The FDA sent a letter to Neurochem in August that requested additional efficacy information, as well as a safety update of Kiacta before it would issue approval. The FDA stated that this efficacy information would probably need to be addressed by one or more additional clinical trials.
Amyloid A (AA) amyloidosis is a deadly disease which often destroys kidney function and there is still no specific treatment for AA amyloidosis.
The response by Neurochem included the data on safety and efficacy from a follow-up of all 183 patients who were enrolled in the phase II/III clinical trial. Neurochem retrieved the most recent health information for all study subjects which involved around three years of follow up research.
Neurochem reported that the follow-up analysis on the composite endpoint was in favor of the Kiacta group as fewer patients progressed to dialysis, end stage kidney disease or death versus the placebo group.
“The results from the follow-up analysis continue to provide evidence of the benefit of Kiacta for the treatment of AA amyloidosis and Kiacta's favorable effect on slowing the progression of renal impairment,” said Dr Francesco Bellini, CEO of Neurochem.