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February 11, 2004
Neurochem granted Fast Track Designation from U.S. FDA
for Fibrillex™.
The Company plans to file its U.S. NDA in 2005.
- Neurochem, Inc. (NASDAQ: NRMX, TSX: NRM) - today announced that the U.S. Food and Drug Administration
(FDA) has designated the Company's most advanced investigational drug candidate, Fibrillex™, as
a Fast Track Product (FTP) for the treatment of secondary Amyloid A (AA) amyloidosis, a serious and
life-threatening condition which develops as a consequence of chronic infections or chronic
inflammatory diseases, such as Rheumatoid Arthritis (RA). Secondary AA amyloidosis (better known as
AA amyloidosis) has a poor prognosis, with a five-year survival rate of approximately 50%.
1 Left untreated, patients gradually progress to end-stage renal failure, which
is the cause of death in at least 35% of the cases.2,3
As a result of the FTP designation, Neurochem will submit a request for a rolling New Drug Application
(NDA) for Fibrillex™, enabling the FDA to commence review of portions of the application before
the end of the on-going two-year Phase II/III trial. Furthermore, the Company is eligible for priority
reviews by the FDA.
"AA amyloidosis is a severe disease. No specific therapy currently exists," stated Denis
Garceau, Ph.D., Vice President of Drug Development at Neurochem. "We are pleased that
Fibrillex™ received FTP designation. The designation reflects the FDA's acknowledgement of the
life-threatening nature of AA amyloidosis, which often results in end-stage renal disease. The FDA's
decision also highlights the Company's commitment to studying the effects of Fibrillex™ on
endpoints of clear clinical benefit, including preservation of kidney function and preventing patients
from undergoing dialysis."
The FTP designation, originally enacted under the FDA's Modernization Act of 1997, expedites the
development and review of a new drug that is intended for the prevention or treatment of a serious
or life-threatening condition, and demonstrates the potential of a drug candidate to address unmet
medical needs for such a condition.
This designation favorably adds to Fibrillex™'s existing orphan drug status designation in the
United States and Europe, which designation normally provides a product seven and ten years of
market exclusivity, respectively, upon market entry.
About Fibrillex™
The Phase II/III clinical trial for Fibrillex is a two-year, international, multi-center, randomized,
double-blind, placebo-controlled, and parallel-designed trial to evaluate the safety and efficacy of
Fibrillex™ in patients suffering from AA amyloidosis. Neurochem has successfully completed the
enrollment of 183 patients for the Phase II/III trial, which is being conducted at 27 sites located
across North America, Europe and Israel. Neurochem anticipates completing the trial by January 2005. To
date, 19 patients have completed the two-year treatment period with Fibrillex™. A two-year
open-label extension study for the drug candidate is ongoing.
Fibrillex™ is an oral drug candidate for the treatment of AA amyloidosis through the prevention
of amyloid fibril formation. Fibrillex™ belongs to the class of glycosaminoglycan (GAG) mimetics,
better know as disease-modifiers. It competes with natural GAGs for the same binding sites on the
AA protein. By preventing natural GAGs from binding to the AA protein, Fibrillex™ is expected
to prevent AA fibril formation and the deposition of AA fibrils in organs.
About AA Amyloidosis
AA amyloidosis is a progressive and fatal condition that occurs in a proportion of patients with
chronic inflammatory diseases, including RA, 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 kidney malfunction. 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.
About Neurochem
Neurochem is focused on the development and commercialization of innovative therapeutics for
neurological disorders. The Company's staged pipeline of proprietary, disease-modifying, oral products
addresses critical unmet medical needs. Fibrillex™, designated an orphan drug, is in a Phase
II/III clinical trial for AA amyloidosis. Alzhemed™ has completed a Phase II clinical trial for
the treatment of Alzheimer's Disease. Cerebril™ is in a Phase II trial for the prevention of
hemorrhagic stroke caused by cerebral amyloid angiopathy. For additional information on Neurochem,
please visit our website at: (www.neurochem.com).
1 - Pepys, M., et al. Amyloidosis. Oxford Textbook of Medicine (4th ed.) Oxford: Oxford University
Press 2003; 162-73.
2 - Gertz, M.A., et al. Medicine (Baltimore) 1991 Jul: 70(4); 246-56.
3 - Joss, N., et al. QJMed. 2000: 93; 535-42.
This news release contains forward-looking statements regarding the potential for Fibrillex™
and further development efforts. These statements are based on the current expectations of management.
Drug development involves numerous risks and uncertainties, which could cause actual results to differ
materially. Promising results and successes in early stage clinical trials do not ensure that later
stage or larger scale clinical trials will be successful. For instance, Fibrillex™ may not show
the same benefits, results or safety profile in later stage or larger scale clinical trials.
Additional risks and uncertainties include: the impact of general economic conditions, general
conditions in the pharmaceutical industry, changes in the regulatory environment in the
jurisdictions in which Neurochem does business, stock market volatility, fluctuations in costs,
and changes to the competitive environment due to consolidation or otherwise. Neurochem does not
undertake any obligation to publicly update its forward-looking statements, whether as a result of
new information, future events, or otherwise.
For further Information, please contact:
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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