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news.Neurochem has submitted to the FDA the final modules of its new drug application seeking marketing authorization for Fibrillex for the treatment of Amyloid A amyloidosis.
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The submission completes the “rolling” new drug application (NDA) that was initiated last August, under the continuous marketing application (CMA) pilot 1 program.
AA amyloidosis is a progressive and fatal condition that 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 by AA amyloidosis, and progression to dialysis and end stage renal disease is the most common cause of death in this disease.
New approaches to treatment for AA amyloidosis are urgently needed due to lack of specific therapies. Fibrillex has received orphan drug designation status in the US as it is estimated that there are about 20,000 patients in North America.
“We believe that the NDA package supports the clinically and statistically meaningful effect of this product candidate on the preservation of kidney function in this patient population and its benign safety profile,” said Dr Francesco Bellini, Chairman, President and CEO.