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October 13, 1999
Neurochem Inc. receives "Orphan Drug" status from FDA for Fibrillex™
Neurochem Inc., a Canadian-based biopharmaceutical company, has received "Orphan Drug
Designation" status from the U.S. Food and Drug Administration (FDA) for Fibrillex™,
a drug under development for the treatment of patients suffering from Secondary
Amyloidosis.
Secondary Amyloidosis is a disease caused by deposits of amyloid A (AA) protein mainly in
the kidneys, the spleen, the liver and the adrenals causing dysfunction. The disease is
associated with chronic inflammatory conditions, chronic infections and Familial Mediterranean
Fever. Rheumatoid arthritis is the major cause of AA amyloidosis in Western Europe and
North America. The market potential for this treatment is estimated at U.S. $180 million
annually.
" No specific therapy for amyloidosis exists, as yet," said Dr. Allan Cohen who
is Chairman of Neurochem’s Scientific Advisory Committee and internationally recognized
amyloid scientist "The FDA orphan drug designation will help us get this drug to market
more quickly and treat the patients who presently suffer from this serious disease,"
he added.
"Thousands of people around the world are presently suffering from Secondary Amyloidosis
but unfortunately do not have access to a cure," said Dr. Louis R. Lamontagne,
Neurochem's President and CEO. "FDA support is a decisive stepping-stone in the
process of obtaining approval of Fibrillex™," he added.
The provisions of the Orphan Drug Act in the United States are intended to stimulate
the research, development and approval of products that treat rare diseases. Under the
orphan drug program, Neurochem will receive seven years of market exclusivity following
approval of Fibrillex™. It will also be eligible to tax incentives for clinical
research, research study design assistance and possible access to grant funding to defray
costs of qualified clinical testing.
Fibrillex™ has undergone extensive toxicity and pharmacokinetic investigations in
animal species. Neurochem is especially proud of its excellent safety profile. The
drug's safety, tolerability and pharmacokinetic profile in healthy male adult volunteers
has also been excellent with no major adverse events reported. It is advancing to pivotal
Phase II/III clinical trials which should be completed in approximately 18-24 months.
Neurochem was established in 1993 and is an industry leader in the development of a novel,
proprietary series of compounds that inhibit the formation, deposit and toxic effects of
amyloid fibrils on cells within the body. Neurochem's research team has been working with
international amyloid experts on the development of therapeutic cures to three diseases:
Alzheimer's disease, Secondary Amyloidosis and Diabetes Type II. Neurochem has advanced
two drug candidates to clinical trials: Alzhemed™, for the treatment of Alzheimer's
disease and Fibrillex™, for Secondary Amyloidosis. Neurochem is located in Montreal,
Canada (www.neurochem.com).
Neurochem's financial investors comprise Atlas Venture, the Business Development Bank of
Canada, BioCapital, le Fonds de solidarité des travailleurs du Québec (FTQ),
Quorum Group of Companies, la Société Financière d'Innovation Inc.,
and la Société Innovatech du Grand Montréal.
Contacts for Neurochem Inc:
Dr. Louis Lamontagne
President & CEO
Dr. Lise Hébert
Vice President, Corporate Communications
lhebert@neurochem.com
275 Armand-Frappier
Laval (Quebec)
H7V 4A7
Tel: (450) 680-4500
Fax: (450) 680-4501
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