Neurochem Inc. has received an approvable letter from the US Food and Drug Administration (FDA) for eprodisate (Kiacta - formerly Fibrillex) for the treatment of Amyloid A (AA) amyloidosis.
According to a company press release, 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 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, 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 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 programmes.