CALL TO ACTION: Rare Disease Patients Lose Hope for Only Treatment

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 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.”

famililal Chlyomicronemia SyndromeSIGNS and SYMPTOMS of Familial Chylomicronemia Syndrome

The detectable sign of FCS is extremely elevated triglycerides. However, patients often live with symptoms like:

  • abdominal pain (daily low-level to debilitating)
  • nausea
  • diarrhea
  • bloating
  • physical weakness
  • constipation
  • indigestion
  • acute pancreatitis
  • fatigue
  • impaired memory
  • difficulty concentrating and problem solving
  • “brain fog”
  • anxiety/fear/worry about health
  • eruptive xanthomas (pink bumps that appear on your body, arms and legs)
  • lipaemia retinalis (milky appearance of blood vessels in the eye)
  • hepatosplenomegaly
  • social isolation (due to diet restrictions)

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:

  • Acute pancreatitis can cause fluid and debris to collect in cyst-like pockets in your pancreas. These can rupture and cause complications like internal bleeding and infection
  • Acute pancreatitis can lead to infection which could require intensive treatment like surgery to correct
  • Kidney failure. Acute pancreatitis may cause kidney failure, which might require dialysis
  • Breathing problems. Acute pancreatitis can cause chemical changes in your body that affect your lung function and cause the level of oxygen in your blood to fall to dangerously low levels
  • Damage to insulin-producing cells in your pancreas from chronic pancreatitis can lead to diabetes
  • Pancreatitis can result in fewer of the enzymes needed to break down and process nutrients from food. This can lead to malnutrition, diarrhea and weight loss, even though your diet hasn’t changed
  • Pancreatic cancer. Long-standing inflammation in your pancreas caused by chronic pancreatitis is a risk factor for developing pancreatic cancer


The easiest way for you to do something to correct this is CLICK HERE > to go to the petition asking the FDA to make this treatment, the only potential treatment, available to patients with FCS.

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