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Pharming And Orphan Drugs

April 10, 2015

”What is “pharming”? What is the relationship between orphan drugs and pharming? This Blog Post defines pharming and gives examples of orphan drugs manufactured from pharming.

I – “Pharming” Is …

Pharming is the combination of the concepts of “farming” and “pharmaceutical”. Pharming is an inexpensive way for producing drugs from genetically altered plants or animals. According to an online Yahoo article:

“In general, the idea behind pharming (for plant-derived drugs) is to slip the genetic blueprints for a particular protein into a plant and let the plant’s protein-making machinery go to work. Then the protein can be extracted from plant tissues. While tobacco plants are a mainstay of such work, proteins also have been produced in other plants, such as safflower and potato.”

II – Ruconest For Hereditary Angioedema (HAE)

A successful animal-derived drug, is orphan drug Ruconest (C1-esterase inhibitor (recombinant)), developed in partnership by Salix Pharmaceuticals and Pharming N.V. Ruconest is purified from the milk of genetically modified (transgenic) rabbits. It is approved by the FDA (July 2014)  and commercialized since 2010 in Europe, for the treatment of HAE attacks. Ruconest is intended to restore the level of functional C1-esterase inhibitor in a patient’s plasma, thereby treating the acute attacks of swelling associated with HAE.

III – Atryn For Hereditary Antitrombin Deficiency

Another animal-derived drug is orphan drug Atryn. In February 2009, the FDA approves rEVO Biologics‘ Atryn (Recombinant Human Antithrombin) for the prevention of peri-operative and peri-partum thromboembolic events, in patients with hereditary antithrombin deficiency. Atryn is made from the milk of goats that have been genetically modified to produce human antithrombin, a plasma protein with anticoagulant properties.

IV – Elelyso For Gaucher Disease

A successful plant-derived drug is the orphan drug Elelyso (Taliglucerase Alfa). Elelyso is the first drug to win FDA approval (May 2012) for a drug produced inside modified plant cells (carrot cells) for a human disease. It is approved for the rare disease, Gaucher Disease. Elelyso is made by Israel’s Protalix Biotherapeutics, and marketed with Pfizer.

Protalix Biotherapeutics puts a normal version of the human gene that affects Gaucher Disease, into modified carrot cells and extracts the enzyme the cells produce. This enzyme is then processed into an injectable solution for long-term Enzyme Replacement Therapy (ERT).

V – ZMapp For The Ebola Virus

Another plant-derived drug is orphan drug ZMapp (August 2014). ZMapp is an experimental anti-Ebola drug developed by Mapp Biopharmaceutical, made in tobacco plants. Tobacco plants provide a fast and inexpensive way for producing new biotechnology treatments.

ZMapp is made up of three humanized monoclonal antibodies manufactured in plants, specifically Nicotiana or tobacco plants. The antibodies are manufactured in tobacco plants at Kentucky BioProcessing (Owensboro. Kentucky). It is an optimized cocktail that was first identified as a drug candidate in January 2014.

In February 2015, LeafBio, the commercial arm of Mapp Biopharmaceutical, announces that it receives approval of its application for a FDA Investigational New Drug (IND) application. The acceptance of the IND will allow for clinical trials of ZMapp to begin in Liberia. The U.S. National Institutes of Health (NIH) and the Liberian government will oversee the clinical trials.

Please Note: “Gerste im Fokusbereich” by Luca Jose Inverso (Own work) [CC-BY-SA-3.0] | Wikimedia Commons.

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.

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