NAV

Recombinant alpha 1-antitrypsin

Identification

Summary

Recombinant alpha 1-antitrypsin is a medication indicated to treat patients with chronic alpha1-proteinase inhibitor deficiency.

Generic Name
Recombinant alpha 1-antitrypsin
DrugBank Accession Number
DB05481
Background

Alpha 1-antitrypsin is a glycoprotein primarily produced by hepatocytes, and to a lesser extent, immune system cells. Recombinant alpha 1-antitrypsin (rAAT) is produced from yeast, which unlike commercially available forms of plasma-derived AAT, eliminates the risk associated with blood-borne infectious agents and allows for a increased manufacturing. rAAT belongs to a family of structurally-related proteins classified as serine protease inhibitors or SERPINS, which are known to inhibit several proteases including trypsin, cathepsin G, thrombin, tissue kallikrein, as well as neutrophil elastase. The proteinase/antiproteinase balance is believed to be important for maintaining healthy skin. The rAAT topical gel (Dermolastin™) is indicated for patients with atopic dermatitis and psoriasis. The company has indicated that other formulations for gastroenterological and urological indications will also be developed.

Type
Biotech
Groups
Investigational
Biologic Classification
Protein Based Therapies
Other protein based therapies
Protein Chemical Formula
Not Available
Protein Average Weight
Not Available
Sequences
Not Available
Synonyms
  • Recombinant alpha 1-antitrypsin

Pharmacology

Indication

Investigated for use/treatment in alpha 1 antitrypsin deficiency, atopic dermatitis, and chronic obstructive pulmonary disease (COPD).

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Associated Conditions
Contraindications & Blackbox Warnings
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Pharmacodynamics

Alpha 1-antitrypsin is a glycoprotein primarily produced by hepatocytes, and to a lesser extent, immune system cells. Recombinant alpha 1-antitrypsin (rAAT) is produced from yeast, which unlike commercially available forms of plasma-derived AAT, eliminates the risk associated with blood-borne infectious agents and allows for a increased manufacturing. rAAT belongs to a family of structurally-related proteins classified as serine protease inhibitors or SERPINS, which are known to inhibit several proteases including trypsin, cathepsin G, thrombin, tissue kallikrein, as well as neutrophil elastase. The proteinase/antiproteinase balance is believed to be important for maintaining healthy skin.

Mechanism of action

rAAT belongs to a family of structurally-related proteins classified as serine protease inhibitors or SERPINS, which are known to inhibit several proteases including trypsin, cathepsin G, thrombin, tissue kallikrein, as well as neutrophil elastase.

TargetActionsOrganism
UAlpha-1-antitrypsinNot AvailableHumans
Absorption

Not Available

Volume of distribution

Not Available

Protein binding

Not Available

Metabolism
Not Available
Route of elimination

Not Available

Half-life

Not Available

Clearance

Not Available

Adverse Effects
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Toxicity

Well tolerated in clinical trials.

Pathways
Not Available
Pharmacogenomic Effects/ADRs
Not Available

Interactions

Drug Interactions
This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.
Not Available
Food Interactions
Not Available

Products

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International/Other Brands
Dermolastin

Categories

Drug Categories
Not Available
Chemical TaxonomyProvided by Classyfire
Description
Not Available
Kingdom
Organic Compounds
Super Class
Organic Acids
Class
Carboxylic Acids and Derivatives
Sub Class
Amino Acids, Peptides, and Analogues
Direct Parent
Peptides
Alternative Parents
Not Available
Substituents
Not Available
Molecular Framework
Not Available
External Descriptors
Not Available
Affected organisms
Not Available

Chemical Identifiers

UNII
Not Available
CAS number
Not Available

References

General References
  1. Brown WM: rAAt (dermatological) Arriva/ProMetic. Curr Opin Mol Ther. 2006 Feb;8(1):69-75. [Article]
  2. Brown WM: rAAt (inhaled) Arriva/Hyland Immuno. Curr Opin Mol Ther. 2006 Feb;8(1):76-82. [Article]
PubChem Substance
347910164

Clinical Trials

Clinical Trials
PhaseStatusPurposeConditionsCount
1, 2CompletedTreatmentAlpha-1 Anti-trypsin Deficiency2

Pharmacoeconomics

Manufacturers
Not Available
Packagers
Not Available
Dosage Forms
FormRouteStrength
Powder, for solutionIntravenous1000 MG/40ML
Powder, for solutionParenteral1000 MG/40ML
Powder, for solution
Powder, for solutionIntravenous; Parenteral4000 MG
Powder, for solutionIntravenous; Parenteral5000 MG
Solution, concentrateIntravenous1 g
Prices
Not Available
Patents
Not Available

Properties

State
Solid
Experimental Properties
Not Available

Targets

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Details1. Alpha-1-antitrypsin
Kind
Protein
Organism
Humans
Pharmacological action
Unknown
General Function
Serine-type endopeptidase inhibitor activity
Specific Function
Inhibitor of serine proteases. Its primary target is elastase, but it also has a moderate affinity for plasmin and thrombin. Irreversibly inhibits trypsin, chymotrypsin and plasminogen activator. T...
Gene Name
SERPINA1
Uniprot ID
P01009
Uniprot Name
Alpha-1-antitrypsin
Molecular Weight
46736.195 Da

Drug created at November 18, 2007 18:25 / Updated at September 29, 2023 14:17