FDA gives new drug application approval to Isis
Drug developer Isis Pharmaceuticals Inc. and partner Genzyme announced Tuesday that the U.S. Food and Drug Administration has approved their new drug application for Kynamro, clearing the way for Isis' first drug to reach market.
Kynamro is an injectable drug designed to treat patients who are genetically predisposed to have high levels of LDL cholesterol, known as the "bad" cholesterol. The drug has been approved for use as an adjunct to lipid-lowering medications and diet for patients with homozygous familial hypercholesterolemia.
HoFH is a rare inherited condition that makes the body unable to remove LDL cholesterol from the blood, causing abnormally high levels of circulating LDL cholesterol. In the United States, HoFH occurs in about one in one million individuals, according to the company.
"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," Genzyme president and CEO, David Meeker, said in a statement.
Isis, based in Carlsbad, Calif., has a deal with Genzyme, a unit of French drugmaker Sanofi SA, to market the drug if it is approved. The FDA approval triggers a $25 million milestone payment to Isis from Genzyme.
Approval of the drug was not certain in the past. There were questions about its links to cancer. But the companies said in October that an advisory panel to the FDA decided there was sufficient data on the drug's safety and effectiveness to allow sales of Kynamro.
The companies said Tuesday that the drug may cause serious side effects, including liver problems.
Shares of Isis shot up $1.03, or 7.70 percent, to $14.41 in after-hours trading on the news.