LAVAL, QC, Aug. 11 /PRNewswire-FirstCall/ - Neurochem Inc. announces that it has received an approvable letter from the U.S. Food and Drug Administration (FDA) for eprodisate (Kiacta(TM) - formerly Fibrillex(TM)) for the treatment of Amyloid A (AA) amyloidosis.
In its action letter, the FDA requested additional efficacy information, as well as a safety update. The FDA stated that this efficacy information would probably need to be addressed by one or more additional clinical trials. As an alternative, the FDA also stated that significant findings obtained from a complete follow-up of patients in the existing study could be persuasive. The FDA asked for further manufacturing and pharmacokinetic information, and acknowledged that a QT clinical study should be submitted as part of a Phase 4 (post approval) commitment.
"We are pleased with the FDA's response and the constructive relationship we have with them," said Dr. Francesco Bellini, Chairman, President and CEO of Neurochem. "We are working closely with the agency to secure as soon as possible the final approval without conducting a new efficacy clinical trial. We are already starting to collect additional information as suggested by the FDA," he concluded.
A progressive and fatal condition, AA amyloidosis occurs in a proportion of patients with chronic inflammatory disorders, chronic infections and inherited diseases such as Familial Mediterranean Fever. The kidney is the organ most frequently affected and progression to dialysis and end stage renal disease is the most common cause of death in this disease. Currently, there is no FDA-approved therapy to treat AA amyloidosis and half of all patients diagnosed with the disease die within five years of diagnosis.
Kiacta(TM) underwent a landmark international, randomized, double-blind, placebo-controlled, and parallel-designed clinical trial in which 183 patients were enrolled in 27 sites around the world.
In April 2006, the FDA granted Priority Review to Kiacta(TM), a classification reserved for product candidates that would offer a significant improvement in the treatment, diagnosis or prevention of a disease or that address an unmet medical need. Over the course of its development, Kiacta(TM) received Orphan Drug Designation and Fast Track status by the FDA and was accepted by the Agency for the Continuous Marketing Application (CMA) Pilot 1 and 2 programs.
Neurochem will host a conference call at 08H30 Eastern Time, Monday, August 14, 2006.
To participate in the conference call, please dial the following numbers approximately 10 minutes prior to the start of the call: 416-644-3430 or 1-800-814-4861. A replay of the conference call will be available for one week (until August 21, 2006), commencing one hour after the end of the call. The instant replay numbers are 416-640-1917 or 1-877-289-8525. The access code for the replay is 21200044. Please mention that you are calling for the Neurochem conference replay.
Neurochem is focused on the development and commercialization of innovative therapeutics to address critical unmet medical needs. Eprodisate (Kiacta(TM)) is currently being developed for the treatment of AA amyloidosis and has received in August 2006 from the FDA an approvable letter. Tramiprosate (Alzhemed(TM)), for the treatment of Alzheimer's disease, is currently in Phase III clinical trials in both North America and Europe and tramiprosate (Cerebril(TM)), for the prevention of Hemorrhagic Stroke caused by Cerebral Amyloid Angiopathy, has completed a Phase IIa clinical trial.
To Contact Neurochem
For additional information on Neurochem and its drug development programs, please call the North American toll-free number 1-877-680-4500 or visit our Web Site at www.neurochem.com.
This news release contains forward-looking statements regarding Kiacta(TM), as well as regarding continuing and further development efforts. These statements are based on the current analysis and expectations of management. Drug development necessarily involves numerous risks and uncertainties, which could cause actual results to differ materially from this current analysis and these expectations. Analysis regarding the results of clinical trials may not provide definitive results regarding safety, tolerability or therapeutic benefits. Even if all the endpoints sought in the clinical trials were met (which is not certain), there is no certainty that regulators would ultimately approve Kiacta(TM) for sale to the public. Risks and uncertainties may include: failure to demonstrate the safety, tolerability and efficacy of our product, the expense and uncertainty of obtaining regulatory approval, including from the FDA, and the possibility of having to conduct additional clinical trials. Further, even if regulatory approval is obtained, therapeutic products are generally subject to: stringent on-going governmental regulation, challenges in gaining market acceptance, and competition. Neurochem does not undertake any obligation to publicly update its forward-looking statements, whether as a result of new information, future events, or otherwise. Please see the Annual Information Form for further risk factors that might affect the Company and its business.