Neurochem receives approvable letter for eprodisate (KIACTA(TM)) for treatment of AA amyloidosis
ECUBLENS, Switzerland, July 17 /CNW Telbec/ - Neurochem (International)
Limited (Neurochem), a wholly-owned subsidiary of Neurochem Inc. (NASDAQ:
NRMX; TSX: NRM), announces that the Company has received a second approvable
letter from the United States Food and Drug Administration (FDA) for
eprodisate (KIACTA(TM)) for the treatment of Amyloid A (AA) amyloidosis.
In its action letter, the FDA indicates that the Phase II/III clinical
trial provided some evidence of the effectiveness of eprodisate (KIACTA(TM))
in the treatment of the renal manifestations of amyloidosis; however, the FDA
also indicated that an additional efficacy trial with a target p-value of
0.05 will be necessary before the FDA could approve the investigational
product candidate. Further, the approvable letter states that additional
submissions, filed by Neurochem as part of its complete response to this
approvable letter, may address issues raised in this letter. The FDA has
indicated that such additional submissions could persuade the agency to
eliminate the requirement for an additional trial.
The FDA also asked for additional information, including further
pharmacokinetic studies, and again acknowledged that a QT clinical study
should be submitted as part of a Phase IV (post-approval) commitment.
The Company expects to file a complete response to this approvable letter
in the near future.
"We are disappointed that the FDA has not yet approved KIACTA(TM). This
is a rare disease with no specific treatment available for the patients,"
commented Dr. Francesco Bellini, Neurochem's Chairman, President and CEO. "We
will continue to work closely with the agency with the hope of securing final
approval of this product candidate," he concluded.
About eprodisate (KIACTA(TM))
Eprodisate (KIACTA(TM)) was investigated in an international, randomized,
double-blind, placebo-controlled, and parallel-designed Phase II/III clinical
trial in which 183 AA amyloidosis patients were enrolled at 27 sites around
the world (Eprodisate for AA Amyloidosis Trial, EFAAT). Patients who completed
the clinical trial were eligible for enrollment in an ongoing open-label
extension study, some of whom have now been receiving eprodisate (KIACTA(TM))
for more than five years. Eprodisate (KIACTA(TM)) has received orphan drug
status in the United States, the European Union and in Switzerland.
Neurochem Inc. sponsored the EFAAT study.
About Amyloid A (AA) Amyloidosis
A progressive and fatal condition, AA amyloidosis 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 and progression to dialysis/end-stage renal
disease is the most common clinical manifestation in this disease. Currently,
there is no approved therapy to treat AA amyloidosis and about half of all
patients diagnosed with the disease die within five years of diagnosis.
About Neurochem
Neurochem Inc. is focused on the development and commercialization of
innovative therapeutics to address critical unmet medical needs. Eprodisate
(KIACTA(TM)) is currently being developed for the treatment of Amyloid A (AA)
amyloidosis, and is under regulatory review for marketing approval by the
United States Food and Drug Administration, European Medicines Agency and
Swissmedic. Tramiprosate (ALZHEMED(TM)), for the treatment of Alzheimer's
disease, has completed a Phase III clinical trial in North America and is
currently in a Phase III clinical trial in Europe, while tramiprosate
(CEREBRIL(TM)), for the prevention of Hemorrhagic Stroke caused by Cerebral
Amyloid Angiopathy, has completed a Phase IIa clinical trial.
To Contact Neurochem
For additional information on Neurochem and its drug development
programs, please call the North American toll-free number 1 877 680-4500 or
visit our Web Site at www.neurochem.com.
This news release contains forward-looking statements regarding
eprodisate (KIACTA(TM)), as well as regarding continuing and further
development efforts. These statements are based on the current analysis and
expectations of management. Drug development necessarily involves numerous
risks and uncertainties, which could cause actual results to differ materially
from this current analysis and these expectations. Analysis regarding the
results of clinical trials may not provide definitive results regarding
safety, tolerability or therapeutic benefits. There is no certainty that
regulators will ultimately approve eprodisate (KIACTA(TM)) for sale to the
public. Risks and uncertainties may include: failure to demonstrate the
safety, tolerability and efficacy of our product, that actual results may vary
once the final and quality-controlled verification of data and analyses has
been completed, the expense and uncertainty of obtaining regulatory approval,
including from the FDA, and the possibility of having to conduct additional
clinical trials. Further, even if regulatory approval is obtained, therapeutic
products are generally subject to: stringent on-going governmental regulation,
challenges in gaining market acceptance, and competition. Neither Neurochem
Inc., nor Neurochem (International) Limited undertake any obligation to
publicly update any forward-looking statements, whether as a result of new
information, future events, or otherwise. Please see Neurochem Inc.'s Annual
Information Form for further risk factors that might affect Neurochem Inc.,
Neurochem (International) Limited and their respective businesses.
For further information: Lise Hébert, PhD, Vice President, Corporate Communications, (450) 680-4572, lhebert@neurochem.com
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