NEUROCHEM RESPONDS TO FDA APPROVABLE LETTER FOR KIACTA

Neurochem has submitted a complete response to the FDA's August approvable letter for Kiacta. The company is seeking marketing approval of Kiacta (eprodisate) for the treatment of Amyloid A amyloidosis. There is currently no treatment for the deadly disease, which often destroys kidney function, according to the company.

The complete response includes the data on safety and efficacy from a follow-up of all 183 patients who were enrolled in the Phase II/III clinical trial. Following the suggestion of the FDA in its approvable letter, Neurochem successfully retrieved the most recent health information for all 183 study subjects, including patients currently enrolled in the open-label extension study and all patients who discontinued their participation in the study. The median time of follow-up was approximately 36 months.

The statistical methodology, as discussed with the FDA, includes the analysis to compare the time required to progress to dialysis/end-stage renal disease (ESRD) and to progress to a composite endpoint of dialysis/ESRD or death (all causes) between the two groups originally randomized to either Kiacta or placebo. The results of the follow-up analysis include: Fewer patients progressed to dialysis/ESRD in the Kiacta group (18) than in the placebo group (32); 56 patients progressed to death from all causes, 25 from the group originally randomized Kiacta and 31 from the group originally randomized to placebo; and the updated safety information shows that Kiacta continues to be safe and well-tolerated over long-term exposure.

Kiacta has received orphan drug designation in both the U.S. Europe.