KYNAMRO (mipomersen sodium) injection
Our flagship product, KYNAMRO, is being marketed in the United States by Genzyme for patients with
HoFH. These are patients who are at high cardiovascular risk and who are not able to reduce their LDL-C
sufficiently with currently available lipid-lowering therapies. KYNAMROwas approved by the FDA in January
2013 as an adjunct to lipid-lowering therapy and diet to reduce LDL-C, apolipoprotein-B, or apo-B, total
cholesterol and non-high-density lipoprotein-cholesterol, or non-HDL-cholesterol, in patients with HoFH.
KYNAMRO is available in the United States under a risk evaluation and mitigation strategy, or REMS, with a
BoxedWarning citing the risk of hepatic toxicity.
Genzyme is executing a comprehensive plan to address a global commercial market that consists of patients
who are in desperate need of new treatment options for HoFH. Genzyme has obtained marketing approval for
KYNAMRO in the United States, South Korea, Argentina, Mexico and Peru for use in patients with HoFH and
is continuing to pursue approval in multiple additional markets. In order to reach patients with HoFH in the
United States, Genzyme is concentrating marketing and sales efforts on lipid specialists, cardiologists, and
physicians who treat these types of patients. In the United States, Genzyme has established the KYNAMRO
Cornerstone, a program offering services related to HoFH and KYNAMRO, including dedicated case
management, reimbursement support, financial assistance for those who qualify, in-person injection training, and
disease and product education for healthcare providers, patients, families, and caregivers. Genzyme also
continues to raise awareness of HoFH. These activities include supporting continued medical educational
programs to inform physicians about HoFH and partnering with key advocacy groups, such as the Familial
Hypercholesterolemia Foundation, the National LipidAssociation, American College of Cardiology, the
International Symposium onAtherosclerosis and theAmerican Heart Association.
KYNAMRO is a novel, first-in-class, apo-B synthesis inhibitor for the reduction of LDL-C. It is an
antisense drug we discovered and licensed to Genzyme in 2008. KYNAMRO acts by decreasing the production
of apo-B. Apo-B provides the structural core for atherogenic lipids, including LDL-C, which carry cholesterol
through the bloodstream. KYNAMRO reduces LDL-C and other key atherogenic lipids linked to cardiovascular
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