CSL Behring announced today that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended granting marketing authorization for Respreeza®, a highly purified alpha-1 protein derived from human plasma, indicated to treat patients with alpha-1 antitrypsin deficiency (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. Respreeza® replaces the protein that these patients are missing and raises the alpha-1 antitrypsin levels in their blood, which can help to protect the lungs from damage due to inflammation.
"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," said Lutz Bonacker, Senior Vice President & General Manager Commercial Operations, Europe, CSL Behring.
The CHMP positive opinion will be transmitted to the European Commission (EC) to start the EC decision-making process. The EC may then grant a marketing authorization for Respreeza® as a maintenance treatment to slow the progression of emphysema in adults with documented severe alpha-1 proteinase inhibitor deficiency. CSL Behring's RAPID (randomized, placebo-controlled trial of augmentation therapy in alpha-1 proteinase inhibitor deficiency) study results were considered as part of this submission. According to findings of the study, patients with AATD treated with alpha-1 proteinase inhibitor therapy exhibited a lower annual rate of lung density decline compared to placebo, when measured using chest computed tomography, at full inspiration. This demonstrated that Respreeza® significantly slows the progression of emphysema in these critically ill patients.
About CSL Behring's Pulmonary Products
Respreeza® will be indicated for maintenance treatment, and to slow the progression of emphysema in adults with documented severe alpha-1 proteinase inhibitor deficiency (e.g. genotypes PiZZ, PiZ(null), Pi(null,null), PiSZ). Patients are to be under optimal pharmacologic and non-pharmacologic treatment and show evidence of progressive lung disease (e.g. lower forced expiratory volume per second (FEV1) predicted, impaired walking capacity or increased number of exacerbations) as evaluated by a healthcare professional experienced in the treatment of alpha-1 proteinase inhibitor deficiency. Respreeza® is contraindicated in patients with hypersensitivity to the active substance or to any of the excipients and IgA deficient patients with known antibodies against IgA, due to the risk of severe hypersensitivity and anaphylactic reactions.
Zemaira® is a highly-purified form of alpha-1 proteinase inhibitor (human) currently approved in Brazil, New Zealand, and the US, where it is indicated for chronic augmentation and maintenance therapy in adults with AATD and clinical evidence of emphysema. Zemaira® is contraindicated in patients with a history of severe systemic reaction to the product or to A1-PI protein, including anaphylaxis. Due to the risk of severe hypersensitivity, Zemaira® is also contraindicated in immunoglobulin A-deficient patients with antibodies against IgA.
About Alpha-1 Antitrypsin Deficiency
Alpha-1 Antitrypsin Deficiency is an inherited condition that can result in severe lung disease in adults and liver disease at any age, as well as other less known manifestations such as paniculitis, a skin disease. AATD is the most commonly known genetic risk factor for emphysema and is commonly referred to as genetic COPD. Low levels or absence of the protective protein alpha-1 antitrypsin, which is produced by the liver, characterize AATD.
About CSL Behring
CSL Behring is a leader in the plasma protein therapeutics industry. Committed to saving lives and improving the quality of life for people with rare and serious diseases, the company manufactures and markets a range of plasma-derived and recombinant therapies worldwide.
CSL Behring therapies are used around the world to treat coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies, hereditary angioedema and inherited respiratory disease, and neurological disorders in certain markets. The company's products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic disease of the newborn.
CSL Behring operates one of the world's largest plasma collection networks, CSL Plasma. CSL Behring is a global biopharmaceutical company and a member of the CSL Group of companies. The parent company, CSL Limited (ASX:CSL), is headquartered in Melbourne, Australia. For more information, visit www.cslbehring.com.
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Contact:
Jennifer Seiler, External Communications Manager
CSL Behring
O: +1 610-878-4802
jennifer.seiler@cslbehring.com