Anticlotting Drug From Genetically Engineered Goats Is Safe Says FDA Panel

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Main Category: Regulatory Affairs / Drug Approvals
Also Included In: Blood / Hematology;  Pharma Industry / Biotech Industry;  Cardiovascular / Cardiology
Article Date: 12 Jan 2009 - 1:00 PDT

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An expert panel that advises the US Food and Drug Adminstration (FDA) voted that the anti-clotting drug ATryn, which is made from the milk of genetically engineered goats, is safe and effective and be approved for the treatment of people with a rare inherited disorder that makes them susceptible to life-threatening blood clots. The drug, which is made by the biotechnology company GTC Biotherapeutics Inc based in Massachusetts, is already approved in Europe.

ATryn is made from a human protein extracted from the milk of goats that have been genetically altered to produce it. If approved, the drug will be the first commercially available pharmaceutical in the US to be made from "transgenic" animals, that is animals that have had genetic material from another species (in this case from humans into goats) inserted in their DNA.

GTC is licensing the drug to OVATION, a biotech company based in Deerfield, Illinois who will be marketing the drug in the US. Both companies are seeking FDA approval for ATryn in the "prevention and treatment of venous thromboembolism in hereditary antithrombin deficient patients undergoing surgery or childbirth procedures", said a recent statement from GTC.

A majority of the 19-member FDA Blood Products Advisory Committee voted that the drug was safe and effective after reviewing data supplied by the manufacturer. The FDA does not have to follow the recommendation of its Advisory Committees but it usually does. Their decision is expected by 7th of February.

The panel's consumer representative, Dr Richard Colvin, who is a a clinical assistant in medicine at Massachusetts General Hospital, told the press that this action "set a precedent for what will happen in the future", reported Reuters.

Chairman and CEO of GTC, Dr Geoffrey F Cox said both companies were very pleased with the panel's recommendation:

"ATryn is the first transgenically produced therapeutic to achieve approval in Europe and undergo review by the FDA."

The new drug has the potential to provide " an important new treatment option for patients with hereditary antithrombin deficiency," he explained.

President and CEO of OVATION, Jeffrey S Aronin said the panel's decision meant they were a step closer to "making the drug available to people in the US with hereditary antithrombin deficiency, a rare clotting disorder associated with severe complications for which there are few treatment options".

Antithrombin works as a natural blood clotter or anticoagulant by controlling thrombin, which is key to the formation of blood clots. ATryn is a purified recombinant antithrombin with the same amino acid sequence as the antithrombin in human plasma, said a GTC statement.

People with hereditary antithrombin deficiency are more susceptible to venous blood clots, including pulmonary embolism and deep vein thrombosis (DVT). About one in two to three thousand of the general population has the disorder, half of whom are likely to experience a thrombosis by the time they are 25 years old, and according to one study cited by the GTC press statement, up to 85 per cent may experience an embolism before they are 51.

The drug is already approved for use in the European Union where the regulating authority is the European Medicines Agency (EMEA). The EMEA approved label carries information about the most common adverse events that may occur from use of ATryn. These include: dizziness, nausea, headache, bleeding, bleeding at the site of the injection, and increased bleeding during treatment.

GTC also warned that "as with any intravenous protein product, allergic type hypersensitivity reactions are possible".

Sources: Reuters, GTC Biotherapeutics.

Written by: Catharine Paddock, PhD


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