New Therapy Approved by the FDA, New Hope for FH Patients

Eades FamilyToday families living with familial hypercholesterolemia (FH) have new hope that they can win the fight against aggressive premature heart disease.  Individuals with FH need all the help they can get to manage this risk factor encoded in their genes. Fortunately, more help is on the way for those who need it.

The Food and Drug Administration (FDA) announced today that the PCSK9 inhibitor alirocumab, known as brand name Praluent (Sanofi/Regeneron), was approved “for use in addition to diet and maximally tolerated statin therapy in adult patients with heterozygous familial hypercholesterolemia (HeFH) or patients with clinical atherosclerotic cardiovascular disease such as heart attacks or strokes, who require additional lowering of LDL cholesterol,” according to the FDA’s announcement.

” The approval of this PCSK9 inhibitor is a landmark event in the treatment of FH.  The many FH patients who simply cannot sufficiently reduce their LDL-C levels with existing therapies now have another highly-effective option for lowering LDL.  Thousands of FH patients around the world have participated in clinical trials to prove that PCSK9 inhibitors are efficacious and safe.  And the Family Heart Foundation played a key role at the crucial FDA Advisory Committee meetings in making clear the importance of these medicines for patients with FH. ” 

Dan Rader, MD
Perelman School of Medicine, University of Pennsylvania
Chief Scientific Advisor, Family Heart Foundation
But, we can’t stop here.  Existing and new treatments are only helpful if people know they have FH and are treated effectively. FH affects as many as 1 in 250 people, but fewer than 10% of them are diagnosed.  We can change that and save lives.

the Family Heart Foundation’s research efforts are contributing to the understanding of FH and the significant unmet needs of FH patients. Our awareness and outreach efforts are reaching patients and healthcare providers with valuable information about this severe genetic predisposition to early cardiovascular disease.  the Family Heart Foundation needs your continued support to help us find the 90% of individuals who are unaware they have this inherited condition so they also have a fighting chance to prevent heart disease.

One Response to “New Therapy Approved by the FDA, New Hope for FH Patients”

  1. Ellen Wolfson

    I have no options left. Have been approved for Praluent Patient Assistance program and will be getting Praluent soon!!! So thrilled to have a chance to get treatment. I am a patient of Emil deGoma, MD, who is in Dan Rader’s group.


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