GW Pharma’s cannabis drug granted orphan drug status by US FDA
GW Pharmaceuticals plc, a biopharmaceutical company focused on discovering, developing and commercializing novel therapeutics from its proprietary cannabinoid product platform,announced on April 21 that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation for cannabidiol (CBD) for the treatment of Tuberous Sclerosis Complex (TSC).
Under the Orphan Drug Act, the FDA may grant orphan drug designation to drugs intended to treat a rare disease or condition – generally a disease or condition that affects fewer than 200,000 individuals in the United States.
TSC is a genetic disorder that causes non-malignant tumors to form in many different organs, with the brain and skin being the most commonly affected tissues. Its most common symptom is epilepsy which occurs in around 80 to 90% of TSC patients and is a significant cause of morbidity and mortality.The majority of children with TSC have onset of seizures during the first year of life, and up to one third of children with TSC will develop infantile spasms.Almost all seizure types can be seen in individuals with tuberous sclerosis complex. The seizures are often severe, and up to two-thirds of TSC patients do not respond adequately to available medical therapies. There are significant co-morbidities associated with TSC including cognitive impairment, autism spectrum disorders, and neurobehavioral disorders in individuals with TSC.
On April 11, 2016, GW announced it has commenced a Phase 3 clinical trial of Epidiolex as an adjunctive therapy for the treatment of seizures associated with TSC.Epidiolex, the company’s lead cannabinoid product candidate, is a liquid formulation of pure plant-derived CBD, which is in development for the treatment of a number of rare pediatric epilepsy disorders. GW has conducted extensive pre-clinical research of CBD in epilepsy since 2007.This research has shown that CBD has significant anti-epileptiform and anticonvulsant activity using a variety of in vitro and in vivo models and has the ability to treat seizures in acute animal models of epilepsy with significantly fewer side effects than existing anti-epileptic drugs.
TSC is the third orphan indication that GW is targeting within its Epidiolex (CBD) clinical development programme, which includes four Phase 3 pivotal trials in Dravet syndrome and Lennox-Gastaut syndrome, both rare and catastrophic forms of childhood-onset epilepsy.GW is currently evaluating additional clinical development programmes in other orphan seizure disorders.
Source: GW Pharm
Category: Features, Pharmaceuticals
