Rare disease patients with Familial Chylomicronemia Syndrome lost hope for their only treatment when it was derailed by FDA, Take action: Urge FDA to Reverse its Decision.
Patients, providers, and advocates are banding together to try to reverse the FDA’s controversial 2018 decision to derail approval for Waylivra (a.k.a. Volanesorsen, made by Akcea Therapeutics) which would have treated Familial chylomicronemia syndrome (FCS).
Waylivra would have been the first and only treatment for FCS, an ultra-rare, devastating hereditary disease that causes unpredictable and potentially fatal acute pancreatitis, complications from organ damage, and a severe impact on daily living and quality of life. Currently, the only action patients can take involves a severely restricted low fat diet of 10-20 grams of fat a day, which, even when strictly adhered to, does not decrease triglycerides to normal levels or remove the threat of pancreatitis for most patients.
A change.org petition has already garnered more than 14,000 signatures, and advocates are calling their legislators and the FDA to take action on Waylivra, which is being reviewed in the EU and is already available to Familial Chylomicronemia Syndrome patients in the UK under the Medicines and Healthcare products Regulatory Agency’s Early Access to Medicines Scheme. The US has passed bills in recent years meant to make such innovations available to patients who are out of options. The 21st Century Cures Act and the Right To Try Act were passed expressly to make getting access to necessary treatments possible, yet Waylivra remains unapproved without adequate explanation by the FDA.
An article in the PRNewswire quotes Melissa Goetz and Lindsey Sutton, co-founders and presidents of the FCS Foundation, who 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.”
Additionally, 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.”
The detectable sign of FCS is extremely elevated triglycerides. However, patients often live with symptoms like:
Waylivra is meant to reduce triglyceride levels which in turn reduce the risk for inflammation of the pancreas (pancreatitis). Pancreatitis can cause serious complications, including:
The easiest way for you to do something to correct this is CLICK HERE > to go to the Change.org petition asking the FDA to make this treatment, the only potential treatment, available to patients with FCS.
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