GW Pharmaceuticals PLC- ADR (NASDAQ:GWPH) a biopharmaceutical firm focused on discovering, advancing and commercializing innovative therapeutics, reported that the U.S. FDA has approved Orphan Drug Designation for CBD for the cure of Tuberous Sclerosis Complex.
TSC comes in the list of rare genetic disorders, and its most common symptom is epilepsy, which occurs in nearly 80% to 90% of TSC patient. Tuberous Sclerosis Complex is the third orphan symptom that GW Pharmaceuticals is aiming within its ‘Epidiolex®’ clinical development plan.
This clinical program includes 4 Phase III pivotal studies in Lennox-Gastaut syndrome and Dravet syndrome, both catastrophic and rare types of childhood-onset epilepsy. Earlier in month, GW reported that it has initiated a Phase III clinical study of ‘Epidiolex®’ as an adjunctive treatment for the cure of seizures related with TSC.
The product candidate
Epidiolex, GW Pharmaceuticals’ lead CBD product candidate is in development for the cure of a number of rare and complex pediatric epilepsy disorders. The company has performed thorough pre-clinical research of cannabinoids in epilepsy.
This research has highlighted that CBD has considerable anticonvulsant and anti-epileptiform activity using numerous in vivo and in vitro models and possess the ability to cure seizures in acute animal subjects of epilepsy with considerably fewer side effects compared to existing anti-epileptic medications.
To date, GW Pharmaceuticals has obtained Orphan Drug status from the U.S. FDA for Epidiolex targeting Dravet syndrome, TSC and Lennox-Gastaut syndrome. Additionally, the company has obtained Fast Track status from the FDA and also Orphan Designation from the EMA for Epidiolex for cure of Dravet syndrome. For now, the company is currently assessing new clinical development plans in other orphan seizure problems, and it will update on these development plans as and when they materializes.
In last trading session, the stock price of GW Pharmaceuticals surged 0.15% to close the trading session at $86.36.