FDA PCSK9 Hearing

News of the Week for Many, News of a Lifetime for Those With FH

The FDA Advisory Committee’s decision June 9th and 10th to recommend the approval of two novel LDL-lowering treatments (known as PCSK9 inhibitors) was the news of the week for many. But for people living with familial hypercholesterolemia, it was the news of a lifetime. 


FH is surprisingly common and vastly underdiagnosed. There are roughly as many people in the U.S. who have HIV as have FH. The same is true of Type 1 Diabetes. FH is more common than BRCA breast cancer, another autosomal dominant genetic disorder. But if you walked down the street before last week and asked 1,000 people if they had heard of FH or familial hypercholesterolemia, 999 would have said “no.”


FH is unheard of in the public conversation, and yet it is a major public health issue.

And heartbreakingly, FH is a family condition. As a matter of fact, it so easily inherited that it is just a flip of a coin – a 50/50 chance that each child of an affected mother or father will inherit this deadly condition. Even though families have usually never heard the words familial hypercholesterolemia, they know only too well that heart disease steals their loved ones away in the prime of their lives. People with FH often have heart attacks in their 20’s, 30’s, and 40’s. Every male with FH has a 50% chance of having a heart attack before 50 years old, unless he is diagnosed and treated early. Women with FH have an 80% chance of having a heart attack during their lives.


FH is dangerously misconstrued as just “high cholesterol.” Cholesterol is not a pathology, but heart disease is. In fact, heart disease is the biggest single killer in the modern world, killing 5 times as many people as all cancers combined each year.


There is a dire need for additional therapies for the FH population. Statins are the mainstay of treatment and have been proven to significantly reduce the risk of early cardiac events, including in those who are born with FH. Nevertheless, statins and other lipid lowering therapies such as ezetimibe and bile acid sequestrants are often not enough for many with FH, and some cannot tolerate them. the Family Heart Foundation was at the FDA Advisory Committee hearings last week to speak to the unmet needs of the approximately 1.5 million in the U.S. with FH. The data we presented from our National FH Registry, CASCADE FH, demonstrates that many people with FH do not reach ideal LDL levels, even on maximum available therapy.


the Family Heart Foundation mobilized the FH community. Many women and men stood up to speak. They spoke to the fear of dying while their children are young, the fear that any day could be the day they have their first or fourth heart attack. They spoke to the fear that their children affected by FH will never have the chance to live a full life, especially if they are impacted by the most severe form, homozygous FH. Families reach out the Family Heart Foundation from around the world for guidance and support. Every day, we work diligently alongside FH experts to bring credible information to the medical community and the public in innovative and impactful ways.  Our vision, our reason for being, is to raise awareness of FH through education, advocacy, and research. We do this work with a singular focus and a deep sense of urgency. We understand that people are dying and families are suffering needlessly. What is unique about the Family Heart Foundation is that we are an organization created by patients for patients in collaboration with leading FH experts. 


Last week was historic in terms of how many people were talking about FH. The conversation was not just about us, it included us, and we believe that made a difference.

Katherine Wilemon
Family Heart Foundation Founder, President, and CEO

3 Responses to “FDA PCSK9 Hearing”

  1. Trabalho de conscientização global torna a Hipercolesterolemia Familiar mais conhecida

    […] por falar em tratamento, o segundo inibidor PCSK9 da Amgen, Repatha (evolocumab), foi aprovado em 27 de agosto último pela U.S. Food and Drug Administration (FDA). Agora é […]

  2. Judith VonDerGathen

    I need to be on a PCSK9 inhibitor but was told the cost is $700 for 2 shots. I do not have insurance right now. Is there any way I can get access to this medication?

    • Colleen McCready

      The two companies that make the inhibitors have special patient copay assistance programs that may be able to help you.


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