- London-based GW Pharmaceuticals, a rare diseases company with a focus on cannabinoids, has published Phase 3 data for its drug Epidiolex in the New England Journal of Medicine. The study involved treatment-resistant children with the rare childhood epilepsy Dravet syndrome being treated with Epidiolex, a liquid formulation of purified, plant-derived cannabidiol (CBD).
- Adding Epidiolex to anti-epilepsy drugs reduced the monthly convulsive seizure frequency by 39%, compared with 13% for placebo. More than three of every five patients treated with Epidiolex showed an improvement on the Caregiver Global Impression of Change scale, compared with 34% for placebo.
- The biotech plans to file for U.S. approval of the drug in mid-2017 for both Dravet syndrome and Lennox-Gastaut syndrome, another rare and difficult-to-treat childhood epilepsy, with a European submission to follow.
From an area initially treated with some level of suspicion, cannabinoid drug development is buzzing these days, and is picking up the confidence of the investors, especially as a growing number of U.S. states are legalizing the possession of marijuana. GW Pharmaceuticals has been working in this field since the late 1990s, with a multiple sclerosis spasticity drug, Sativex, approved in 31 countries outside the U.S.
In latter years the company has switched its focus to rare diseases, and Epidiolex is in Phase 3 for Dravet syndrome, Lennox-Gastaut syndrome, tuberous sclerosis and infantile spasms. Data released for Epidiolex in September 2016 in a Phase 3 trial in Lennox-Gastaut syndrome showed a 42% reduction in the frequency of seizures. This followed similarly positive results in LGS released in June 2016.
The New England Journal of Medicine publication is the first release of the full Phase 3 data for Epidiolex in Dravet syndrome, following topline results in March 2016, and brings the company a step closer to submission for approval in the U.S. for the two indications.
Dravet syndrome begins in babies under a year old. Even with treatment these infants and children may have hundreds of seizures per month, leading to intellectual and development disabilities, and even death. There is no approved treatment.
"The publication of these highly-anticipated positive results represents an important milestone for the Dravet syndrome community in that it provides hope that a new treatment option is within sight for this rare and devastating disease," said Nicole Villas, scientific director of the Dravet Syndrome Foundation.