FDA Approves Orphan Drug to Treat Rare Inherited Cholesterol Disease

The U.S. Food and Drug Administration (FDA) yesterday approved a new cholesterol drug, mipomersen sodium, marketed under the name Kynamro. This powerful new drug is to be used in conjuction with other lipid-lowering medications and diet to treat patients with a rare type of high cholesterol called homozygous familial hypercholesterolemia (HoFH).

The addition of Kynamro to a patients drug therapy helps to reduce low-density lipoprotein-cholesterol (LDL-C), apolipoprotein B, total cholesterol, and non-high density lipoprotein-cholesterol (non HDL-C). It is currently available by injection only.

This rare form of hypercholesterolemia, called HoFH, is a genetic condition, inherited from one’s parents, and affects about one out of every one million people in the United States. This condition occurs when the bodies ability to remove bad cholesterol, LDL-C, from the blood is restricted, thereby causing abnormally high levels of circulating LDL-C. Persons not receiving treatment for HoFH are at greater risk for heart attacks and death before the age of 30.

Mipomersen Sodium was developed to treat a disorder affecting fewer than 200,000 people, giving it orphan drug status and FDA approval as such.

“Kynamro, an injection given once a week, works with other lipid-lowering medications and diet to impair the creation of the lipid particles that ultimately give rise to LDL-C,” said Eric Colman, M.D., deputy director of the Division of Metabolism and Endocrinology Products at the FDA’s Center for Drug Evaluation and Research.

Kynamro is manufactured by the Genzyme Corp in Cambridge, Massachusetts, a subsidary of Sanofi-Aventis, in conjuction with Isis Pharmaceuticals in Carlsbad, California.

The FDA approval was good news for the management at Genzyme.

“Today’s FDA approval of KYNAMRO is great news for patients with HoFH who are in need of additional treatment options for this rare, and often under-diagnosed disease,” said Genzyme President and CEO, David Meeker, M.D. ‘As the leader in treatments for rare diseases, we are pleased to bring our expertise to HoFH patients living with this serious condition to better help them manage their disease.”

This is great news for people suffering from homozygous familial hypercholesterolemia.

“People living with Homozygous FH may not appear to be sick, but they live with the burden of this rare disease every day,” said Katherine Wilemon, President and Founder the FH Foundation “The approval of KYNAMRO gives the HoFH community hope that HoFH can be effectively managed.

In December 2012, the Food and Drug Administration approved lomitapide to reduce LDL-C, total cholesterol, apolipoprotein B, and non HDL-C in patients with homozygous familial hypercholesterolemia.

The news was well received by Sanofi’s partner, Isis Pharmaceuticals.

“KYNAMRO is the first systemic antisense drug to reach the market and is the culmination of two decades of work to create a new, more efficient drug technology platform. As evidenced by our robust pipeline, our antisense drug discovery technology is applicable to many different diseases, including the treatment of a chronic and rare disease, like HoFH,” said Stanley T. Crooke, M.D., Ph.D., Chairman of the Board and CEO of Isis. “We look forward to continuing to work with Genzyme toward a successful commercial launch of KYNAMRO and global expansion into other markets.”

The safety and effectiveness of Kynamro were evaluated in a clinical trial of 51 patients with HoFH. On average, levels of LDL-C fell by about 25 percent during the first 26 weeks in those receiving the drug. Kynamro carries a Boxed Warning on the serious risk of liver toxicity because it is associated with liver enzyme abnormalities and accumulation of fat in the liver, which could lead to progressive liver disease with chronic use.

The FDA approved Kynamro with a Risk Evaluation and Mitigation Strategy (REMS) with elements to assure safe use, which requires a prescription authorization form for each new prescription.

Adverse side effects and reactions in the clinical trial included injection site reactions, flu-like symptoms, nausea, headache and elevations in liver enzymes.

Jim Donahue

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