Alpha-1 Antitrypsin Deficiency also known as Alpha-1, A1AD or AATD is an inherited, genetic condition that is passed on from generation to generation. As the name suggests it is a deficiency of alpha-1 antitrypsin (AAT) in the bloodstream. AAT is an enzyme produced in the liver to help protect the tissues of the body during infections. The low level of AAT in the blood occurs because the AAT is abnormal and cannot be released from the liver at the normal rate. This leads to a build up of abnormal AAT in the liver that can cause liver disease and a decrease of AAT in the blood that can lead to lung disease.
CSL Behring Receives Marketing Authorization for Respreeza® in Europe
Respreeza® is a maintenance treatment for severe Alpha-1 Antitrypsin Deficiency patients and has been shown to slow the progression of emphysema
CSL Behring announced today that the European Commission (EC) has granted marketing authorization in all European Union (EU) member states for Respreeza® to treat patients with Alpha-1 antitrypsin deficiency (AATD). Respreeza®, a highly purified Alpha-1 protein derived from human plasma, is the only Alpha-1 proteinase inhibitor that has been proven in a prospective double blind, placebo controlled trial (the RAPID study) to significantly reduce the loss of lung tissue, slowing the progression of emphysema due to AATD. AATD is a hereditary condition marked by a lack of the Alpha-1 antitrypsin protein, whose main function is to protect the lungs from inflammation.
“AATD is a potentially debilitating disease and many affected individuals suffer from serious lung disease," said Professor Helmut Teschler, MD, Director of the West German Lung Centre at the University of Essen. "With the approval of Respreeza®, healthcare professionals can now provide patients with severe AATD in Europe with a next generation Alpha1-proteinase inhibitor (A1-PI) that provided additional evidence that this augmentation therapy can slow the accelerated loss of lung tissue."
Frank Willersinn, M.D., Alpha-1 Global Steering Committee Chair and patient representative in Europe added, “We are so glad that Respreeza® has been approved by the European Medicines Agency. We commend CSL Behring for their long-lasting commitment to the Alpha-1 community, now bringing their established Alpha 1-antitrypsin product to Europe, allowing it to be a cornerstone for treatment of AATD patients in the near future.”
Source: CSL Behring 25/08/2015