•  
  •  
 

Pharmacy and Wellness Review

Abstract

Homozygous familial hypercholesterolemia (HoFH) is a rare disease that involves mutations in the genes coding for low density lipoprotein (LDL) receptors, preventing the uptake of LDL cholesterol from the serum and resulting in extremely high cholesterol levels.1 Between December 2012, and January 2013, two orphan drugs were approved by the U.S. Food and Drug Administration (FDA) for the treatment of HoFH. Mipomersen (Kynamro®) is a subcutaneous injection that functions as an antisense oligonucleotide inhibitor and ultimately prevents the translation of mRNA coding for apolipoprotein B (apoB)-100 which binds to LDL and very low density lipoprotein (vLDL) cholesterol.7 Lomitapide (Juxtapid®) is an oral drug that inhibits microsomal triglyceride protein (MTP), an enteric and hepatic protein that promotes the lipid transfer to apoB and allows a complex to form. Through the inhibition of MTP, vLDL cholesterol and chylomicrons are not formed. Each of these drugs, when combined with a low-fat diet and additional lipid-lowering therapy, which may include statins, resins and LDL-apheresis, can produce a clinically significant reduction in serum LDL cholesterol.7,14 Before the approval of these two drugs, patients faced a greatly shortened lifespan, uncertain and nonspecific treatment options and serious complications secondary to HoFH.1,3-5

Share

COinS
 
 

To view the content in your browser, please download Adobe Reader or, alternately,
you may Download the file to your hard drive.

NOTE: The latest versions of Adobe Reader do not support viewing PDF files within Firefox on Mac OS and if you are using a modern (Intel) Mac, there is no official plugin for viewing PDF files within the browser window.