GW Pharma's orphan drug gains fast-track status
- GW Pharma, based in the UK, has developed an investigational drug for the treatment of Dravet syndrome---a rare form of severe, treatment-resistant childhood epilepsy.
- The drug, Epidiolex, is a cannabidiol, which is an active cannabinoid found in cannabis.
- Epidiolex now has the benefit of both fast-track status and orphan-drug desgination.
According to information from GW Pharma, there are approximately 5,400 children with Dravet in the US, and another 6,710 in Europe. These children not only suffer with severe seizures, but experience progressive decline starting in late infancy. Currently, the mainstay treatments are anticonvulsants and rescue medications. GW Pharma seeks to change that through Epidiolex. They are designing trials to test seizure control efficacy with this drug in the target population.
Having both fast-track status and orphan-drug designation gives companies like GW Pharma numerous advantages. Fast-track status expedites the review of the drug and gives the company greater access to FDA, both in terms of more frequent meetings, as well as more correspondence and availability to answer questions. Orphan drug status provides other benefits, including tax credits on clinical research, technical assistance during submission, and seven years of marketing exclusivity. Having support for the development of a drug that affects less than 15,000 people worldwide is not only beneficial, but absolutely critical to move humanitarian drug development forward.