GW Pharmaceuticals granted orphan designation from EMA on cannabinoid product
Gw Pharmaceuticals Plc - American Depositary Shares
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19:31 08/12/22
Cambridge-based biopharmaceutical firm GW Pharmaceuticals has been granted orphan drug designation for its proprietary cannabinoid product platform for the treatment of tuberous sclerosis (TS) from the European Medicines Agency.
GW, which had been recruiting for a phase three clinical trial of its cannabidiol, Epidiolex, as an adjunctive therapy for the treatment of seizures associated with TS, expected to receive results from its study in the second half of 2018 and, subject to positive results, the Nasdaq-listed group planned to submit regulatory applications for Epidiolex in both the US and Europe in 2019.
Tuberculosis sclerosis, a multisystem, genetic disease that causes benign tumours to grow in the brain and on other vital organs, can be life-threatening in patients with severe symptoms, including drug-resistant seizures and kidney failure, with as many as 80-90% of individuals with TS likely to develop epilepsy during their lifetime.
The seizures are often severe, with up to two-thirds of patients failing to respond adequately to available medical therapies, leading to cognitive impairment, autism spectrum disorders, developmental delay with severe learning disabilities 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," said 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 medicines intended for use against rare conditions 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 chronically debilitating, including 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.
As of 1340 GMT, shares had picked up 2.62% to $118.33 each.