KiactaTreatment for Amyloid A Amyloidosis
Neurochem Initiates Submission of NDA for Fibrillex
ECUBLENS, Switzerland, August 22, 2005 - Neurochem (International) Limited (Neurochem), a wholly-owned subsidiary of Neurochem Inc. , announced today that it has initiated, under the Continuous Marketing Application (CMA) Pilot 1 program, the submission of a "rolling" New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking marketing approval for its investigational product candidate, Fibrillex, for the treatment of Amyloid A (AA) Amyloidosis. Neurochem plans to complete the submission of the NDA at or around year-end. As announced last June, the FDA has agreed to file and review this NDA. AA Amyloidosis is a serious medical condition for which no specific treatment exists and frequently leads to organ dysfunction and subsequent death.
"We are pleased to report that we have initiated the submission of our NDA for review ahead of schedule," said Denis Garceau, PhD, Neurochem's Senior Vice President, Drug Development. "Fibrillex appears to delay the progression of renal disease and to be well tolerated. To date, the results from the Phase II/III clinical trial have been well received by the physicians. We are committed to working closely with the FDA in an effort to make this potential treatment available to patients. There are over 40,000 patients presently diagnosed with the disease in North America and Europe."
As previously announced in December 2004, Neurochem shares an exclusive collaboration and distribution agreement for Fibrillex with Centocor, Inc., of Malvern, PA (USA).
Fibrillex is an oral investigational product candidate for the treatment of AA Amyloidosis through the prevention of amyloid fibril formation. In 1999 Fibrillex received U.S. Orphan Drug Designation, in 2001 the Orphan Medicinal Product (OMP) designation in Europe, in 2004 the Fast Track Designation in the U.S. and was accepted into the Pilot 2 and Pilot 1 programs by the FDA's Division of Cardio-Renal Drug Products in 2004 and 2005, respectively.
As per the Orphan Drug Status designation in the U.S. and the Orphan Medicinal Product designation in Europe, Fibrillex should normally benefit from market exclusivity for seven and ten years respectively in each of these jurisdictions.
About AA Amyloidosis
AA Amyloidosis is a progressive and fatal condition that occurs in a proportion of patients with chronic inflammatory diseases, including rheumatoid arthritis, 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 renal dysfunction. 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. End- stage renal failure is the main cause of death in 40-60% of cases. The median survival time from diagnosis varies from two to ten years depending on the stage of the disease at the time of diagnosis.
No specific treatment is currently available for this orphan disease. The goal of existing therapies is limited to the control of the underlying chronic inflammatory disease.
Posted: August 2005
- Neurochem Moves Eprodisate (Kiacta) Drug Development Program Forward for Amyloid A Amyloidosis - March 13, 2008
- Neurochem Announces Eprodisate (Kiacta) Receives Acknowledgement of Complete Response and is Granted Class 2 Review - December 7, 2007
- Neurochem Receives Approvable Letter for Eprodisate (Kiacta) for Treatment of AA Amyloidosis - July 18, 2007
- Neurochem's Eprodisate (Kiacta) PDUFA Date Extended Three Months by FDA - April 11, 2007
- Neurochem submits a complete response to FDA approvable letter for Kiacta - October 16, 2006
- Neurochem Receives Approvable Letter for Eprodisate (Kiacta) for Treatment of AA Amyloidosis - August 11, 2006
- Neurochem Announces Fibrillex NDA Filed and Granted Priority Review - April 18, 2006
- Neurochem Submits NDA for Fibrillex - February 13, 2006
- FDA Agrees to File and Review NDA for Fibrillex - June 30, 2005