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 has announced today that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended granting a market authorisation to CSL’s alpha-1 antitrypsin augmentation therapy product Respreeza®.Receiving a positive CHMP opinion (See EMA Website) marks an important milestone in the process of Respreeza® obtaining a market authorisation (= drug license) in all EU Member States, including the UK. The CHMP positive opinion will be transmitted to the European Commission (EC) to start the EC decision-making process on granting a centralised European drug license for Respreeza®, which we hope to be concluded later this year.
Lutz Bonacker, Senior Vice President & General Manager Commercial Operations, Europe, CSL Behring said: “CSL Behring continues to make strides towards fulfilling our promise to improve the lives of our patients. This positive opinion from CHMP brings us closer to providing Respreeza® as a new treatment option to the AATD community in Europe.”However, once a drug license is granted by the EC, access to Respreeza® to UK patients will be subject to reimbursement of the drug by the NHS. As use of Respreeza® will be indicated in adults with documented severe alpha-1 antitrypsin deficiency and evidence of progressive lung disease as evaluated by a healthcare professional experienced in the treatment of Alpha-1, it is important that patients gain access to the care provided by a physician with sufficient experience in the field of Alpha-1, e.g. at the recently established West Midlands Alpha-1 Services