NEUROCHEM RECEIVES APPROVABLE LETTER FOR KIACTA

Neurochem has received an approvable letter from the FDA for Kiacta (eprodisate) for the treatment of Amyloid A amyloidosis. In the letter, the FDA requested additional efficacy information, as well as a safety update. The FDA stated that this efficacy information would probably need to be addressed by one or more additional clinical trials. As an alternative, the FDA also stated that significant findings obtained from a complete follow-up of patients in the existing study could be persuasive.

A progressive and fatal condition, Amyloid A amyloidosis occurs in a proportion of patients with chronic inflammatory disorders, chronic infections and inherited diseases. The kidney is the organ most frequently affected, and progression to dialysis and end-stage renal disease is the most common cause of death. There is no FDA-approved therapy to treat the disease, and half of all patients affected die within five years of diagnosis.

The FDA in April granted Kiacta priority review status, a classification reserved for product candidates that would offer a significant improvement in the treatment, diagnosis or prevention of a disease or that address an unmet medical need. The drug also received orphan drug designation and fast-track status.