The approval of mipomersen for the treatment of homozygous FH is a groundbreaking event for the patients who have extremely elevated LDL-cholesterol and inadequate therapeutic options. This drug has a first-in-class mechanism of action and has been effective for these patients in clinical trials so far. Though it will require careful monitoring, we hope that the addition of mipomersen to our therapeutic arsenal will help us lower the LDL in these patients to “healthier” levels, translating into less heart attacks and potentially longer life for these very high-risk patients.– Joshua W. Knowles, MD, PhD, Chief Medical Officer of the FH Foundation
On January 29th, 2013 …. announced that the FDA approved a new HoFH drug, KYNAMRO (mepomersen). Within only two months, two drugs have become available for those affected by Homozygous Familial Hypercholesterolemia. We will be updating you with more news about this exciting development as it breaks. Watch this space!