Neurochem (International) Ltd (Neurochem), a wholly-owned subsidiary of Neurochem Inc., announces that the company has received a second approvable letter from the United States Food and Drug Administration (FDA) for eprodisate (KIACTA) for the treatment of Amyloid A (AA) amyloidosis.

In its action letter, the FDA indicates that the phase II/III clinical trial provided some evidence of the effectiveness of eprodisate (KIACTA) in the treatment of the renal manifestations of amyloidosis; however, the FDA also indicated that an additional efficacy trial with a target p-value of 0.05 will be necessary before the FDA could approve the investigational product candidate. Further, the approvable letter states that additional submissions, filed by Neurochem as part of its complete response to this approvable letter, may address issues raised in this letter. The FDA has indicated that such additional submissions could persuade the agency to eliminate the requirement for an additional trial.

The FDA also asked for additional information, including further pharmacokinetic studies, and again acknowledged that a QT clinical study should be submitted as part of a phase IV (post-approval) commitment.

The company expects to file a complete response to this approvable letter in the near future.

"We are disappointed that the FDA has not yet approved KIACTA. This is a rare disease with no specific treatment available for the patients." commented Dr. Francesco Bellini, Neurochem's chairman, president and CEO. "We will continue to work closely with the agency with the hope of securing final approval of this product candidate," he concluded.

Eprodisate (KIACTA) was investigated in an international, randomized, double-blind, placebo-controlled, and parallel-designed Phase II/III clinical trial in which 183 AA amyloidosis patients were enrolled at 27 sites around the world (Eprodisate for AA Amyloidosis Trial, EFAAT). Patients who completed the clinical trial were eligible for enrolment in an ongoing open-label extension study, some of whom have now been receiving eprodisate (KIACTA) for more than five years. Eprodisate (KIACTA) has received orphan drug status in the United States, the European Union and in Switzerland.

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/end-stage renal disease is the most common clinical manifestation in this disease. Currently, there is no approved therapy to treat AA amyloidosis and about half of all patients diagnosed with the disease die within five years of diagnosis.