Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in
innovative pharmaceutically-produced transdermal cannabinoid
therapies for orphan neuropsychiatric disorders, today announced
that the European Commission (EC) has granted orphan drug
designation to cannabidiol, the active ingredient in its
transdermal gel, Zygel™, for the treatment of 22q11.2 deletion
syndrome (22q).
“We are pleased to have been granted orphan drug designation in
the U.S. and now the EU, which we believe reflects the unmet
medical need for new therapies to treat 22q,” said Armando Anido,
Chairman and Chief Executive Officer of Zynerba. “Orphan drug
designation is another important step forward in advancing Zygel as
a potential new treatment option for patients with 22q.”
Orphan drug designation is granted to medicines that treat,
prevent or diagnose a life-threatening or chronically debilitating
rare disease, with a prevalence in the European Union (EU) of not
more than 5 in 10,000, and with either no currently approved method
of diagnosis, prevention or treatment or with significant benefit
to those affected by the disease. The designation provides certain
benefits to Zynerba, including 10-year EU market exclusivity upon
regulatory approval, if received, reductions in European Medicines
Agency application fees, and access to protocol assistance.
The Company believes there are approximately 112,000 patients
with 22q in the EU and approximately 129,000 in Europe including
the United Kingdom.
Zygel was previously granted orphan drug designation by the EC
for the treatment of Fragile X syndrome (FXS) and by U.S. Food and
Drug Administration (FDA) for the treatment of FXS and 22q.
Additionally, the FDA has granted Fast Track designation for Zygel
for the treatment of behavioral symptoms associated with FXS.
In June 2022, the Company announced positive topline results
from the exploratory, open label Phase 2 INSPIRE trial of Zygel in
children and adolescents with 22q (Press Release). Based on the
positive Phase 2 data, the Company requested and has been granted
an initial meeting with the FDA before the end of 2022 to obtain
feedback on the Phase 2 data and regulatory pathway for Zygel in
patients with 22q.
About Zygel
Zygel is the first and only pharmaceutically-manufactured
cannabidiol formulated as a patent-protected permeation-enhanced
clear gel, designed to provide controlled drug delivery into the
bloodstream transdermally (i.e. through the skin). Recent studies
suggest that cannabidiol may modulate the endocannabinoid system
and improve certain behavioral symptoms associated with
neuropsychiatric conditions. Zygel is an investigational drug
product in development for the potential treatment of behavioral
symptoms associated with Fragile X syndrome (FXS), 22q11.2 deletion
syndrome (22q) and autism spectrum disorder (ASD). The Company has
received orphan drug designation for cannabidiol, the active
ingredient in Zygel, from the FDA and the European Commission in
the treatment of FXS and the treatment of 22q. Additionally, Zygel
has been designated a Fast Track development program for treatment
of behavioral symptoms of FXS.
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 83,000 people
living with 22q in the U.S. In addition, Zynerba believes that
there are approximately 112,000 patients with 22q in the EU and
approximately 129,000 in Europe if the United Kingdom is
included.
About Zynerba Pharmaceuticals, Inc.
Zynerba Pharmaceuticals is the leader in innovative
pharmaceutically-produced transdermal cannabinoid therapies for
orphan neuropsychiatric disorders. We are committed to improving
the lives of patients and their families living with severe,
chronic health conditions including Fragile X syndrome, 22q11.2
deletion syndrome and autism spectrum disorder. Learn more at
www.zynerba.com and follow us on Twitter at
@ZynerbaPharma.
Cautionary Note on Forward-Looking
Statements
This press release contains forward-looking statements within
the meaning of The Private Securities Litigation Reform Act of
1995. We may, in some cases, use terms such as “predicts,”
“believes,” “potential,” “proposed,” “continue,” “estimates,”
“anticipates,” “expects,” “plans,” “intends,” “may,” “could,”
“might,” “will,” “should” or other words that convey uncertainty of
future events or outcomes to identify these forward-looking
statements. Such statements are subject to numerous important
factors, risks and uncertainties that may cause actual events or
results to differ materially from the Company’s current
expectations. Management’s expectations and, therefore, any
forward-looking statements in this press release could also be
affected by risks and uncertainties relating to a number of other
factors, including the following: the Company’s cash and cash
equivalents may not be sufficient to support its operating plan for
as long as anticipated; the Company’s expectations, projections and
estimates regarding expenses, future revenue, capital requirements,
incentive and other tax credit eligibility, collectability and
timing, and availability of and the need for additional financing;
the Company’s ability to obtain additional funding to support its
clinical development programs; the results, cost and timing of the
Company’s clinical development programs, including any delays to
such clinical trials relating to enrollment or site initiation;
clinical results for the Company’s product candidates may not be
replicated or continue to occur in additional trials and may not
otherwise support further development in a specified indication or
at all; actions or advice of the U.S. Food and Drug Administration,
the European Medicines Agency and other foreign regulatory agencies
may affect the design, initiation, timing, continuation and/or
progress of clinical trials or result in the need for additional
clinical trials; the Company’s ability to obtain and maintain
regulatory approval for its product candidates, and the labeling
under any such approval; the Company’s reliance on third parties to
assist in conducting pre-clinical and clinical trials for its
product candidates; delays, interruptions or failures in the
manufacture and supply of the Company’s product candidates the
Company’s ability to commercialize its product candidates; the size
and growth potential of the markets for the Company’s product
candidates, and the Company’s ability to service those markets; the
Company’s ability to develop sales and marketing capabilities,
whether alone or with potential future collaborators; the rate and
degree of market acceptance of the Company’s product candidates;
the Company’s expectations regarding its ability to obtain and
adequately maintain sufficient intellectual property protection for
its product candidates; the extent to which health epidemics and
other outbreaks of communicable diseases, including COVID-19, could
disrupt our operations or adversely affect our business and
financial conditions; and the extent to which inflation or global
instability, including political instability, may disrupt our
business operations or our financial condition. This list is not
exhaustive and these and other risks are described in the Company’s
periodic reports, including the annual report on Form 10-K,
quarterly reports on Form 10-Q and current reports on Form 8-K,
filed with or furnished to the Securities and Exchange Commission
and available at www.sec.gov. Any forward-looking statements that
the Company makes in this press release speak only as of the date
of this press release. The Company assumes no obligation to update
forward-looking statements whether as a result of new information,
future events or otherwise, after the date of this press
release.
Zynerba Contact
Peter VozzoICR WestwickeOffice: 443.213.0505Cell:
443.377.4767Peter.Vozzo@Westwicke.com
Zynerba Pharmaceuticals (NASDAQ:ZYNE)
Historical Stock Chart
From Jun 2024 to Jul 2024
Zynerba Pharmaceuticals (NASDAQ:ZYNE)
Historical Stock Chart
From Jul 2023 to Jul 2024