FRAMINGHAM, Mass.--(BUSINESS WIRE)--Feb. 23, 2006
GTC Biotherapeutics, Inc. ("GTC", Nasdaq: GTCB) announced today that it has been told that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMEA)
intends to issue a negative opinion on the market authorization application (MAA) for ATryn(R), GTC's recombinant form of human antithrombin. On the basis of recent conversations, GTC understands that the CHMP, after excluding data from pregnant patients, determined that an insufficient number of surgical patients were enrolled to support approval. In addition, GTC understands that the CHMP has concerns about sufficient immunologic data and the lack of clinical data from ATryn(R)produced with an additional filtration step. GTC intends to take advantage of the appeal process to request a CHMP re-examination of GTC's submission.
GTC's European development and commercialization partner, LEO Pharma A/S, has affirmed that they remain committed to their collaboration with GTC and will continue to pursue the primary goal of
development of an acquired deficiency indication for Europe. Acquired deficiency indications represent the most significant market opportunity for the product. GTC and LEO entered into a collaborative development and commercialization agreement for ATryn(R) in Europe, Canada, and the Middle East in November 2005. GTC is also continuing a multinational study of ATryn(R) in hereditary antithrombin deficient patients in preparation for submission of a Biologics License
Application (BLA) in the U.S. Enrollment is planned to be completed in 2006 and a BLA submitted in the first half of 2007. "Although we are very disappointed with the late-breaking concerns
on the clinical data and the negative opinion, we are pleased by the commitment of LEO Pharma to continue working on further development of this important product in Europe in acquired antithrombin deficiency,"
stated Geoffrey F. Cox, Ph.D., GTC's Chairman of the Board and Chief Executive Officer. "In addition we remain committed to completing our submission for BLA approval in the US. The US remains a major long term opportunity for ATryn(R) in a range of hereditary and acquired deficiency indications. It is also important for our transgenic technology platform that the CHMP has not identified to us any
significant outstanding issues related to the manufacturing and production control systems for ATryn(R)."
Antithrombin is a protein in human plasma that has anticoagulant and anti-inflammatory properties. Patients that have a hereditary deficiency indication are prone to developing blood clots during high-risk procedures, such as surgery and childbirth. Providing supplemental antithrombin during these procedures may prevent the occurrence of DVT's and other thromboembolic events. Antithrombin supplementation therapy may also be useful in the treatment of patients who acquire temporary antithrombin deficiency during certain
clinical situations such as burns, coronary artery bypass surgery, disseminated intravascular coagulation, sepsis, and bone marrow transplant.