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Alpha1-Proteinase Inhibitor
Alpha1-proteinase inhibitor (AAT) replaces deficient or dysfunctional alpha-1 antitrypsin protein to protect lung tissue from enzymatic degradation.
Alpha1-proteinase inhibitor (AAT) replaces deficient or dysfunctional alpha-1 antitrypsin protein to protect lung tissue from enzymatic degradation. Used for Alpha-1 antitrypsin deficiency with emphysema or chronic obstructive pulmonary disease (COPD), Alpha-1 antitrypsin deficiency with progressive lung disease.
At a glance
| Generic name | Alpha1-Proteinase Inhibitor |
|---|---|
| Also known as | ARALAST Fr.IV-1, ARALAST NP, GLASSIA, Zemaira®, Aralast NP |
| Sponsor | Baxalta now part of Shire |
| Drug class | Protease inhibitor (protein replacement therapy) |
| Target | Neutrophil elastase (indirect target via AAT inhibition) |
| Modality | Biologic |
| Therapeutic area | Pulmonology / Respiratory |
| Phase | FDA-approved |
Mechanism of action
Alpha-1 antitrypsin (AAT) is a serine protease inhibitor that normally protects lung tissue from neutrophil elastase and other proteases released during inflammation. In alpha-1 antitrypsin deficiency, insufficient AAT levels allow unopposed protease activity, leading to progressive lung destruction. Exogenous AAT replacement therapy augments circulating and lung AAT levels to slow or prevent emphysema progression.
Approved indications
- Alpha-1 antitrypsin deficiency with emphysema or chronic obstructive pulmonary disease (COPD)
- Alpha-1 antitrypsin deficiency with progressive lung disease
Common side effects
- Infusion reactions
- Headache
- Dizziness
- Fever
- Fatigue
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:
- Alpha1-Proteinase Inhibitor CI brief — competitive landscape report
- Alpha1-Proteinase Inhibitor updates RSS · CI watch RSS
- Baxalta now part of Shire portfolio CI