Recombinant Alpha-1-antitrypsin
Also known as: AAT augmentation therapy (recombinant), Alpha-1-proteinase inhibitor (recombinant), Alpha‑1‑antitrypsin, rAAT, recombinant AAT, Recombinant human alpha‑1‑antitrypsin, rhA1AT, SERPINA1, SERPINA1 recombinant protein
Source Recombinant Alpha-1-antitrypsin at Peptiology
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Summary
Recombinant human alpha‑1‑antitrypsin (rhA1AT) is a genetically engineered SERPIN designed to replace deficient endogenous A1AT in patients with A1AT deficiency, aiming to reduce emphysema progression and liver injury.
Mechanism of Action
Inhibits neutrophil elastase and other proteases, protecting lung and liver tissue from proteolytic damage.
Routes of Administration
Goals & Uses
- Type 1 diabetes / islet protectionMetabolic/autoimmune DiseaseLow
- Lung inflammation and emphysema preventionPulmonaryModerate
- Reduce liver fibrosis in A1AT deficiencyHepaticLow
- Graft-versus-host disease (GvHD) prevention/treatmentImmunomodulationModerate
- Alpha-1-antitrypsin deficiency (AATD) augmentationGenetic/metabolic DiseaseModerate
- Prevent progression of emphysema in A1AT deficiencyPulmonaryModerate
- Inflammatory bowel diseaseGastroenterologyLow
Contraindications
- Hypersensitivity to AAT or excipientsAllergy/immunologyHigh
- IgA deficiency with anti-IgA antibodiesImmunologicalModerate
- Active malignancy requiring immunosuppression cautionOncologyModerate
- Known hypersensitivity to recombinant A1AT or any excipientAllergyHigh
- Severe congestive heart failureCardiovascularModerate
Adverse Effects
- AnaphylaxisImmunologicRareSevere life-threatening allergic reaction
- HeadacheNeurologicUncommonPain in the head or upper neck
- NauseaGastrointestinalUncommonFeeling of sickness or urge to vomit
- Infusion‑related reactions (e.g., flushing, rash)GeneralCommon
- Elevated liver enzymesHepaticRareIncrease in AST/ALT or other hepatic markers
- Infusion-related reactionsHypersensitivityUncommon
- DizzinessNeurologicUncommonFeeling faint, lightheaded, or unsteady
- Immunogenicity / antibody formationImmunologicUnknown
Drug Interactions
- Other protease inhibitorsModerate
- Immunosuppressants (e.g., corticosteroids, tacrolimus)Moderate
Population Constraints
- PregnancyReproductive SafetyRelative
- Pediatric patientsAgeRelative
- Pregnant womenReproductiveRelative
- Renal or hepatic impairmentOrgan DysfunctionRelative
Regulatory Status
- European UnionInvestigationalPhase III trials ongoing.
- United StatesInvestigationalUnder IND for A1AT deficiency.
- United KingdomInvestigationalClinical trials authorized.
Plasma-derived AAT products are FDA-approved for AATD; recombinant forms (e.g., produced in yeast, CHO cells, or transgenic systems) are investigational. No rAAT product has received FDA or EMA approval as of the knowledge cutoff. Orphan drug designation has been sought for some rAAT programs.
Evidence & Sources
No sources recorded yet.
Frequently Asked Questions
What is Recombinant Alpha-1-antitrypsin?
Recombinant human alpha‑1‑antitrypsin (rhA1AT) is a genetically engineered SERPIN designed to replace deficient endogenous A1AT in patients with A1AT deficiency, aiming to reduce emphysema progression and liver injury.
What is Recombinant Alpha-1-antitrypsin used for?
Recombinant Alpha-1-antitrypsin is educationally associated with: Type 1 diabetes / islet protection, Lung inflammation and emphysema prevention, Reduce liver fibrosis in A1AT deficiency, Graft-versus-host disease (GvHD) prevention/treatment, Alpha-1-antitrypsin deficiency (AATD) augmentation, Prevent progression of emphysema in A1AT deficiency, Inflammatory bowel disease. Educational only — not medical advice.
How is Recombinant Alpha-1-antitrypsin administered?
Recorded routes of administration: Inhaled, Intravenous, Subcutaneous.
What are the potential side effects of Recombinant Alpha-1-antitrypsin?
Reported adverse effects include: Anaphylaxis, Headache, Nausea, Infusion‑related reactions (e.g., flushing, rash), Elevated liver enzymes, Infusion-related reactions, Dizziness, Immunogenicity / antibody formation. This list is not exhaustive — consult a qualified clinician.
Who should avoid Recombinant Alpha-1-antitrypsin?
Recorded contraindications: Hypersensitivity to AAT or excipients, IgA deficiency with anti-IgA antibodies, Active malignancy requiring immunosuppression caution, Known hypersensitivity to recombinant A1AT or any excipient, Severe congestive heart failure. Consult a qualified clinician before use.