, it addresses the root cause of disease progression.
Approved indications
- Transthyretin cardiac amyloidosis (ATTR-CM)
- Hereditary transthyretin amyloidosis (hATTR)
Common side effects
- Peripheral edema
- Dyspnea
- Fatigue
- Diarrhea
Key clinical trials
- A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis (CARES) (PHASE3)
- A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis (CARES) (PHASE3)
- A Study to Evaluate the Safety and Tolerability of CAEL-101 in Patients With AL Amyloidosis (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 |
|---|---|
| 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:
- CAEL-101 CI brief — competitive landscape report
- CAEL-101 updates RSS · CI watch RSS
- Alexion Pharmaceuticals, Inc. portfolio CI