Genzyme and Aegerion’s Treatments for HoFH, Explained

Having pills for breakfast

In late December 2012 and January 2013, the FDA approved Genzyme’s Kynamro and Aegerion Pharmaceutical’s Juxtapid, two products that treat homozygous familial hypercholesterolemia (HoFH). These drugs were both granted orphan status, as HoFH affects about 3,000 people in the United States. Although it is unusual […]

CHI Reports – How Biotech Companies are Shaping the Future in Rare Diseases


Biotech companies are navigating new trends in addressing rare diseases, conditions effecting 200,000 or fewer Americans. Jeremy Springhorn (Alexion Pharmaceuticals), Andrew Curtis (Pfizer), Marc Beer, (Aegerion Pharmaceuticals) and Susan Kahn (National Tay-Sachs & Allied Disease Association), shared their perspectives on the future of new treatments […]