The U.S. Drug Administration (FDA) has in some way the stall door to a new era drug therapy should open. A herd of 200 goats to produce future drugs that protect patients with congenital antithrombin deficiency against fatal blood clots. More products will follow.
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The European Medicines Agency EMEA ATryn ® has indeed in 2006, admitted last year it was in Germany on the market. The turnover is so far small, which is unlikely to change even after the U.S. approval of the drug.
Because treatment with antithrombin alfa, the active substance in ATryn, is rarely necessary. First, the congenital antithrombin deficiency is not very often. The FDA estimates that between one in 5000 adults in the formation of the thrombin antagonist is reduced for genetic reasons.
This requires a degree of thrombophilia. The press release of the producer, half of those affected suffered before 25 Age of thrombosis until the 50th Years it will be even 85 percent.
A therapy for prevention of thromboembolic complications is (currently), however, considered only in high-risk situations as indicated. These are major operations, or (at the U.S. approval) and obstetric interventions, the drug is usually combined with heparin, which improves the protection against thrombosis.
The effectiveness of ATryn was in a relatively small study in only 31 patients examined with congenital antithrombin deficiency, 30 of which were successfully protected against a need of treatment of thrombosis - most common side effect: bleeding and reactions at the injection site.
The specificity of ATryn is the fact that the recombinant protein is not like any other drugs by using genetically engineered bacteria or mammalian cultures is produced, but is made from the milk of goats. The production is not in huge tanks in a sterile factory, but on a small farm in Massachusetts.
The researchers introduced the gene into fertilized eggs and have them fight it, of surrogate animals. Since then, it is in all cells of the genetically modified animals available and later the gene product is excreted in milk, in an amount of 10 grams per liter, as they say. An animal produces in a year, the same amount as antithrombin contained in 90,000 blood units.
The so called pharming bietet sich als eine kostengünstige Alternative zur derzeitigen Produktion an. Denn die Musterfarmen sind (im Prinzip) wesentlich kostengünstiger als die Fabriken. Steigt die Nachfrage an Medikamenten, kann die Produktion durch eine Vergrößerung der Herden jederzeit gesteigert werden (“Breed more if you need more”).
Da sich im Rahmen des Pharmings (im Prinzip) alle gentechnischen Medikamente herstellen lassen, wundert es, dass bisher nicht weitere Firmen ihr Interesse angekündigt haben. Beobachter erwarten, dass die Leitlinien, welche das Center for Veterinary Medicine der FDA im Januar 2009 festgelegt hat, hier einen gewissen Anreiz schaffen, da sie die Bedingungen festlegen.
Pharming, a small company based in Leiden in the Netherlands will, reportedly produced a recombinant human C1 inhibitor (for treatment of hereditary angioedema) using rabbits. PharmAthene of Annapolis, Maryland is planning the production of a recombinant protective antigen as an antidote for anthrax poisoning, which is to be produced in goats. These products would serve only a small niche indication.
would be much larger market for the many monoclonal antibodies, which came in the last few years for the treatment of autoimmune diseases and cancer on the market. But the big manufacturers show little apparent interest.
Whether they will one day competition of biosimilar companies will grow to bring their products cheaply by Pharming in the market, it is difficult to predict. It should, in any case that the high costs are due to the development of drugs than on biotechnology as the clinical development.
Pharming could be associated with its own risks. This should be less in the possibility that some animals escaped into the wilderness and the therapeutic genes to spread in the environment. Much unpleasant could be a scenario where the production of essential medicines for humans fail, because the animals are given because of illness no more milk.
© rme/aerzteblatt.de / FDA
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