AVI BioPharma's Drug Candidate AVI-5038 Receives European Orphan Drug Designation for Duchenne Muscular Dystrophy
February 05 2010 - 8:00AM
Marketwired
AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based drugs,
today announced that it received an orphan drug designation from
the Committee for Orphan Medical Products of the European Medicines
Agency (EMEA) for AVI-5038, a drug candidate being developed by AVI
for the treatment of Duchenne Muscular Dystrophy (DMD). DMD is a
genetic muscle-wasting disease caused by failure to produce
dystrophin. The orphan drug designation potentially may provide AVI
up to 10 years of market exclusivity if the drug candidate is
approved for marketing in the European Union (EU). AVI-4658,
another drug being developed by AVI for DMD, received European
orphan drug designation in 2008, and also potentially may receive
up to 10 years of marketing exclusivity if approved in the EU.
"The EMEA's granting of orphan drug designation to AVI-5038
provides important regulatory support for our continuing commitment
to develop disease modifying drugs for DMD patients," stated Dr.
Leslie Hudson, President and CEO, AVI BioPharma, Inc. "We look
forward to the opportunity to report continuing progress in our DMD
program throughout the year, particularly with respect to our lead
DMD drug candidate, AVI-4658, which is in an ongoing Phase 1b/2
clinical trial."
Products granted an orphan drug designation by the EMEA are
intended for the diagnosis, prevention or treatment of
life-threatening or chronically debilitating conditions that affect
no more than five in 10,000 people in the EU, or are medicines
which, for economic reasons, would unlikely be developed without
incentives. The aim of the EU orphan medicines designation is to
stimulate research and development of medicinal products for rare
diseases by providing incentives to the pharmaceutical industry.
This initiative helps to give patients suffering from rare diseases
access to the same quality of treatment as other patients.
Applications for designation of orphan medicines are reviewed by
the EMEA through the Committee for Orphan Medicinal Products.
About Duchenne Muscular Dystrophy
DMD is one of the most common fatal genetic disorders to affect
children around the world. Approximately one in every 3,500 boys
worldwide is afflicted with DMD with 20,000 new cases reported each
year. It is a devastating and incurable muscle-wasting disease
associated with specific inborn errors in the gene that codes for
dystrophin, a protein that plays a key structural role in muscle
fiber function. Symptoms usually appear in male children by age
three. Progressive muscle weakness of the legs and pelvis
eventually spreads to the arms, neck, and other areas. By age 10,
braces may be required for walking, and most patients are confined
to a wheelchair by age 12. Eventually, this progresses to complete
paralysis and increasing difficulty in breathing requiring
ventilatory support. The condition is terminal and death usually
occurs before the age of 30. The outpatient cost of care for a
non-ambulatory DMD boy is among the highest of any disease. There
is currently no cure for DMD, but for the first time ever, there
are promising therapies in or moving into development.
About AVI BioPharma
AVI BioPharma is focused on the discovery and development of
RNA-based drugs utilizing proprietary derivatives of its antisense
chemistry (morpholino-modified phosphorodiamidate oligomers or
PMOs) that can be applied to a wide range of diseases and genetic
disorders through several distinct mechanisms of action. Unlike
other RNA therapeutic approaches, AVI's antisense technology has
been used to directly target both messenger RNA (mRNA) and its
precursor (pre-mRNA), allowing for both up- and down-regulation of
targeted genes and proteins. AVI's RNA-based drug programs are
being evaluated for the treatment of Duchenne muscular dystrophy,
including an ongoing systemic Phase 1b/2 clinical trial of exon
skipping with AVI-4658. AVI's antiviral programs have demonstrated
promising outcomes in Ebola Zaire and Marburg Musoke virus
infections and may prove applicable to other viral targets such as
Junín, influenza, HCV or Dengue viruses. For more information,
visit www.avibio.com.
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Reform Act of 1995: The statements that are not historical facts
contained in this release are forward-looking statements that
involve risks and uncertainties, including, but not limited to, the
results of research and development efforts, the results of
preclinical and clinical testing, the effect of regulation by the
FDA and other agencies, the impact of competitive products, product
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other risks detailed in the company's Securities and Exchange
Commission filings.
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