SAN DIEGO, Sept. 6, 2018 /PRNewswire/ -- The FCS Foundation, the only patient group in the United States focused on supporting patients and families living with familial chylomicronemia syndrome (FCS), is urging the U.S. Food and Drug Administration (FDA) to approve WAYLIVRA (volanesorsen) and to provide commercial access to patients who are desperately in need.
The FDA recently issued a Complete Response Letter (CRL) for the New Drug Application for WAYLIVRA, meaning the agency will not approve the drug at this time. This decision follows a positive vote in favor of approval by the FDA Advisory Panel in May 2018, during which more than two dozen members of the FCS community testified in support of approval. Many speakers participated in the clinical trials for WAYLIVRA and spoke of its positive impact on their life, despite some side effects. WAYLIVRA, the only drug of its kind, was shown to lower triglycerides an average of 77% in patients with FCS. This FDA decision leaves FCS patients with no clinical treatment for the disease.
FCS is an ultra-rare, devastating disease that leaves those affected at risk of unpredictable and potentially fatal acute pancreatitis, chronic complications due to permanent organ damage, and other symptoms that can have a severe impact on the ability to work and take part in daily activities. Currently the only "treatment" available is an extremely low-fat diet (below 10-20 grams a day – there are about five grams of fat in a pat of butter). But the risk of symptoms and burden of FCS persist even when patients adhere to the diet. Standard lipid-lowering therapies target a mechanism that is impaired in people with FCS and so also do not work effectively. Despite the FDA having a department that is committed to understanding the impact and burden of rare diseases, it appears they have not taken the patient experience or testimony into consideration in their decision to not approve WAYLIVRA.
Members of the FCS community are outraged and demand action from the FDA. Melissa Goetz and Lindsey Sutton, co-founders and presidents of the FCS Foundation, said, "We are heartbroken for the FCS community and for ourselves and our loved ones, who will have no relief from long hospital stays, daily debilitating pain, isolation, and burden of disease. If the FDA truly listened to our patient stories and experiences with FCS and treatment with WAYLIVRA, this drug would not have been rejected. We demand the FDA reconsider this decision and approve WAYLIVRA for use."
Matt Alsante, executive director of the National Pancreas Foundation, said, "Familial chylomicronemia syndrome is a devastating disease that causes acute pancreatitis among other severe complications. This drug meets a huge unmet need as there are no approved treatments for FCS patients. The FDA should let patients, families, and their physicians make the decision on the risks and benefits of this important therapy."
For more information on FCS, how this decision affects our community, or for ways to help visit www.livingwithfcs.org, www.facebook.com/fightfcs, www.twitter.com/livingwithfcs
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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The FDA recently issued a Complete Response Letter (CRL) for the New Drug Application for WAYLIVRA, meaning the agency will not approve the drug at this time. This decision follows a positive vote in favor of approval by the FDA Advisory Panel in May 2018, during which more than two dozen members of the FCS community testified in support of approval. Many speakers participated in the clinical trials for WAYLIVRA and spoke of its positive impact on their life, despite some side effects. WAYLIVRA, the only drug of its kind, was shown to lower triglycerides an average of 77% in patients with FCS. This FDA decision leaves FCS patients with no clinical treatment for the disease.
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FCS is an ultra-rare, devastating disease that leaves those affected at risk of unpredictable and potentially fatal acute pancreatitis, chronic complications due to permanent organ damage, and other symptoms that can have a severe impact on the ability to work and take part in daily activities. Currently the only "treatment" available is an extremely low-fat diet (below 10-20 grams a day – there are about five grams of fat in a pat of butter). But the risk of symptoms and burden of FCS persist even when patients adhere to the diet. Standard lipid-lowering therapies target a mechanism that is impaired in people with FCS and so also do not work effectively. Despite the FDA having a department that is committed to understanding the impact and burden of rare diseases, it appears they have not taken the patient experience or testimony into consideration in their decision to not approve WAYLIVRA.
Members of the FCS community are outraged and demand action from the FDA. Melissa Goetz and Lindsey Sutton, co-founders and presidents of the FCS Foundation, said, "We are heartbroken for the FCS community and for ourselves and our loved ones, who will have no relief from long hospital stays, daily debilitating pain, isolation, and burden of disease. If the FDA truly listened to our patient stories and experiences with FCS and treatment with WAYLIVRA, this drug would not have been rejected. We demand the FDA reconsider this decision and approve WAYLIVRA for use."
Matt Alsante, executive director of the National Pancreas Foundation, said, "Familial chylomicronemia syndrome is a devastating disease that causes acute pancreatitis among other severe complications. This drug meets a huge unmet need as there are no approved treatments for FCS patients. The FDA should let patients, families, and their physicians make the decision on the risks and benefits of this important therapy."
For more information on FCS, how this decision affects our community, or for ways to help visit www.livingwithfcs.org, www.facebook.com/fightfcs, www.twitter.com/livingwithfcs
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/the-familial-chylomicronemia-syndrome-foundation-implores-fda-to-approve-much-needed-therapy-300707992.html
SOURCE The FCS Foundation
