GW Pharmaceuticals Receives Orphan Drug Designation from the European Medicines Agency (EMA) for Cannabidiol for the Treatment of Tuberous Sclerosis

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Feb 27, 2018

London, UK, Carlsbad, CA, February 27, 2018 – 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-She...

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

 

Stephen Schultz, VP Investor Relations (U.S.)

917 280 2424 / 401 500 6570

EU Media Enquiries:

FTI Consulting

 

Con Franklin

+44 (0) 7817 573 659

U.S. Media Enquiries:

Sam Brown Inc. Healthcare Communications

 

Christy Curran

615 414 8668

 

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