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SAN DIEGO, March 6, 2019 /PRNewswire/ -- The FCS Foundation, the nation's leading advocacy and support organization for people affected by familial chylomicronemia syndrome (FCS), today announced completion of a follow-up discussion via teleconference on February 25 with members of the U.S. Food and Drug Administration (FDA) to call for renewed efforts to find a path forward for approval of the investigational therapy WAYLIVRA™ (volanesorsen). The meeting was held just prior to announcement on March 1 that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending conditional marketing authorization for WAYLIVRA as an adjunct to diet in adult patients living with FCS.
"In August 2018, the FDA declined to approve WAYLIVRA as the first-ever treatment for FCS. We recognize that many people do not fully understand the devastating physical and emotional impact FCS has on patients and families and we appreciate any opportunity to educate others including regulators about our experiences to help them make informed decisions," said Lindsey Sutton, co-founder of The FCS Foundation and an FCS patient. "The decision by the CHMP in Europe to authorize approval of a treatment for FCS gives us hope that the FDA will recognize that patients in the U.S. deserve the same opportunity for a treatment that can lower our triglyceride levels and reduce our risk of potentially fatal acute pancreatitis."
Following discussions with the FDA, the co-founders of The FCS Foundation, Lindsey Sutton and Melissa Goetz, also met with representatives of several members of Congress in Washington, D.C. to discuss the impact of FCS and the need for a treatment. They shared perspectives from their own experience and from discussions with patients in the U.S. and around the world.
"We want to help more members of Congress understand the unique challenges that people with rare diseases face, and that the FDA should learn about the experiences of patients and consider their opinions in reviews of potentially life-saving treatments," said Melissa Goetz, co-founder of The FCS Foundation. "In many cases, drugs to treat rare diseases are approved with requirements in monitoring and safety that are very workable for patients and caregivers. The members of the FCS community want the same flexibility in reviews of any investigational drugs to treat FCS, because without a treatment we remain at risk of severe and potentially fatal attacks of pancreatitis."
About The Familial Chylomicronemia Syndrome FoundationFounded in 2016, The FCS Foundation's mission is to establish a global support network to promote advocacy and education for patients and caregivers living with FCS. The FCS Foundation is the only foundation dedicated to patients suffering from familial chylomicronemia syndrome. For more information, visit: www.livingwithfcs.org.
Media Contacts:Melissa Goetz, Co-President, FCS Foundation(518) [email protected]
Lindsey Sutton, Co-President, FCS Foundation(619) [email protected]
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"In August 2018, the FDA declined to approve WAYLIVRA as the first-ever treatment for FCS. We recognize that many people do not fully understand the devastating physical and emotional impact FCS has on patients and families and we appreciate any opportunity to educate others including regulators about our experiences to help them make informed decisions," said Lindsey Sutton, co-founder of The FCS Foundation and an FCS patient. "The decision by the CHMP in Europe to authorize approval of a treatment for FCS gives us hope that the FDA will recognize that patients in the U.S. deserve the same opportunity for a treatment that can lower our triglyceride levels and reduce our risk of potentially fatal acute pancreatitis."
Following discussions with the FDA, the co-founders of The FCS Foundation, Lindsey Sutton and Melissa Goetz, also met with representatives of several members of Congress in Washington, D.C. to discuss the impact of FCS and the need for a treatment. They shared perspectives from their own experience and from discussions with patients in the U.S. and around the world.
"We want to help more members of Congress understand the unique challenges that people with rare diseases face, and that the FDA should learn about the experiences of patients and consider their opinions in reviews of potentially life-saving treatments," said Melissa Goetz, co-founder of The FCS Foundation. "In many cases, drugs to treat rare diseases are approved with requirements in monitoring and safety that are very workable for patients and caregivers. The members of the FCS community want the same flexibility in reviews of any investigational drugs to treat FCS, because without a treatment we remain at risk of severe and potentially fatal attacks of pancreatitis."
About The Familial Chylomicronemia Syndrome FoundationFounded in 2016, The FCS Foundation's mission is to establish a global support network to promote advocacy and education for patients and caregivers living with FCS. The FCS Foundation is the only foundation dedicated to patients suffering from familial chylomicronemia syndrome. For more information, visit: www.livingwithfcs.org.
Media Contacts:Melissa Goetz, Co-President, FCS Foundation(518) [email protected]
Lindsey Sutton, Co-President, FCS Foundation(619) [email protected]
View original content:http://www.prnewswire.com/news-releases/familial-chylomicronemia-syndrome-foundation-completes-follow-up-discussion-with-fda-in-effort-to-find-path-forward-for-investigational-treatment-300807734.html
SOURCE The FCS Foundation
