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Alpha 1-Antitrypsin
Alpha 1-Antitrypsin is a Protease inhibitor; protein replacement therapy Small molecule drug developed by University of Minnesota. It is currently FDA-approved for Alpha 1-antitrypsin deficiency with emphysema or chronic obstructive pulmonary disease (COPD), Alpha 1-antitrypsin deficiency with progressive lung disease. Also known as: Aralast NP, Glassia, Kamada alpha 1-antitrypsin for inhalation, AAT.
Alpha 1-antitrypsin is a protease inhibitor that replaces deficient or dysfunctional alpha 1-antitrypsin protein to prevent neutrophil elastase-mediated tissue destruction in the lungs.
Alpha-1-Antitrypsin is a protein used to treat conditions such as Liver Fibrosis, Alpha-1 Antitrypsin Deficiency, and Chronic Obstructive Pulmonary Disease. It is administered through an intravenous catheter and is also being studied as an oligonucleotide modality, specifically as Fazirsiran Sodium.
At a glance
| Generic name | Alpha 1-Antitrypsin |
|---|---|
| Also known as | Aralast NP, Glassia, Kamada alpha 1-antitrypsin for inhalation, AAT, Prolastin C |
| Sponsor | University of Minnesota |
| Drug class | Protease inhibitor; protein replacement therapy |
| Target | Neutrophil elastase |
| Modality | Small molecule |
| Therapeutic area | Pulmonology; Genetic/Metabolic Disorders |
| Phase | FDA-approved |
Mechanism of action
Alpha 1-antitrypsin (AAT) is a serine protease inhibitor naturally produced by the liver that protects lung tissue from degradation by neutrophil elastase released during inflammation. In patients with alpha 1-antitrypsin deficiency, insufficient levels of this protective protein lead to progressive lung damage. Replacement therapy restores adequate AAT levels to inhibit elastase activity and 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
- 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 CI brief — competitive landscape report
- Alpha 1-Antitrypsin updates RSS · CI watch RSS
- University of Minnesota portfolio CI
Frequently asked questions about Alpha 1-Antitrypsin
What is Alpha 1-Antitrypsin?
How does Alpha 1-Antitrypsin work?
What is Alpha 1-Antitrypsin used for?
Who makes Alpha 1-Antitrypsin?
Is Alpha 1-Antitrypsin also known as anything else?
What drug class is Alpha 1-Antitrypsin in?
What development phase is Alpha 1-Antitrypsin in?
What are the side effects of Alpha 1-Antitrypsin?
What does Alpha 1-Antitrypsin target?
Related
- Drug class: All Protease inhibitor; protein replacement therapy drugs
- Target: All drugs targeting Neutrophil elastase
- Manufacturer: University of Minnesota — full pipeline
- Therapeutic area: All drugs in Pulmonology; Genetic/Metabolic Disorders
- Indication: Drugs for Alpha 1-antitrypsin deficiency with emphysema or chronic obstructive pulmonary disease (COPD)
- Indication: Drugs for Alpha 1-antitrypsin deficiency with progressive lung disease
- Also known as: Aralast NP, Glassia, Kamada alpha 1-antitrypsin for inhalation, AAT, Prolastin C
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing