Auspex Pharmaceuticals,
Inc. ASPX, a biopharmaceutical company dedicated to developing
innovative medicines for people with movement disorders and other rare
diseases, today announced that the U.S. Food and Drug Administration (FDA) has
granted orphan drug designation of Auspex's investigational compound SD-809
for treatment of Huntington's disease. Auspex is currently evaluating the
efficacy and safety of SD-809 for treating chorea associated with this
disorder in a Phase 3 registration clinical trial and is on track to announce
topline data from this study in December 2014.
"The orphan drug designation of SD-809 marks a major regulatory milestone for
Auspex and a significant step forward in our goal to bring this novel
treatment approach to patients as quickly as possible," said Pratik Shah,
president and CEO of Auspex. "Huntington's disease is a rare and devastating
genetic disorder with very limited treatment options. The orphan drug
designation recognizes the significant need that exists among individuals
living with this disease."
The FDA's Orphan Drug program offers orphan status to drugs and biologics that
are intended for the treatment of rare diseases affecting fewer than 200,000
people in the U.S. The designation provides sponsors with development and
commercial incentives for designated compounds and medicines, including
eligibility for a seven-year period of market exclusivity in the U.S. after
product approval, FDA assistance in clinical trial design and an exemption
from FDA user fees.
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