Here’s What the FDA Has to Say About Isis Pharmaceuticals

Recent trial data released by Isis Pharmaceuticals (NASDAQ:ISIS) showed that the company’s treatment for a rare genetic disease caused abnormal growths. A report released Tuesday by the U.S. Food and Drug Administration announced that the regulator would review the cancer risk for the drug, Kynamro. Following the FDA’s report, shares for the pharmaceutical manufacturer dropped 14 percent to $11.19.

According to Bloomberg, this is the drug manufacturer’s greatest fall in more than 20 months. Previously, Shares in the California-based company had risen 68 percent over the last 12 months through Monday.

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During a conference call, held in late August to review the drug’s trial data, Isis Chairman and CEO, Dr. Stanley Crooke, said the company was “well along in preparations for the FDA Advisory Committee panel in October.” However, the FDA’s report revealed that in clinical trials, growths known as neoplasms developed in 3.1 percent of patients treated with Kynamro and 0.9 percent of patients who took the placebo.

Bloomberg reported that the FDA said the trial data was “limited by a small sample size and a short treatment time.”

“This imbalance in neoplasms will need to be assessed further in on-going and future studies and post-marketing (if approved),” said the FDA in the report.

The Kynamro injection, which Isis partnered with Sanofi (NYSE:SNY) to develop, lowers LDL cholesterol, said the FDA staff. The drug targets a genetic disease, homozygous familial hypercholesterolemia, that causes abnormalities in the liver cells tasked with clearing LDL from the blood. Those that suffer from the disease can have cholesterol levels two to four times higher than normal, and do not respond to standard medications, like Pfizer’s (NYSE:PFE) Lipitor. The disease can cause heart attacks and can potentially be fatal.

Similar risks were noted by the FDA in a treatment developed by Aegerion Pharmaceuticals (NASDAQ:AEGR) to fight the disease. However, the agency said the company has a plan to manage the risk, and therefore supports its approval for use in adults. Approval will not be decided until December.

In a press release issued at the end of March, Isis said the drug’s third-phase clinical trial data showed that “robust LDL-C reductions are seen in patients treated for 2 years and more.” Isis’s third-phase study included 141 patients, all of whom completed one of the earlier trials, and lasted six months. An additional 40 patients have agreed to continue the trial for a further two years. According to the company’s data, the drug lowered LDL cholesterol by 25 percent in four of the late-stage trials, compared to 3.3 percent of those on the placebo.

The FDA will make a final decision on Kynamro in January of next year, depending on whether the treatment’s effect on the liver can be mitigated.

If the drug is approved, Kynamro could generate $183 million in the U.S. by 2016, according to analysts surveyed by Bloomberg. The agreement Sanofi signed with Isis in 2008, the France-based company will receive between 30 and 50 percent of the drug’s profits.

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