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alpha-1-Proteinase Inhibitor
Alpha-1-proteinase inhibitor (AAT) replaces or augments deficient alpha-1 antitrypsin to prevent elastin degradation in the lungs and other tissues.
Alpha-1-proteinase inhibitor (AAT) replaces or augments deficient alpha-1 antitrypsin to prevent elastin degradation in the lungs and other tissues. Used for Alpha-1 antitrypsin deficiency with emphysema, Alpha-1 antitrypsin deficiency with progressive lung disease.
At a glance
| Generic name | alpha-1-Proteinase Inhibitor |
|---|---|
| Also known as | Zemaira, Prolastin |
| Sponsor | Institute for Human Genetics and Biochemistry |
| Drug class | Protease inhibitor / Enzyme replacement therapy |
| Target | Neutrophil elastase |
| Modality | Biologic |
| Therapeutic area | Pulmonology / Genetic disease |
| Phase | FDA-approved |
Mechanism of action
Alpha-1 antitrypsin is a serine protease inhibitor that protects tissues from neutrophil elastase and other proteases. In alpha-1 antitrypsin deficiency, unopposed protease activity leads to progressive lung and liver damage. Exogenous AAT replacement therapy restores the protease-antiprotease balance, slowing or halting tissue destruction.
Approved indications
- Alpha-1 antitrypsin deficiency with emphysema
- Alpha-1 antitrypsin deficiency with progressive lung disease
Common side effects
- Infusion reactions
- Headache
- Dizziness
- Fever
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 |
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