Neurochem has announced that it has initiated, under the Continuous Marketing Application Pilot 1 program, the submission of a "rolling" New Drug Application (NDA) to the US Food and Drug Administration (FDA) seeking marketing approval for its investigational product candidate Fibrillexâ„¢ (1,3-propanedisulfonate) for the treatment of Amyloid A (AA) Amyloidosis. Neurochem plans to complete the submission of the NDA at or around year-end. The FDA agreed to file and review the NDA last June.

Approximately 40,000 patients in North America and Europe with chronic or inherited inflammatory diseases such as rheumatoid arthritis, ankylosing spondylitis, Familial Mediterranean Fever, and Crohn's disease have been diagnosed with AA Amyloidosis, a progressive condition causing widespread organ dysfunction and end-stage renal failure for which no specific treatment exists. Fibrillex has received Orphan Drug Status designation and Fast Track Product designation in the US and Orphan Medicinal Product designation in Europe. Neurochem will provide additional follow-up clinical data collected from the ongoing open-label extension study as part of its NDA submission. The results of Neurochem's single Phase II/III trial were mixed, with missed primary endpoint and unaffected relative risk of death (all-cause mortality), but demonstrated clinical effect and potential delay in renal disease progression.

—Nicholas K. Zittell