Isis Pharmaceuticals recently announced that the Endocrinologic and Metabolic Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) found the company’s drug, Kynamro (the trade name for mipomersen sodium) has been demonstrated to exhibit “sufficient” efficacy and safety for the treatment of Homozygous Familial Hypercholesterolemia (HoFH). HoFH is an aggressive, genetic cardiovascular disorder characterized by high levels of “bad” cholesterol, or low-density lipoprotein.
The committee voted 9 to 6 to recommend Kynamro, and Isis will include the recommendation in its application for FDA approval. Though the vote will be considered when the drug is reviewed, the FDA is not bound by the recommendation.
“We are very encouraged by the support for Kynamro at today’s Advisory Committee meeting, which marks a significant and positive step in our efforts to bring this important new therapy to patients and families affected by this often unrecognized genetic disorder,” said David Meeker, President and CEO of Genzyme, Isis’ marketing partner for the new drug. “There is still a great need for the HoFH patients, who have exhausted conventional medications and still have LDL cholesterol levels 2 to 4 times above normal. Genzyme looks forward to working with the FDA as it completes its review of the Kynamro application.”
In making its decision, the committee reviewed data from a phase 3 study of Kynamro in HoFH patients, three other “supportive ” phase 3 studies of high-risk hypercholesterolemia patients, and and ongoing long term extension study