FDA Denies the Only Treatment for FCS patients!

FDA Denies the Only Treatment for FCS patients!

14,285 have signed. Let’s get to 15,000!
Petition to
U.S. Food & Drug and

Why this petition matters

Started by Yeun Kim

The U.S. Food & Drug Administration recently denied WAYLIVRA, potentially the FIRST and ONLY treatment option for people living with FCS (Familial Chylomicronemia Syndrome).

There are approximately 3,000 to 5,000 people diagnosed with FCS around the world with no treatment options available.  FCS is an ultra-rare, devastating and debilitating genetic 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 any and all parts of daily living.  Without a treatment option, they will continue to suffer from pancreatitis, more hospitalizations, abdominal pain and fatigue, brain fog, cognitive impairment, social and emotional isolation and the daily fear of having another attack of pancreatitis.

Mark continues to be hospitalized with pancreatitis dozens of times per year (every 4-6 weeks) since 2010, lives with severe daily pain and fatigue, lost his job in 2013 due to frequent hospitalizations and now living on disability.  He's underwent plasmapheresis up to 3 times a week for 2 years for temporary relief, which has lost its effectiveness.  He owes tens of thousands of dollars in medical bills.  His Pancreatologist has told him that WAYLIVRA is his only hope to avoid removing his pancreas.

Lindsey has had over 30 attacks of pancreatitis, lives with daily pain and severe fatigue.

Alvin's first pancreatitis attack at the age of 50 left him in a coma for 42 days and a hospital bill of $2.2 million dollars.

Mary has been hospitalized 15 times for pancreatitis, on life support for 8 days and given a 50/50 chance of survival.  She has lost hope of having children.  Her marriage was destroyed, lost her career and has had to file for bankruptcy.

There are so many others suffering from this disease and WAYLIVRA has all the potential to minimize and even stop the symptoms and hospitalizations!  It is the one and only light in our pitch black tunnel of this disease.  We never believed it would be possible to have a shot at a semi-normal life again, until we discovered this treatment.

The FDA Advisory Committee voted in favor of approving WAYLIVRA earlier this year.  Given the tremendous physical, emotional and social burdens of FCS, the community's participation in the clinical studies, and our overwhelming support for approval, we are extremely disappointed that the Division's decision has left patients in the U.S. without a treatment. 

As a wife and caregiver to an FCS patient who is debilitating right before my eyes, more and more everyday, I can say that we cannot afford to wait any longer!  I cannot remember the last time my husband went a day without pain and symptoms like vomiting and/or diahrrea.  We have missed weddings, graduations, birthdays and holidays, nor can we plan vacations or social events because of this horrible rare disease! 

WAYLIVRA needs to be approved as it would be THE ONLY treatment option for FCS!


14,285 have signed. Let’s get to 15,000!