Neurochem, Inc. (headquartered in Québec, Canada) announced that it has received an Approvable Letter from the US FDA for eprodisate (1,3-propanedisulfonate, Kiacta™, formerly Fibrillex™), an oral investigational product candidate for the treatment of Amyloid A (AA) amyloidosis. According to the company, the FDA requested additional safety and efficacy data, as well as further manufacturing and pharmacokinetic information. The FDA stated that the efficacy information would probably need to be addressed by one or more additional clinical trials, but left open the possibility that that significant findings obtained from a complete follow-up of patients in the ongoing eprodisate phase II/III open-label extension study could be sufficient. The FDA also stated that a "QT" clinical study should be submitted as part of a phase IV (post approval) commitment. The company is currently gathering additional information for the FDA and hopes to receive final marketing approval without conducting a new efficacy clinical trial.

The phase II/III clinical trial for Kiacta was a 2-year, international, multicenter, randomized, double-blind, placebo-controlled, and parallel-designed trial to evaluate the efficacy and safety of eprodisate in patients suffering from AA amyloidosis confirmed by biopsy and renal involvement. The study, conducted at 27 sites located across North America, Europe, North Africa, and Israel, enrolled 183 patients and was completed in 2005. All patients who completed the trial were invited to join the ongoing open-label phase II/III extension study.
 
AA amyloidosis is a progressive and fatal condition that occurs in a proportion of patients with chronic inflammatory disorders including rheumatoid arthritis and Crohn's disease, chronic infections and inherited diseases such as Familial Mediterranean Fever. The kidney is the organ most frequently affected by AA amyloidosis, and progression to dialysis and end-stage renal disease is the most common cause of death in this disease.  Patients suffering from AA amyloidosis have a poor prognosis with 5- to 15-year survival rates of 50% and 25%, respectively. 

Neurochem has granted Centocor, Inc, a wholly owned subsidiary of Johnson & Johnson, exclusive distribution rights for Kiacta worldwide, with the exception of Canada, Switzerland, Japan, China, South Korea, and Taiwan.

—A. Techman

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