This is a promising time for all who live with familial hypercholesterolemia, but it is perhaps especially important for those in our community who live with the most severe form of this life-threatening genetic condition - Homozygous FH (HoFH).
The past 5 years have ushered in a new era of incredible hope for the management of HoFH. The Food and Drug Administration approved two treatments for Homozygous FH: lomitapide (Juxtapid) in 2012 and mipomersen (Kynamro) in 2013. In 2015, the FDA approved two drugs in a promising new class of treatments known as PCSK9 inhibitors: evolocumab (Repatha) and alirocumab (Praluent). Both were specifically approved for Heterozygous FH, with evolocumab also approved for Homozygous FH. Rosuvastatin (Crestor) received FDA supplemental approval in 2016 for a new indication for treatment of pediatric patients 7 to 17 years of age with homozygous familial hypercholesterolemia (HoFH).
"Individuals who step forward to participate in research are vital to the scientific process and the search for more therapies."
Katherine Wilemon, Founder and CEO, FH Foundation
There is more on the horizon for FH, including several new drugs in development for Homozygous FH, as well as a novel gene therapy, that are actively recruiting for clinical trials. Because HoFH is rare, it is especially difficult to find enough HoFH patients to enroll in clinical trials - these studies are looking for participants.