British biopharmaceutical firm GW Pharmaceuticals has received orphan drug designation from the US Food and Drug Administration (FDA) for its Epidiolex as a treatment for Lennox-Gastaut syndrome (LGS), a rare and severe form of childhood-onset epilepsy.
GW’s product candidate Epidiolex is an oral liquid formulation of a highly purified extract of plant-derived Cannabidiol (CBD), a non-psychoactive molecule from the cannabis plant.
Lennox-Gastaut syndrome is a severe form of childhood-onset epilepsy and most children with it experience some degree of impaired intellectual functioning or information processing, along with developmental delays and behavioural disturbances.
The syndrome can be caused by brain malformations, perinatal asphyxia, severe head injury, central nervous system infection, as well as inherited degenerative or metabolic conditions.
GW chief executive officer Justin Gover said: “We are now in active discussions with the FDA regarding the US regulatory pathway for Epidiolex and believe that this medicine has the potential to meet the significant unmet need of children suffering with severe seizures where all options to control those seizures have been exhausted.
“GW is responding to this need with the goal of providing an FDA-approved prescription CBD medicine that physicians have confidence in prescribing and parents can trust for quality, consistency and access.”
In 2013, the FDA conferred the orphan drug status to the cannabidiol drug ‘Epidiolex’ for treatment of Dravet syndrome, a genetic epilepsy condition that is drug resistant.
The FDA orphan drug designation entitles GW to a seven-year exclusive marketing period in the US for Epidiolex as treatment of Dravet syndrome and LGS.
At present, the company intends to advance a full clinical development programme for Epidiolex for the treatment of both Lennox-Gastaut syndrome and Dravet syndrome.
The company expects to initiate an initial Phase II clinical trial for Epidiolex in the second half of 2014 and if successful it is expected to lead to Phase III pivotal trial programmes in both Dravet syndrome and LGS, intended to support new drug applications (NDA) with the FDA.