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Alpha-1 antitrypsin (AAT)
Alpha-1 antitrypsin (AAT) is a protease inhibitor that replaces deficient endogenous AAT to prevent neutrophil elastase-mediated tissue destruction in the lungs and other organs.
Alpha-1 antitrypsin (AAT) is a protease inhibitor that replaces deficient endogenous AAT to prevent neutrophil elastase-mediated tissue destruction in the lungs and other organs. Used for Alpha-1 antitrypsin deficiency with emphysema, Alpha-1 antitrypsin deficiency with progressive lung disease, Alpha-1 antitrypsin deficiency-related liver disease.
At a glance
| Generic name | Alpha-1 antitrypsin (AAT) |
|---|---|
| Also known as | Alpha-1 proteinase inhibitor (A1-P1) |
| Sponsor | CSL Behring |
| Drug class | Protease inhibitor / Protein replacement therapy |
| Target | Neutrophil elastase |
| Modality | Biologic |
| Therapeutic area | Pulmonology / Genetic / Rare Disease |
| Phase | Phase 3 |
Mechanism of action
AAT is a serine protease inhibitor naturally produced by the liver that protects tissues from degradation by neutrophil elastase released during inflammation. In alpha-1 antitrypsin deficiency, insufficient AAT levels allow unopposed elastase activity, leading to progressive lung and liver damage. Augmentation therapy with exogenous AAT restores protective capacity and slows disease progression.
Approved indications
- Alpha-1 antitrypsin deficiency with emphysema
- Alpha-1 antitrypsin deficiency with progressive lung disease
- Alpha-1 antitrypsin deficiency-related liver disease
Common side effects
- Infusion reactions
- Headache
- Fatigue
- Dizziness
- Injection site reactions
Key clinical trials
- Role of Genetic Factors in the Development of Lung Disease
- A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD) (PHASE1, PHASE2)
- Gene Therapy for Alpha 1- Antitrypsin Deficiency (PHASE1)
- A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD) (PHASE1)
- Phase 4, Double-blind Study Evaluating the Response on Computed Tomography (CT) Lung Density Decline Rates of Respreeza / Zemaira Weekly for 3 Years in Adults With alpha1 Antitrypsin Deficiency (AATD) (PHASE4)
- Study to Learn About the Safety of Fazirsiran and if it Can Help People With Alpha-1 Antitrypsin Liver Disease With Mild Liver Scarring (Fibrosis) (PHASE3)
- Study to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein (PHASE3)
- Efficacy and Safety of Alpha1-Proteinase Inhibitor (Human), Modified Process (Alpha-1 MP) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency (AATD) (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:
- Alpha-1 antitrypsin (AAT) CI brief — competitive landscape report
- Alpha-1 antitrypsin (AAT) updates RSS · CI watch RSS
- CSL Behring portfolio CI