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 European Commission (EC) has granted orphan drug
designation to cannabidiol, the active ingredient in its
transdermal gel, Zygel™, for the treatment of Fragile X syndrome
(FXS).
“Orphan drug designation by the EC is another important
milestone for Zygel,” said Armando Anido, Chairman and Chief
Executive Officer of Zynerba. “It underscores the urgent, unmet
medical need for patients diagnosed with Fragile X syndrome, and
will benefit us as we continue to advance our clinical programs and
prepare to bring Zygel to market.”
Zynerba is currently enrolling patients in RECONNECT, a
confirmatory pivotal Phase 3 trial of Zygel in children and
adolescents with FXS, and continues to expect topline results from
this trial in the second half of 2023. As previously announced, the
Company believes, based on the EMA’s scientific advice, that the
successful completion of the current development program for Zygel
in FXS will satisfy the requirements of a marketing authorization
application in the European Union (EU).
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 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 potentially provides
certain benefits to Zynerba, including 10-year EU market
exclusivity upon regulatory approval, if received, reductions in
EMA application fees, and access to protocol assistance.
The Company believes that there are approximately 105,000
patients with FXS in the EU and approximately 121,000 in Europe if
the United Kingdom (UK) is included. Approximately 60% of all
patients are believed to be completely methylated, which indicates
that there are approximately 73,000 patients with FXS who are
completely methylated in the EU and UK.
As previously disclosed, Zygel has also been granted orphan drug
designation by the U.S. Food and Drug Administration (FDA) for the
treatment of FXS and 22q11.2 deletion syndrome. Additionally, the
FDA has granted Fast Track designation for Zygel for the treatment
of behavioral symptoms associated with FXS. Based on latest census
data available, we believe there are approximately 78,000 patients
with FXS in the U.S., and approximately 47,000 of those patients
are completely methylated.
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 endocannabinois 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), autism spectrum
disorder (ASD), and 22q11.2 deletion syndrome (22q). Zygel has been
granted orphan drug designation by the FDA and the EC in the
treatment of FXS and by the FDA for the treatment of 22q.
Additionally, Zygel has been designated a Fast Track development
program for treatment of behavioral symptoms of FXS.
About Fragile X Syndrome (FXS)
FXS is a rare genetic developmental disability that is the
leading known cause of both inherited intellectual disability and
autism spectrum disorder, affecting 1 in 3,600 to 4,000 males and 1
in 4,000 to 6,000 females. It is the most common inherited
intellectual disability in males and a significant cause of
intellectual disability in females, and the leading genetic cause
of autism spectrum disorder (ASD). The disorder negatively affects
synaptic function, plasticity and neuronal connections, and results
in a spectrum of intellectual disabilities and behavioral symptoms,
such as social avoidance and irritability. In the U.S., there are
approximately 78,000 people suffering with FXS, and approximately
121,000 in the EU and UK. We believe that approximately 60% of all
patients with FXS have complete methylation of their FMR1 gene.
About Zynerba Pharmaceuticals, Inc.
Zynerba Pharmaceuticals is the leader in innovative
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, and 22q11.2 deletion syndrome. 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; and 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. 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
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