Neurochem (International) Ltd (ECUBLENS, Switzerland), a wholly-owned subsidiary of Neurochem Inc, announced that it intends to initiate a second phase III clinical for eprodisate (Kiacta™) to obtain market approval for the treatment of amyloid A (AA). Besides having received 2 approvable letters from the US FDA, Neurochem is advancing the drug because amyloidosis is a life-threatening disease for which there is no specific treatment; other reasons for advancing the drug include data obtained from the first phase II/III clinical trial, which yielded promising efficacy results on the study endpoints, on the benign safety profile of Kiacta in patients diagnosed with AA, and on its discussions with the regulatory agencies and related scientific advisory committees.

As part of the drug approval process, both the regulatory agencies in the US and EU recommended an additional confirmatory efficacy phase III clinical trial, which will have a P <.05 target rather than the P <.01, which had been required for an approval based on a single efficacy study. Based on these recommendations, Neurochem will discuss with the US FDA and the European Medicines Agency (EMEA) an agreement on the approval of Kiacta to treat AA. As part of that strategic decision, Neurochem announced that it is withdrawing its current marketing applications for Kiacta in the US, the EU, and Switzerland submitted in February, September, and November 2006, respectively.

The FDA and EMEA have both acknowledged that the first phase II/III clinical trial provided some evidence of the effectiveness of Kiacta in the treatment of the renal manifestations of amyloidosis.

The drug was investigated in an international, randomized, double-blind, placebo-controlled, and parallel-designed phase II/III clinical trial in which 183 AA patients were enrolled at 27 sites worldwide. Patients who completed the clinical trial were eligible for enrollment in an ongoing open-label extension study, some of whom have now been receiving Kiacta for >6 years. The completion of the extension study is scheduled for April 2008.

Kiacta has received orphan drug status in the US and EU, which normally provide 7 and 10 years of market exclusivity, respectively, upon regulatory approval.

A progressive and fatal condition, amyloidosis occurs in a proportion of patients with chronic inflammatory disorders, chronic infections, and inherited diseases such as familial Mediterranean fever. The kidney is most frequently affected and progression to dialysis/endstage renal disease is the most common clinical manifestation in the disease. Currently, there is no approved therapy to treat amyloidosis and about half of all AA patients diagnosed with the disease die within 5 years of diagnosis.