ATryn

A recombinant form of human antithrombin used as a medication

ATryn
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INN
Drug class
Routes of administration	Intravenous
Pregnancy category
Bioavailability
Metabolism
Elimination half-life
Excretion
Legal status
CAS Number
PubChem
DrugBank	DB00030
ChemSpider
KEGG

ATryn is a recombinant form of human antithrombin, a protein that plays a critical role in regulating blood coagulation. It is produced using genetically modified goats and is used as a medication to prevent thromboembolic events in patients with hereditary antithrombin deficiency.

Production

Angora goat used in the production of ATryn

ATryn is produced using a transgenic technology where the gene for human antithrombin is inserted into the DNA of goats. These goats are then able to produce antithrombin in their milk. The milk is collected and processed to extract and purify the antithrombin protein. This method of production allows for large-scale manufacturing of the protein, which is otherwise difficult to obtain in sufficient quantities from human plasma.

Mechanism of Action

Antithrombin is a serine protease inhibitor that inactivates several enzymes of the coagulation cascade, including thrombin and factor Xa. By inhibiting these enzymes, antithrombin helps to prevent the formation of blood clots. ATryn, as a recombinant form of antithrombin, functions in the same way as the naturally occurring protein.

Clinical Use

ATryn is primarily used in patients with hereditary antithrombin deficiency, a rare genetic disorder that increases the risk of venous thromboembolism. It is administered intravenously and is often used during high-risk situations such as surgery or childbirth to prevent thromboembolic events.

Approval and Regulation

ATryn was the first therapeutic product produced using transgenic animals to be approved by the United States Food and Drug Administration (FDA). It was approved in 2009 and is classified as a prescription-only medication.

Advantages and Challenges

The use of transgenic animals for the production of therapeutic proteins offers several advantages, including the ability to produce complex proteins that are difficult to synthesize using traditional methods. However, it also presents challenges such as ethical concerns, regulatory hurdles, and the need for specialized facilities to manage the transgenic animals.

Related pages

• Antithrombin
• Thromboembolism
• Recombinant DNA
• Transgenic animals