FDA EMDAC Meeting – The Patient Voice

On June 9 and 10, members of the familial hypercholesterolemia (FH) Foundation and FH community had the opportunity to share their stories at the U.S. Food & Drug Administration (FDA) Endocrinologic & Metabolic Drugs Advisory Committee (EMDAC) meeting in Washington, D.C. This historic meeting focused on a new class of low density lipoprotein (LDL)-lowering therapies, PCSK9 inhibitors.  These therapies represent a major step forward in reducing the high risk of heart disease for the millions of people living with FH, including Jonathan Karas.  

Jonathan was first diagnosed with high cholesterol at age 13.  At this time he never heard the term FH.  His pediatrician suggested a change in diet and exercise, never informing him that his high cholesterol could be genetic.  Despite his exercise and healthy physique, his LDL numbers continued to climb, reaching the 300s while he was in his teens.  When Jonathan was 28, he suffered a heart attack

“My name is Jonathan Karas and I am a patient with FH and a heart attack survivor,” he said Tuesday as he began sharing his journey of diagnosis and treatment with FH to the EMDAC. “Having a heart attack at 28 is not normal, but my story seems fairly common for FH patients.”

The journey for Jonathan, like many people living with FH, remains challenging. They rely on meetings such as the FDA’s EMDAC to approve new options and treatments to stay alive. “About a year after my heart attack, I started taking a new medication that had been recently approved by the FDA,” Jonathan said after discussing the inability for other options to lower his cholesterol below 100. “Adding that new drug did the trick.”

“I was very lucky. It’s been 12 years since my heart attack, my LDL is consistently below 100, and I feel great. Having another treatment for my FH changed my life.” 

Unfortunately, not everyone is as lucky as Jonathan.  Family Heart Foundation-led discussion groups consist of concerns from people around the world who are unable to get their LDL numbers at suggested goal levels.  Unease of the safety and side effects of medications, inability to tolerate the options available, and struggles with insurance companies are common in the daily posts.

“I am here to tell my story and hopefully influence this Agency on the importance of options for the FH community. A common message from other patients on the Family Heart Foundation’s Facebook group is that having options equals hope.  Each FH patient is different, therefore we need to continue to promote research for safe, new drugs and therapies.”

Watch Jonathan’s full story here:

Today, the FDA voted in favor of Praluent 13-3 – primarily because of the clear need for the FH community.

Tomorrow, a second therapy in the class, Repatha, will be considered by the panel.  For more information on the meeting CLICK HERE

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