GW Pharmaceuticals Receives Orphan Drug Designation by FDA for Epidiolex(R) in the Treatment of Lennox-Gastaut Syndrome
28 Februar 2014 - 2:00PM
GW Pharmaceuticals plc (AIM:GWP) (Nasdaq:GWPH) ("GW") announced
today that the U.S. Food and Drug Administration (FDA) has granted
orphan drug designation for Epidiolex®, GW's product candidate that
contains plant-derived Cannabidiol (CBD) as its active ingredient,
for use in treating children with Lennox-Gastaut syndrome (LGS), a
rare and severe form of childhood-onset epilepsy. Epidiolex is an
oral liquid formulation of a highly purified extract of CBD, a
non-psychoactive molecule from the cannabis plant.
In November 2013, GW announced that the FDA had granted orphan
drug designation for Epidiolex in the treatment of Dravet syndrome.
Following the successful follow-on offering completed in January
2014, GW intends to advance a full clinical development program for
Epidiolex for the treatment of both LGS and Dravet syndromes.
Working with leading pediatric epilepsy specialists in the United
States, GW expects to commence an initial Phase 2 clinical trial
for Epidiolex in the second half of 2014. This trial, if
successful, is expected to lead to Phase 3 pivotal trial programs
in both Dravet syndrome and LGS intended to support New Drug
Applications with the FDA.
LGS is a severe form of childhood-onset epilepsy. Seizure types,
which vary among patients, include tonic (stiffening of the body,
upward deviation of the eyes, dilation of the pupils, and altered
respiratory patterns), atonic (brief loss of muscle tone and
consciousness, causing abrupt falls), atypical absence (staring
spells), and myoclonic (sudden muscle jerks). Most children with
LGS experience some degree of impaired intellectual functioning or
information processing, along with developmental delays, and
behavioral disturbances. LGS can be caused by brain malformations,
perinatal asphyxia, severe head injury, central nervous system
infection and inherited degenerative or metabolic conditions. It is
estimated that there are approximately 14,000-18,500 patients with
LGS in the United States and 23,000-31,000 patients with LGS in
Europe.
"This orphan designation for Epidiolex, GW's purified CBD
medicine, in Lennox-Gastaut syndrome follows on from the recent FDA
grant of orphan designation in Dravet syndrome. We are now in
active discussions with the FDA regarding the US regulatory pathway
for Epidiolex and believe that this medicine has the potential to
meet the significant unmet need of children suffering with severe
seizures where all options to control those seizures have been
exhausted," stated Justin Gover, GW's Chief Executive Officer. "GW
is responding to this need with the goal of providing an
FDA-approved prescription CBD medicine that physicians have
confidence in prescribing and parents can trust for quality,
consistency and access."
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 ingredient to treat a particular
disease with FDA orphan drug designation is entitled to a
seven-year exclusive marketing period in the U.S. for that product,
for that indication.
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 25 countries.
Sativex is also in Phase 3 clinical development as a potential
treatment of pain in people with advanced cancer. This Phase 3
program is intended to support the submission of a New Drug
Application for Sativex in cancer pain with the U.S. Food and Drug
Administration and in other markets around the world. GW has a deep
pipeline of additional cannabinoid product candidates, including
Epidiolex which has received Orphan Drug Designation from the FDA
for the treatment of Dravet and Lennox-Gastaut syndromes, severe,
drug-resistant epilepsy syndromes. GW's product pipeline also
includes compounds in Phase 1 and 2 clinical development for
glioma, ulcerative colitis, type‑2 diabetes, and schizophrenia. 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 and commercial value of the
company's compounds, 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 |
(Today) + 44 20 7831
3113 |
Justin Gover, Chief Executive Officer |
(Thereafter) + 44 1980 557000 |
Stephen Schultz, VP Investor Relations
(US) |
401 500 6570 |
|
|
FTI Consulting (Media
Enquiries) |
|
Ben Atwell / Simon Conway / John Dineen
(UK) |
+ 44 20 7831 3113 |
Robert Stanislaro (US) |
212 850 5657 |
|
|
Trout Group, LLC (US investor
relations) |
|
Todd James / Chad Rubin |
646 378 2900 |
GW Pharmaceuticals (NASDAQ:GWPH)
Historical Stock Chart
Von Apr 2024 bis Mai 2024
GW Pharmaceuticals (NASDAQ:GWPH)
Historical Stock Chart
Von Mai 2023 bis Mai 2024