FRAMINGHAM, Mass.--(BUSINESS WIRE)--rEVO Biologics Inc., a subsidiary of LFB SA, today announced the submission of an Investigational New Drug (IND) Application with the US Food and Drug Administration (FDA) for ATryn® [antithrombin (Recombinant)] for the treatment of preeclampsia in pregnant women. The objective of the Phase 3 study (PRESERVE-1) is to assess whether ATryn® may safely prolong pregnancy in mothers with very early preterm preeclampsia and consequently decrease prematurity-associated morbidity and mortality of their babies. This study will focus on pregnant women who are at gestational age 24 to 28 weeks – an area of significant unmet need. Babies born at these gestational ages have a high risk of mortality or severe morbidity. During this period, prolongation of pregnancy, even by a few days, can lead to a significant improvement in the well-being of the newborns.
rEVO is seeking to expand the approved labeling for ATryn to include preeclampsia. ATryn is currently indicated for the prevention of peri-operative and peripartum thromboembolic events in hereditary antithrombin deficient patients. It is not indicated for the treatment of thromboembolic events.
Preeclampsia is a life-threatening condition that occurs in approximately six to eight percent of all pregnancies. In the United States (US), more than 200,000 pregnancies are affected by preeclampsia each year. Preeclampsia can occur any time after the twentieth week of pregnancy. It is particularly devastating when it occurs early during the pregnancy as it leads to the birth of severely premature babies. Very early preterm preeclampsia is a severe form of preeclampsia that arises before 28 weeks of gestation. It is estimated that between 0.15 and 0.2 percent of pregnancies in Western countries will be complicated by very early preterm preeclampsia, or 6,000 to 8,000 pregnancies in the US each year. If proven to be safe and effective, ATryn may be the first drug specifically indicated for the management of preterm preeclampsia in pregnant women.
"rEVO's focus is to bring life-changing, life-saving medicines to patients, and we believe that ATryn, the first treatment to be developed specifically for the management of preterm preeclampsia, exemplifies this mission," said Yann Echelard, PhD, President, rEVO Biologics. "We are committed to finding solutions for women with preeclampsia, and to working with the OB/GYN community, maternal fetal medicine specialists and patient advocacy groups to bring this important therapy to patients."
Delivery of the baby provides the only definitive cure for preeclampsia. When preeclampsia occurs at term (37 weeks or later), the decision to deliver the baby is straightforward. However, when preeclampsia occurs very early in pregnancy, obstetricians are faced with a serious dilemma. They must decide between delivering the baby and ending the preeclamptic process resulting in a premature infant, or continue the pregnancy to allow further fetal development with the risk of worsening preeclampsia in the mother. Early delivery, particularly before 28 weeks of gestational age, increases the risk of death and newborn complications, such as respiratory failure, intraventricular hemorrhage (bleeding inside the brain), blindness and necrotizing enterocolitis (death of the tissue lining the intestine) and is associated with severe lifelong disability.
"ATryn has the potential to revolutionize the way we manage preeclampsia in women who are early in their pregnancies by targeting the key underlying mechanisms of preeclampsia," said Michael J. Paidas, MD, Professor and Co-Director, Yale Women and Children's Center for Blood Disorders, and the lead investigator of PRESERVE-1. "This may slow the progression of the condition, allowing doctors to prolong the gestational age of the baby to improve mortality and morbidity."
"We have made great strides with expectant management – where mothers with preeclampsia are safely monitored with the goal of prolonging pregnancy and therefore reducing the degree of the baby's prematurity," said Baha Sibai, MD, Professor of Obstetrics, Gynecology and Reproductive Sciences at the University of Texas Health Science Center at Houston. "With preterm preeclampsia, every day in utero counts for the baby. The proposed Phase 3 trial with ATryn is an unprecedented step toward providing clinicians an important new therapy to reduce maternal morbidity and prevent maternal complications while safely extending a mother's pregnancy to allow further development of her baby."
Preeclampsia is a condition that only occurs in pregnancy and affects the placenta. Abnormal development of blood vessels from the uterus to the placenta may lead to decreased blood flow, nutrients and oxygen from mother to baby, placing the baby at risk for prematurity and abnormal fetal growth. The lining of the blood vessels (endothelium) also becomes damaged causing inflammation that extends from the placenta throughout the mother's blood vessels. As a result, high blood pressure, abnormal blood clotting in the placenta, stroke, seizures and liver and kidney problems can occur in the mother.
Preeclampsia and related conditions like HELLP syndrome (hemolysis, elevated liver enzymes and low platelet count) and eclampsia are characterized by a rapid rise in blood pressure for the mother that may lead to seizure, stroke, multiple organ failure or even death for mother or baby. Preeclampsia is the cause of serious illness or death for hundreds of thousands of women and babies every year. By conservative estimates, preeclampsia is responsible for 76,000 maternal and 500,000 infant deaths each year, worldwide.
The cause of preeclampsia is unknown, but the number of cases is growing – especially severe cases. In the US, identification and/or incidence of the disorder has increased by approximately 25 percent in the last two decades.
Important Safety Information
ATryn is contraindicated in patients with known hypersensitivity to goat and goat milk proteins. Allergic-type hypersensitivity reactions, including anaphylaxis, are possible. Patients must be closely monitored and carefully observed for any symptoms throughout the infusion period. Patients should be informed of the early signs of hypersensitivity reactions including hives, generalized urticaria, tightness of the chest, wheezing, hypotension and anaphylaxis. If these symptoms occur during administration, treatment must be discontinued immediately and emergency treatment should be administered.
The anticoagulant effect of drugs that use antithrombin to exert their anticoagulation may be altered when ATryn is added or withdrawn. To avoid excessive or insufficient anticoagulation, coagulation tests suitable for the anticoagulant used (e.g., aPTT and anti-Factor Xa activity) are to be performed regularly, at close intervals and in particular in the first hours following the start or withdrawal of ATryn. Additionally, patients should be monitored for the occurrence of bleeding or thrombosis in such situations.
The serious adverse reaction that has been reported in clinical studies is hemorrhage (intra-abdominal, hemarthrosis and post procedural). The most common adverse events reported in clinical trials at a frequency of ≥5% are hemorrhage and infusion site reaction.
For more information, please view full Prescribing Information for ATryn, visit www.atryn.com or call 1-800-610-3776.
To report suspected adverse reactions contact rEVO Biologics Inc.at 1-800-610-3776 or the FDA at 1-800-FDA-1088 or www.fda.gov/MedWatch.
About rEVO Biologics
rEVO Biologics Inc. (www.revobiologics.com) is a rare disease company focused on developing and delivering first- and next-generation therapeutics. Through its proprietary rPRO Technology platform, rEVO Biologics is developing safe and reliable therapeutic proteins to address critical medical needs. The company's lead product, ATryn, is the first and only plasma-free antithrombin concentrate. ATryn received FDA approval in 2009. The company has a number of products in its pipeline and in clinical trials in the areas of hematology, oncology, genetic disorders and autoimmune disease, and is actively seeking additional indications for its lead product.
About LFB group
LFB is a biopharmaceutical group that develops, manufactures and markets medicinal products for the treatment of serious and often rare diseases in several major therapeutic fields, including hemostasis, immunology and intensive care. The LFB Group is the leading manufacturer of plasma-derived medicinal products in France and sixth worldwide and is also among the leading European companies for the development of new-generation medicinal products or treatments based on biotechnologies. The LFB Group is pursuing a growth strategy that seeks to extend its activities at the international level and develop innovative therapies. To that end, the LFB Group currently markets its products in 20 countries around the world with a global turnover of €466 million in 2012.