KiactaTreatment for Amyloid A Amyloidosis
FDA Agrees to File and Review NDA for Fibrillex
ECUBLENS, Switzerland, June 30, 2005 - Neurochem (International) Limited (Neurochem), a wholly-owned subsidiary of Neurochem Inc. , today announced that the U.S. Food and Drug Administration (FDA) has agreed to file and review a New Drug Application (NDA) for Fibrillex that would include the efficacy and safety data from Neurochem's single Phase II/III trial. Fibrillex is Neurochem's investigational product candidate for the treatment of Amyloid A (AA) Amyloidosis. This announcement comes as a result of a recent meeting with the Cardio-Renal Division of the FDA to discuss the results of Neurochem's Phase II/III clinical trial. The Agency encouraged Neurochem to look at and provide additional follow-up data collected from the open-label extension study as part of its NDA submission.
"AA Amyloidosis is a serious medical condition that frequently leads to organ dysfunction, subsequent death and for which no specific treatment exists," said Denis Garceau, PhD, Neurochem's Senior Vice President, Drug Development. "The filing of our first NDA is a significant event for Neurochem. We are therefore very pleased that the FDA has agreed to file and review a NDA for Fibrillex, recognizing its Orphan Drug Status. We look forward to working with the regulatory agencies and hope to make this potential treatment available to over 40,000 patients in North America and Europe presently diagnosed with the disease."
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. It has received Orphan Drug Status designation in the United States and Orphan Medicinal Product designation in Europe.
Fibrillex also has been accorded "Fast Track Product" designation by the FDA and has been selected by the Cardio-Renal Drug Product Division of the FDA to be part of the Continuous Marketing Applications Pilot 2 program aimed at further accelerating the development of this product candidate. Under this Pilot 2 program, each FDA division is permitted to select only one product candidate.
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 2 to 10 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.
Neurochem is focused on the development and commercialization of innovative therapeutics for neurological disorders. The Company's pipeline of proprietary, disease-modifying oral products addresses critical unmet medical needs. 1,3-propanedisulfonate (1,3PDS; Fibrillex) is designated as an orphan drug and a Fast Track Product candidate and is also part of an FDA Continuous Marketing Applications Pilot 2 program. The Phase II/III clinical trial of Fibrillex for the treatment of AA Amyloidosis was recently concluded and results have been released between April and June 2005. 3-amino-1-propanesulfonic acid (3APS; Alzhemed), for the treatment of Alzheimer's Disease, is in a Phase III clinical trial and 3APS (Cerebril), for the prevention of Hemorrhagic Stroke caused by Cerebral Amyloid Angiopathy, has completed a Phase IIa clinical trial.
Posted: June 2005
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- Neurochem Initiates Submission of NDA for Fibrillex - August 22, 2005