The FH Foundation started 2021 off with an impactful Listening Session with the Food and Drug Administration (FDA) Center for Drug Evaluation and Research Division of Diabetes, Lipid Disorders, and Obesity (DDLO) on Homozygous Familial Hypercholesterolemia (HoFH) on January 4. Kate Robinson and Scott Radabaugh, FH Advocates for Awareness, Allison Jamison, FH Foundation Board Member, each shared how HoFH has affected their lives. HoFH is a rarer more severe form of familial hypercholesterolemia which can cause heart disease as early as childhood if not diagnosed and treated.
"The burden of living with HoFH as a child goes well beyond the medical concerns, although those will be with us each and every day. The burden of being a child with HoFH spills into everyday life. As a parent of two HoFH children, I simply want more in life for them as they deserve a childhood free of fear and anxiety."
– Kate Robinson
A FDA Listening Session is a small, non-public meeting that offers an opportunity for the FDA to better understand the experience of living with a disease or condition, beyond what they can learn from clinical trial data. The FH Foundation set up this meeting in order to underscore the importance of new treatment options for individuals living with HoFH.
Kate, Scott and Allison powerfully shared their perspectives on how children, teenagers, and adults live with HoFH. They underscored the burden of early cardiovascular disease and the many ways that an HoFH diagnosis impacts the whole family. In addition, they shared why there is a need for additional treatment options to achieve LDL-cholesterol levels low enough to reduce heart disease risk in addition to offering critical peace of mind.
Dr. Mary McGowan, Chief Medical Officer of the FH Foundation, presented data from the FH Foundation's national CASCADE FH Registry, a long term observational research study. The CASCADE FH Registry data specifically highlights the high incidence of cardiovascular disease and treatment challenges for individuals living with HoFH.
"I don’t want to miss out on life, and my children’s lives. I hope we continue to develop new treatments for HoFH that allow patients like me to see past 28 years old, avoid damage to their hearts, and lead a long and happy life."
– Allison Jamison
The FH Foundation would like to thank Kate, Scott and Allison for proudly representing the HoFH Community with the FDA! Many thanks also to the researchers and individuals who participate in the CASCADE FH Registry for their impactful contributions to the understanding and treatment of FH and HoFH.
"This 9-year journey as the dad of a child with HoFH has been long, very difficult and produced very few wins. However, we are in a new time of hope and very excited about what the future holds. My greatest wish is that this generation of people with HoFH come to know the condition as very manageable."
– Scott Radabaugh