GW Pharmaceuticals Announces Epidiolex® Receives Orphan Designation from European Medicines Agency for the Treatment of Dravet Syndrome

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Oct 22, 2014

London, UK; 22 October 2014: GW Pharmaceuticals plc (Nasdaq: GWPH, AIM: GWP, “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 designation to GW’s investigational product Epidiolex® (cannabidiol or CBD) in the treatment of Dravet syndrome, a rare and catastrophic treatment-resistant form of childhood epilepsy.

In addition to this orphan designation by the EMA, GW has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for Epidiolex in the treatment of Dravet syndrome as well as orphan designations in both Dravet syndrome and Lennox-Gastaut syndrome (LGS). GW is about to commence a full clinical development program for Epidiolex in the treatment of both Dravet syndrome and LGS, working with leading pediatric epilepsy specialists across the U.S. The first Phase 2/3 clinical trial is due to commence in the coming weeks.

On October 14th, GW announced updated physician reports of Epidiolex treatment effect in children and young adults with treatment-resistant epilepsy participating in open-label, “expanded access” studies. Of the 58 patients in this report, 12 patients had Dravet syndrome. These Dravet syndrome patients reported a median overall reduction in convulsive seizure frequency of 51%-72% across a range of time points and analyses. The most common adverse events were somnolence and fatigue.

 “Dravet syndrome represents a very substantial unmet need in Europe and a significant therapeutic challenge as many of the children suffering with this condition are resistant to current treatments and have exhausted all options,” stated Justin Gover, GW’s Chief Executive Officer. “GW is now advancing a full clinical development program for Epidiolex in Dravet syndrome and looks forward to commencing this program in the coming weeks. We believe that the clinical effect and safety data recently released on Epidiolex support GW’s confidence in the prospect of ultimately enabling children with Dravet syndrome around the world to have access to an approved prescription CBD medicine.”

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.

About Dravet syndrome

Dravet syndrome, also known as Severe Myoclonic Epilepsy of Infancy (SMEI), is a rare and catastrophic form of epilepsy for which there is currently no cure. Seizures begin in the first year of life in an otherwise typically developing infant. Initial seizures are most often prolonged events (status epilepticus) and, in the second year of life, other seizure types emerge. All seizure types are remarkably resistant to medical therapy and the prognosis for Dravet syndrome is poor. Individuals with Dravet syndrome face a higher incidence of SUDEP (sudden unexplained death in epilepsy) and have associated co-morbid conditions, which also need to be properly managed. Children with Dravet syndrome do not outgrow this condition and it affects every aspect of their daily lives.

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 27 countries outside the United States. Sativex is also in Phase 3 clinical development as a potential treatment of pain associated with advanced cancer. This Phase 3 program has received Fast Track Designation from the U.S. Food and Drug Administration (FDA) and is intended to support the submission of a New Drug Application for Sativex in cancer pain with the FDA and in other markets around the world. GW has a deep pipeline of additional cannabinoid product candidates, including Epidiolex® in the treatment of childhood epilepsy, which has received Fast Track Designation from the FDA for Dravet syndrome as well as Orphan Drug Designations from the FDA in both the treatment of Dravet syndrome and Lennox-Gastaut syndrome. 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.

Note Regarding Expanded Access Studies

Expanded access studies are uncontrolled, carried out by individual investigators, and not typically conducted in strict compliance with Good Clinical Practices, all of which can lead to a treatment effect which may differ from that in placebo-controlled trials. Data from these studies provide only anecdotal evidence of efficacy for regulatory review, contain no control or comparator group for reference and are not designed to be aggregated or reported as study results. Moreover, data from such small numbers of patients may be highly variable.  Such information may not reliably predict data collected via systematic evaluation of the efficacy in company-sponsored clinical trials.  Reliance on such information may lead to Phase 2 and 3 clinical trials that are not adequately designed to demonstrate efficacy and could delay or prevent GW’s ability to seek approval of Epidiolex. Expanded access programs may provide supportive safety information for regulatory review.  Physicians conducting these studies may use Epidiolex in a manner inconsistent with the protocol, including in children with conditions different from those being studied in GW-sponsored trials.  Any adverse events or reactions experienced by subjects in the expanded access program may be attributed to Epidiolex and may limit GW’s ability to obtain regulatory approval with labeling that GW considers desirable, or at all.

Forward-looking statements

This news release may contain forward-looking statements that reflect GWs current expectations regarding future events, including statements regarding the benefits of the Company’s products including Epidiolex®, therapeutic and commercial value of 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, 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 3727 1000

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 3727 1000

Robert Stanislaro (US)

212 850 5657

Trout Group, LLC (US investor relations)

 

Todd James / Chad Rubin

646 378 2900

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