GW Pharmaceuticals PLC Orphan Drug Designation for Cannabidiol in TSC (9096V)
21 April 2016 - 9:00PM
UK Regulatory
TIDMGWP
RNS Number : 9096V
GW Pharmaceuticals PLC
21 April 2016
GW Pharmaceuticals Receives Orphan Drug Designation from FDA for
Cannabidiol for the Treatment of Tuberous Sclerosis Complex
London, UK, 21 April 2016: 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(R)
(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(R) 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 TSC(1) . 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=585
2: 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(R), 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(R) (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(R), 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(R), Epidiolex(R), 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
FleishmanHillard (U.S. Media)
Paddi Hurley / Adam Silverstein 212 453 2382 / 917 697 9313
Peel Hunt LLP (UK NOMAD)
James Steel +44 20 7418 8900
This information is provided by RNS
The company news service from the London Stock Exchange
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