GW Pharmaceuticals Receives Orphan Drug Designation from the European Medicines Agency (EMA) for Cannabidiol for the Treatmen...
February 27 2018 - 7:00AM
GW Pharmaceuticals plc (Nasdaq:GWPH) (“GW,” “the Company” or “the
Group”), a biopharmaceutical company focused on discovering,
developing and commercializing novel therapeutics from its
proprietary cannabinoid product platform, today announced that the
European Medicines Agency (EMA) has granted orphan drug designation
for cannabidiol (CBD) for the treatment of tuberous sclerosis (TS).
GW is currently recruiting a Phase 3 clinical trial of Epidiolex®
(cannabidiol) as an adjunctive therapy for the treatment of
seizures associated with TS, with data expected in the second half
of 2018. Subject to positive results, GW expects to submit
regulatory applications in 2019 for Epidiolex in TS in both the
U.S. and Europe.
TS is multisystem, genetic disease that causes
benign tumors to grow in the brain and on other vital organs. TS
can be life threatening in patients with severe symptoms, including
drug resistant seizures and kidney failure. Up to 80 to 90%
of individuals with TS will develop epilepsy during their lifetime,
with onset typically in childhood. The seizures are often severe,
and up to two-thirds of TS patients do not respond adequately to
available medical therapies. There are significant co-morbidities
associated with TS including cognitive impairment, autism spectrum
disorders, developmental delay with severe learning disability and
neurobehavioral disorders in individuals with TS.
“GW‘s decision to evaluate Epidiolex in patients
with tuberous sclerosis is based on findings from the physician-led
Epidiolex expanded access program where the results of this
open-label use of Epidiolex in children with TS have been very
encouraging,” stated Justin Gover, GW’s Chief Executive Officer.
“GW has successfully submitted regulatory applications for
Epidiolex in both the US and Europe for the treatment of seizures
associated with Lennox-Gastaut syndrome and Dravet syndrome. TS
represents a near term opportunity to expand the potential
indications for Epidiolex and reflects GW’s ongoing commitment to
addressing the needs of patients with highly treatment-resistant
seizures.”
The EMA orphan designation is a status assigned
to a medicine intended for use against a rare condition (prevalence
of the condition in the European Union must not be more than 5 in
10,000) and allows a pharmaceutical company to benefit from
incentives offered by the EU to develop a medicine for the
treatment, prevention or diagnosis of a disease that is
life-threatening or a chronically debilitating rare disease. These
incentives can include reduced fees and protection from competition
once the medicine is placed on the market.
GW has already received orphan drug designation
from the FDA for CBD in the treatment of TS.
About Tuberous Sclerosis
(TS)
Tuberous sclerosis, also called tuberous
sclerosis complex (TSC), is a rare, multi-system genetic disease
that causes benign tumors to grow in the brain and on other vital
organs such as the kidneys, heart, eyes, lungs, and skin. It
usually affects the central nervous system and results in a
combination of symptoms including seizures, developmental delay,
behavioral problems, skin abnormalities, and kidney disease. At the
time of designation, tuberous sclerosis affected approximately 1 in
10,000 people in the European Union. This was equivalent to a total
of around 52,000 people.* TSC occurs in all races and ethnic
groups, and in both genders. The majority of children with TS have
onset of seizures during the first year of life, and up to one
third of children with TS will develop infantile spasms. Almost all
seizure types can be seen in individuals with tuberous sclerosis,
including tonic, clonic, tonic-clonic, atonic, myoclonic, atypical
absence, partial, and complex partial. The seizures are often
severe, and up to two-thirds of TS patients do not respond
adequately to available medical therapies. There are significant
co-morbidities associated with TS including cognitive impairment,
autism spectrum disorders, and neurobehavioral disorders in
individuals with TS.
*For the purpose of the designation, the number of patients
affected by the condition is estimated and assessed on the basis of
data from the European Union (EU 28), Norway, Iceland and
Liechtenstein. This represents a population of 515,700,000
(Eurostat 2017).
References:
https://www.ninds.nih.gov/Disorders/Patient-Caregiver-Education/Fact-Sheets/Tuberous-Sclerosis-Fact-Sheet#3220_1
http://www.tsalliance.org/pages.aspx?content=585
http://www.medscape.com/viewarticle/495644
About GW Pharmaceuticals plc and
Greenwich Biosciences
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, along with its U.S.
subsidiary Greenwich Biosciences, is advancing an orphan drug
program in the field of childhood-onset epilepsy with a focus on
Epidiolex (cannabidiol), for which GW has submitted regulatory
applications in the U.S. and Europe for the adjunctive treatment of
Lennox-Gastaut syndrome and Dravet syndrome. The Company continues
to evaluate Epidiolex in additional rare epilepsy conditions and
currently has ongoing clinical trials in Tuberous Sclerosis Complex
and Infantile Spasms. GW commercialized the world’s first
plant-derived cannabinoid prescription drug, Sativex® (nabiximols),
which is approved for the treatment of spasticity due to multiple
sclerosis in numerous countries outside the United States and for
which the company is now planning a US Phase 3 trial. The Company
has a deep pipeline of additional cannabinoid product candidates
which includes compounds in Phase 1 and 2 trials for epilepsy,
gliobastoma, and schizophrenia. For further information, please
visit www.gwpharm.com.
Forward-looking statements
This news release contains forward-looking
statements that reflect GW's current expectations regarding future
events, including statements regarding financial performance, the
timing of clinical trials, the timing and outcomes of regulatory or
intellectual property decisions, the relevance of GW products
commercially available and in development, the clinical benefits of
Epidiolex (cannabidiol) and the safety profile and commercial
potential of Epidiolex. 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 GW’s research strategies, the
applicability of the discoveries made therein, the successful and
timely completion and 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 and uncertainties associated with an
investment in GW can be found in GW’s filings with the U.S.
Securities and Exchange Commission, including the most recent Form
20-F filed on 4 December 2017. 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 |
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Stephen Schultz, VP Investor Relations (U.S.) |
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280 2424 / 401 500 6570 |
EU Media
Enquiries:FTI Consulting |
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Con
Franklin |
+44
(0) 7817 573 659 |
U.S. Media Enquiries:Sam Brown Inc.
Healthcare Communications |
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Christy Curran
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615 414 8668 |
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