MELBOURNE, Australia,
Sept. 5, 2014 /PRNewswire/
-- Prana Biotechnology (ASX: PBT) has today announced the US
Food and Drug Administration (FDA) has granted Orphan Drug
designation to PBT2 for the treatment of Huntington Disease.
Orphan drug designation is granted by the FDA to promote the
development of drugs for diseases affecting less than 200,000
people in the United States.
Orphan drug designation entitles Prana to seven years of market
exclusivity for the use of PBT2 in the treatment of Huntington
disease; protocol assistance by the FDA to optimize drug
development in the preparation of a dossier that will meet
regulatory requirements; and reduced fees associated with applying
for market approval.
The company will also be applying for Orphan Drug designation in
Europe and other
jurisdictions.
Huntington disease is a neurodegenerative genetic disorder that
affects muscle coordination and leads to cognitive decline and
behavioral symptoms. More than 30,000 people in the US have
Huntington disease and there are currently no treatments for the
cognition or executive function symptoms of the disease.
Prana announced in February 2014
that its lead MPAC (Metal Protein Attenuating Compound) PBT2 had
met its primary end point of safety and tolerability, and improved
measures of cognitive performance - a secondary endpoint in its
Reach2HD Phase 2 clinical trial involving 109 people with
Huntington disease.
"The FDA's decision to grant Orphan Drug designation is a
reflection of the high unmet need for neurological drugs that can
slow, halt or improve the decline of cognition and allow sufferers
to have a better quality of life," said Prana Chairman and CEO
Geoffrey Kempler.
"We are very pleased to have received Orphan Drug designation
from the FDA, which is a highly important input to determining the
commercialisation pathway for PBT2 for the treatment of Huntington
disease, and to bring to market as quickly as possible an improved
treatment for people suffering from Huntington disease."
Prana is preparing its Post Phase 2 Trial dossier for submission
to the FDA to commence discussions on the next development steps
for PBT2.
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Contacts:
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Investor
Relations
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Media
Relations
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Rebecca
Wilson
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Ben Oliver
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T: +61 3 8866
1216
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T: +61 3 8866
1233
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E:
rwilson@buchanwe.com.au
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E:
boliver@buchanwe.com.au
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About Prana Biotechnology Limited
Prana Biotechnology was established to commercialise research
into Alzheimer's disease, Huntington disease and other
neurodegenerative and movement disorders. The Company was
incorporated in 1997 and listed on the Australian Stock Exchange in
March 2000 and listed on NASDAQ in
September 2002. Researchers at prominent international
institutions including The University of
Melbourne, The Mental Health Research Institute
(Melbourne) and Massachusetts
General Hospital, a teaching hospital of Harvard Medical School, contributed to the
discovery of Prana's technology.
Forward Looking Statements
This press release contains "forward-looking statements"
within the meaning of section 27A of the Securities Act of 1933 and
section 21E of the Securities Exchange Act of 1934. The
Company has tried to identify such forward-looking statements by
use of such words as "expects," "intends," "hopes," "anticipates,"
"believes," "could," "may," "evidences" and "estimates," and other
similar expressions, but these words are not the exclusive means of
identifying such statements. Such statements include, but are
not limited to any statements relating to the Company's drug
development program, including, but not limited to the initiation,
progress and outcomes of clinical trials of the Company's drug
development program, including, but not limited to, PBT2, and any
other statements that are not historical facts. Such
statements involve risks and uncertainties, including, but not
limited to, those risks and uncertainties relating to the
difficulties or delays in financing, development, testing,
regulatory approval, production and marketing of the Company's drug
components, including, but not limited to, PBT2, the ability of the
Company to procure additional future sources of financing,
unexpected adverse side effects or inadequate therapeutic efficacy
of the Company's drug compounds, including, but not limited to,
PBT2, that could slow or prevent products coming to market, the
uncertainty of patent protection for the Company's intellectual
property or trade secrets, including, but not limited to, the
intellectual property relating to PBT2, and other risks
detailed from time to time in the filings the Company makes with
Securities and Exchange Commission including its annual reports on
Form 20-F and its reports on Form 6-K. Such statements are
based on management's current expectations, but actual results may
differ materially due to various factions including those risks and
uncertainties mentioned or referred to in this press release.
Accordingly, you should not rely on those forward-looking
statements as a prediction of actual future results.
SOURCE Prana Biotechnology