Global biotherapeutics leader CSL Behring will host an Expert Forum about Alpha-1 Antitrypsin Deficiency (AATD) today during the 26th International Congress of the European Respiratory Society (ERS) in London. Professor N. Gerard McElvaney and Doctor Ilaria Ferrarotti will chair the forum of leading authorities that will address topics including disease modification, irreversible lung tissue loss, and early treatment independent of lung function parameters.
Professor Joanna Chorostowska-Wynimko will discuss disease modification, including the paradigm that early treatment prevents irreversible lung tissue loss. She notes, "Whilst the RAPID trial and RAPID Extension trial indicate that A1-PI [Alpha-1 proteinase inhibitor] therapy is effective irrespective of the time of intervention, lung tissue lost during periods of no treatment cannot be regained."
Professor Kenneth Chapman will talk about changing the course of disease progression, in the context of emphysema, by therapy with A1-PI. He’ll discuss his work with Respreeza®, 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.
In the final session, Professor A. Rembert Koczulla will present a practical view on monitoring Alpha-1 patients and appropriate pharmacological and non-pharmacological treatment options. "With Respreeza® there is now an early treatment option independent of FEV1 values," notes Professor Koczulla.
Jürgen Frischmann is a real life example of an Alpha-1 patient benefitting from Respreeza® not being restricted by FEV1 values. He recently shared his experience in the Alpha-1Journal, an Alpha-1 patient organization magazine:
"I was diagnosed at a very late stage and was told there is no treatment available for me, as my FEV1 value was already less than 30%. When I found out that Respreeza® is indicated for the treatment of Alpha-1 Antitrypsin Deficiency independent of FEV1 values, I requested to receive Respreeza® and encourage all other Alpha-1 patients to do the same. Of course this applies not only to patients with an FEV1 of less than 35% but also to those with an FEV1 of more than 60%."
"Over a century ago, CSL Behring made a promise to protect the health of those stricken with a range of serious medical conditions," says Lutz Bonacker, Senior Vice President Europe, CSL Behring. He adds, "Today, we are delivering on that promise stronger than ever. Our commitment to patients living with AATD is exemplified by bringing innovations such as the very recently approved AATD treatment, Respreeza® to patients in Europe."
In EU, Respreeza® is indicated for maintenance treatment, and to slow the progression of emphysema in adults with documented severe A1-PI 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 A1-PI 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.
About the RAPID Study
The RAPID study (Randomized, Placebo-controlled Trial of Augmentation Therapy in Alpha-1 Proteinase Inhibitor Deficiency) enrolled 180 severely deficient alpha-1 patients in the U.S. and Europe who were treated for 2 years with Respreeza® or Placebo. The study, published in Lancet in June 2015, showed a significant reduction in the rate of lung tissue loss as measured by CT scan lung density. In those patients who continued into the extension study, where all subjects received Respreeza®, the benefit was maintained for another 2 years.
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 global biotherapeutics leader which is driven by its promise to save lives. Focused on serving patients’ needs by using the latest technologies, we develop and deliver innovative therapies that are used to treat coagulation disorders, primary immune deficiencies, hereditary angioedema, inherited respiratory disease, and neurological disorders. 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. The parent company, CSL Limited (ASX:CSL), headquartered in Melbourne, Australia, employs more than 16,000 people with operations in more than 30 countries. For more information visit www.cslbehring.com and follow us on Twitter.
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