Neurochem, Inc. announced that the US Food and Drug Administration (FDA) has designated the company's most advanced investigational drug candidate, Fibrillex, as a Fast Track Product (FTP) for the treatment of secondary Amyloid A (AA) amyloidosis, a serious and life-threatening condition which develops as a consequence of chronic infections or chronic inflammatory diseases, such as Rheumatoid Arthritis (RA). Secondary AA amyloidosis (better known as AA amyloidosis) has a poor prognosis, with a five-year survival rate of approximately 50 per cent. Left untreated, patients gradually progress to end-stage renal failure, which is the cause of death in at least 35per cent of the cases.

As a result of the FTP designation, Neurochem will submit a request for a rolling New Drug Application (NDA) for Fibrillex, enabling the FDA to commence review of portions of the application before the end of the on-going two-year Phase II/III trial.

"AA amyloidosis is a severe disease. No specific therapy currently exists," stated Denis Garceau, vice president of Drug Development at Neurochem. "We are pleased that Fibrillex received FTP designation. The designation reflects the FDA's acknowledgement of the life-threatening nature of AA amyloidosis, which often results in end-stage renal disease. The FDA's decision also highlights the company's commitment to studying the effects of Fibrillex on endpoints of clear clinical benefit, including preservation of kidney function and preventing patients from undergoing dialysis."

The FTP designation, originally enacted under the FDA's Modernization Act of 1997, expedites the development and review of a new drug that is intended for the prevention or treatment of a serious or life-threatening condition, and demonstrates the potential of a drug candidate to address unmet medical needs for such a condition.

This designation favorably adds to Fibrillex' existing orphan drug status designation in the United States and Europe, which designation normally provides a product seven and ten years of market exclusivity, respectively, upon market entry.

AA amyloidosis is a progressive and fatal condition that occurs in a proportion of patients with chronic inflammatory diseases, including RA, ankylosing spondylitis, juvenile rheumatoid arthritis, and Crohn's disease. The disease also occurs in patients suffering from many other conditions ranging from chronic infections to inherited inflammatory diseases such as Familial Mediterranean Fever. The most common clinical presentation of AA amyloidosis is kidney malfunction. Involvement of the gastrointestinal system is also frequent and is usually manifested as chronic diarrhea, gastrointestinal bleeding, abdominal pain and malabsorption. Enlargement of the liver and the spleen may also occur in some patients.