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."
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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.
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