Zynerba Pharmaceuticals Receives Orphan Drug Designation for Cannabidiol for the Treatment of 22q11.2 Deletion Syndrome
September 17 2020 - 1:55PM
Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in
innovative pharmaceutically-produced transdermal cannabinoid
therapies for rare and near-rare neuropsychiatric disorders, today
announced that the U.S. Food and Drug Administration has granted
orphan drug designation for cannabidiol (CBD) for use in treating
22q11.2 deletion syndrome (22q). 22q is a rare midline condition
featuring physical abnormalities and debilitating neuropsychiatric
and behavioral symptoms including anxiety, withdrawn behavior, and
social interaction problems.
“Zynerba is committed to developing Zygel™ CBD gel
in certain rare and near-rare conditions, including 22q, for which
there is an urgent need for new, innovative therapeutics,” said
Armando Anido, Chairman and Chief Executive Officer of Zynerba. “We
are pleased that the FDA shares our sense of urgency regarding the
development of effective therapeutics in this important patient
population. The receipt of this designation represents another
important milestone for us, and we look forward to working closely
with the FDA to develop Zygel in pediatric and adolescent patients
with 22q as expeditiously as possible.”
About Orphan Drug DesignationUnder
the Orphan Drug Act (ODA), the FDA may grant orphan drug
designation to drugs intended to treat rare diseases or conditions
that affect fewer than 200,000 individuals in the U.S. The first
NDA applicant to receive FDA approval for a particular active
moiety to treat a particular disease with FDA orphan drug
designation is entitled to various incentives of the ODA, including
tax credits for qualified clinical testing, waiver of new drug
application (NDA) / biologics license application (BLA) user fees,
and eligibility for a seven-year exclusive marketing period for
that drug and use upon marketing approval.
About 22q11.2 Deletion Syndrome (22q)As the
second most common chromosomal disorder after Down syndrome, 22q is
caused by a small missing piece of the 22nd chromosome. The
deletion occurs near the middle of the chromosome at a location
designated q11.2. It is considered a mid-line condition, with
physical symptoms including characteristic palate abnormalities,
heart defects, immune dysfunction, and esophageal / GI issues, as
well as debilitating neuropsychiatric and behavioral challenges.
Anxiety is among the most common neuropsychiatric symptoms of 22q
and researchers have found that for children with 22q, anxiety is
linked to poorer adaptive behaviors such as self-care and
communication skills that affect daily life. Children with 22q also
experience withdrawn behavior, ADHD, cognitive impairment, and
autism spectrum disorder that affect communication and social
interaction. Later in life, they are at an increased risk of
developing mental illnesses such as schizophrenia. It is estimated
that 22q occurs in between one in 3,000 and one in 6,000 live
births, suggesting that there are approximately 81,000 people
living with 22q in the U.S.
About Zynerba Pharmaceuticals, Inc. Zynerba
Pharmaceuticals is the leader in pharmaceutically-produced
transdermal cannabinoid therapies for rare and near-rare
neuropsychiatric disorders. We are committed to improving the lives
of patients and their families living with severe, chronic health
conditions including Fragile X syndrome, autism spectrum disorder,
22q11.2 deletion syndrome, and a heterogeneous group of rare and
ultra-rare epilepsies known as developmental and epileptic
encephalopathies. Learn more at www.zynerba.com and follow us on
Twitter at @ZynerbaPharma.
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Statements
This press release contains forward-looking statements within
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factors, risks and uncertainties that may cause actual events or
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forward-looking statements in this press release could also be
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equivalents may not be sufficient to support its operating plan for
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periodic reports, including the annual report on Form 10-K,
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filed with or furnished to the Securities and Exchange Commission
and available at www.sec.gov. Any forward-looking statements
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press release.
Investor ContactWilliam Roberts, Vice
President, Investor Relations and Corporate CommunicationsZynerba
Pharmaceuticals484.581.7489 robertsw@zynerba.com
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