GW Pharmaceuticals Receives Orphan Drug Designation from FDA for Cannabidiol for the Treatment of Tuberous Sclerosis Complex
April 21 2016 - 7:00AM
GW Pharmaceuticals plc (Nasdaq:GWPH) (AIM:GWP) (“GW” or “the
Company”), a biopharmaceutical company focused on discovering,
developing and commercializing novel therapeutics from its
proprietary cannabinoid product platform, today announced that the
U.S. Food and Drug Administration (FDA) has granted Orphan Drug
Designation for cannabidiol (CBD) for the treatment of Tuberous
Sclerosis Complex (TSC). TSC is a rare genetic disorder, the
most common symptom of which is epilepsy. Epilepsy occurs in around
80-90% of TSC patients and is a significant cause of morbidity and
mortality. TSC is the third orphan indication that GW is targeting
within its Epidiolex® (CBD) clinical development program, which
includes four Phase 3 pivotal trials in Dravet syndrome and
Lennox-Gastaut syndrome, both rare and catastrophic forms of
childhood-onset epilepsy. On April 11, 2016, GW announced it has
commenced a Phase 3 clinical trial of Epidiolex® as an adjunctive
therapy for the treatment of seizures associated with TSC.
To obtain information about this clinical trial
or eligibility criteria, the treating physician should contact:
medicaldirector@gwpharm.com
About Orphan Drug
Designation
Under the Orphan Drug Act, the FDA may grant
orphan drug designation to drugs intended to treat a rare disease
or condition - generally a disease or condition that affects 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
a seven-year exclusive marketing period for that drug and use,
unless the applicant is unable to assure sufficient quantities of
the drug or another applicant is able to demonstrate that its
version of the drug is clinically superior.
About Tuberous Sclerosis Complex
(TSC)
TSC is a genetic disorder that causes
non-malignant tumors to form in many different organs, with the
brain and skin being the most commonly affected tissues. There are
approximately 50,000 in the United States and nearly 1 million
people worldwide estimated to have TSC1. Epilepsy is the most
common presenting symptom in TSC and is also the most common
medical disorder in TSC. Up to 80 to 90% of individuals with TSC
will develop epilepsy during their lifetime, with onset typically
in childhood. The majority of children with TSC have onset of
seizures during the first year of life, and up to one third of
children with TSC will develop infantile spasms. Almost all seizure
types can be seen in individuals with TSC, including tonic, clonic,
tonic-clonic, atonic, myoclonic, atypical absence, partial, and
complex partial. The seizures are often severe, and up to
two-thirds of TSC patients do not respond adequately to available
medical therapies. There are significant co-morbidities associated
with TSC including cognitive impairment, autism spectrum disorders,
and neurobehavioral disorders in individuals with TSC. 2
References:1: http://www.tsalliance.org/pages.aspx?content=5852:
http://www.medscape.com/viewarticle/495644
About Epidiolex
Epidiolex, GW’s lead cannabinoid product
candidate, is a liquid formulation of pure plant-derived CBD, which
is in development for the treatment of a number of rare pediatric
epilepsy disorders. GW has conducted extensive pre-clinical
research of CBD in epilepsy since 2007. This research has shown
that CBD has significant anti-epileptiform and anticonvulsant
activity using a variety of in vitro and in vivo models and has the
ability to treat seizures in acute animal models of epilepsy with
significantly fewer side effects than existing anti-epileptic
drugs. To date, GW has received Orphan Drug Designation from the
U.S. Food and Drug Administration (FDA) for Epidiolex in the
treatment of Dravet syndrome, Lennox-Gastaut syndrome and TSC.
Additionally, GW has received Fast Track Designation from the FDA
and Orphan Designation from the European Medicines Agency for
Epidiolex for the treatment of Dravet syndrome. GW is currently
evaluating additional clinical development programs in other orphan
seizure disorders.
About GW Pharmaceuticals
plc
Founded in 1998, GW is a biopharmaceutical
company focused on discovering, developing and commercializing
novel therapeutics from its proprietary cannabinoid product
platform in a broad range of disease areas. GW commercialized the
world’s first plant-derived cannabinoid prescription drug,
Sativex®, which is approved for the treatment of spasticity due to
multiple sclerosis in 28 countries outside the United States. GW is
advancing an orphan drug program in the field of childhood epilepsy
with a focus on Epidiolex® (cannabidiol), which is in Phase 3
clinical development for the treatment of Dravet syndrome,
Lennox-Gastaut syndrome and Tuberous Sclerosis Complex. GW has a
deep pipeline of additional cannabinoid product candidates which
includes compounds in Phase 1 and 2 trials for glioma, type 2
diabetes, schizophrenia and epilepsy. For further information,
please visit www.gwpharm.com.
Forward-looking statements
This news release may contain forward-looking
statements that reflect GWs current expectations regarding future
events, including statements regarding the therapeutic benefit,
safety profile and commercial value of the company's
investigational drug Epidiolex®, the development and
commercialization of Epidiolex, plans and objectives for product
development, plans and objectives for present and future clinical
trials and results of such trials, plans and objectives for
regulatory approval. Forward-looking statements involve risks and
uncertainties. Actual events could differ materially from
those projected herein and depend on a number of factors, including
(inter alia), the success of the GW’s research strategies, the
applicability of the discoveries made therein, the successful and
timely completion of uncertainties related to the regulatory
process, and the acceptance of Sativex®, Epidiolex®, and other
products by consumer and medical professionals. A further list and
description of risks, uncertainties and other risks associated with
an investment in GW can be found in GW’s filings with the U.S.
Securities and Exchange Commission. Existing and prospective
investors are cautioned not to place undue reliance on these
forward-looking statements, which speak only as of the date hereof.
GW undertakes no obligation to update or revise the information
contained in this press release, whether as a result of new
information, future events or circumstances or otherwise.
Enquiries:
GW Pharmaceuticals plc |
|
Stephen
Schultz, VP Investor Relations (U.S.) |
917 280
2424 / 401 500 6570 |
|
|
FTI Consulting (Media Enquiries) |
|
Ben
Atwell / Simon Conway |
+ 44 20
3727 1000 |
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|
FleishmanHillard (U.S. Media) |
|
Paddi
Hurley / Adam Silverstein |
212 453
2382 / 917 697 9313 |
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