FDA issues CRL to Cumberland's Acetadote sNDA for non-acetaminophen acute liver failure

Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX) announced today that it has received a Complete Response Letter from the U.S. Food and Drug Administration (FDA) regarding its supplemental new drug application (sNDA) for Acetadote® (acetylcysteine) Injection to treat patients with non-acetaminophen induced acute liver failure.

The FDA issues a Complete Response Letter (CRL) when it has completed its review of an application as a formal communication to identify additional work required or items that must be addressed prior to approval of a new product or indication. In its CRL to Cumberland regarding this new indication for Acetadote, the agency confirmed that patients with Coma Grade I/II observed a numerically higher rate of transplant-free survival, but also noted that there was not sufficient evidence of efficacy for the proposed indication of increasing survival in all patients with acute liver failure.

"We appreciate the FDA's efforts to review this application, and we share the agency's commitment to providing pharmaceutical products that meet the highest standards of safety and efficacy for patient groups who will truly benefit," said A.J. Kazimi, Chief Executive Officer of Cumberland Pharmaceuticals. "We continue to believe that the data and literature supporting Acetadote as a treatment for patients suffering from non-acetaminophen acute liver failure are extremely relevant to a critically ill patient population with few treatment alternatives. We plan to request a meeting with the FDA to resolve the outstanding issues related to this application and look forward to working closely with the agency to gain clarity on the pathway to approval for this important indication."

The sNDA for the new indication, filed in March 2010, was based in part on data from a clinical trial led by investigators at the University of Texas Southwestern Medical Center indicating that acute liver failure patients treated with Acetadote have an improved chance of survival without a transplant. The study also demonstrated that these patients can survive a significant number of days longer without transplant, providing patients requiring transplant increased time for a donor organ to become available. Patients were stratified according to Coma Grade, with Coma Grade I representing the earliest stages of liver failure and Coma Grade IV representing late-stage conditions. Analyses presented in Cumberland's sNDA indicate that transplant-free survival was significantly higher at three weeks, one year and two years for patients in Coma Grades I and II receiving Acetadote than for those receiving placebo. The results from the study, which is the largest clinical trial studying acute liver failure to date, were published in the medical journal Gastroenterology.

Cumberland's request to expand labeling for Acetadote to include the new indication followed a discussion of this data with the FDA. The Company requested and was granted a priority review for the application.

This sNDA is part of Cumberland's commitment to support ongoing development of Acetadote and its other products. Acetadote was initially launched by Cumberland in 2004 as the first injectable drug to treat acetaminophen overdose approved in the United States. Since then, Acetadote has become a standard of care in treating acetaminophen poisoning, which is the leading cause of toxic drug ingestions reported to U.S. poison control centers. In 2006, the FDA approved Acetadote for use in pediatric patients. Cumberland also received FDA approval for updated safety labeling for Acetadote in 2008 based on information from a post-marketing safety study reporting a lower incidence of side effects compared to previously reported data.

In October 2010, Cumberland submitted an application to the FDA for approval of a new, second generation formulation of Acetadote, which is designed to replace the currently marketed product. The Company is currently supporting FDA review of that application and expects to receive agency response regarding the new formulation by early January 2011.