Promising First Ever Treatment for Amyloid A Amyloidosis

From the European League Against Rheumatism

AMSTERDAM, the Netherlands—Progressive and untreatable, amyloid A (AA) amyloidosis might soon have its first ever specific treatment, an investigational drug called eprodisate (Fibrillex; Neurochem) that is under FDA priority review, investigators reported at the European League Against Rheumatism (EULAR)/ European Congress of Rheumatology annual meeting.

AA amyloidosis, a systemic inflammatory condition, most often affects the kidneys and usually progresses to end-stage renal disease and death. About 50% of patients with AA amyloidosis also have rheumatoid arthritis (RA).

IMWR

“We now have no effective treatment for AA amyloidosis. This is good news, since we see in this study a reduction in the progress of renal disease. However, we need to see if it will last. What we have is a good prospect for treating a condition that had none before,” Iain McInnes, chairman of the EULAR Scientific Committee and professor of experimental medicine at the University of Glasgow in Scotland, told .

Results of this trial show that the drug improved renal function and all-cause mortality and was well tolerated.

Investigators randomized 183 adults from 27 centers in 13 countries to either eprodisate or placebo for 2 years. Participants were also being treated for the underlying disease-49% for RA, 12% for ankylosing spondylitis, and 3% for juvenile chronic arthritis.

Treatment with the new drug reduced the risk of progression to kidney failure or death by 42% and the annual loss of kidney function by 30% compared with placebo.

“The significant results indicate that this is the first potential specific treatment for the condition,” investigator Bouke Hazenberg, MD, assistant professor of rheumatology at the University of Groningen in the Netherlands, told IMWR.

He added that the availability of the drug could lead to “the crucial earlier diagnosis and effective treatment of the condition.”

The study received the prestigious EULAR/Abbott Award, which is bestowed on the lead investigators of the 12 most highly rated abstracts from the meeting. The award was presented during the opening ceremony by Tore K. Kvien, MD, PhD, president of EULAR and chair of the Department of Rheumatology at the University of Oslo.

The FDA gave priority review status to eprodisate in April 2006. The review process is scheduled to be completed this month.

According to Neurochem, the developer of the drug, AA amyloidosis affects approximately 17,000 people in the United States. In 2004, Neurochem granted Centocor exclusive worldwide distribution rights for eprodisate (with the exception of Canada, Switzerland, Japan, China, South Korea, and Taiwan).