The European Medicines Agency granted the designation based on findings from the physician-led cannabidiol expanded access programme
GW Pharmaceuticals, a biopharmaceutical company focused on discovering, developing and commercialising novel therapeutics from its proprietary cannabinoid product platform, announced 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 US and Europe.
TS is a multisystem, genetic disease that causes benign tumours 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 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 neuro-behavioural 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 programme where the results of this open-label use of Epidiolex in children with TS have been very encouraging,” said Justin Gover, CEO of GW.
“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 EU 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.