alpha1-proteinase inhibitor   

GtoPdb Ligand ID: 7487

Synonyms: A1AT | A1PI | alpha 1-antitrypsin | Aralast® | Prolastin® | serum trypsin inhibitor
alpha1-proteinase inhibitor is an approved drug
Comment: This is the human protein isolated and purified from donor plasma. There are 4 approved versions (Prolastin-C [7], Zemaira [6], Aralast [4] and Glassia [5]), which show minor chemical differences as a result of the methods used during purification [2]. PEGylated recombinant alpha-1-proteinase inhibitor has been tested [1,3], but no recombinant protein drug as been approved to date.
US FDA approval dates: Prolastin-C (1987), Zemaira (2003), Aralast (2010) and Glassia (2010).
No information available.
Summary of Clinical Use
Exogenous alpha-1-proteinase inhibitor is used to treat emphysemic patients with severe congenital enzyme deficiency (aka augmentation therapy). Alpha-1-proteinase inhibitor deficiency is associated with progressive and ultimately fatal emphysema, due to unfettered neutrophil elastase breakdown of elastin in lung tissue. Enzyme deficiency is also associated with several forms of liver disease, including cirrhosis, neonatal hepatitis, and hepatocellular carcinoma. If intravenous augmentation therapy is unsucessful, organ transplantation may be the only option for patients with end-stage lung or liver disease.
Mechanism Of Action and Pharmacodynamic Effects
Inhibits neutrophil elastase in the lungs. Currently available drugs are administered intravenously. Inhalation formulations are being evaluated as a method of delivering the drug directly to the lungs.