GW Pharmaceuticals plc (GWPH) Receives Orphan Drug Designation from the European Medicines Agency

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).