In late 2017, when the US Food and Drug Administration (FDA) accepted for review the New Drug Application (NDA) for an investigational cannabidiol (CBD) for the adjunctive treatment of Lennox-Gastaut syndrome (LGS) and Dravet syndrome (DS), it marked a significant milestone for Greenwich Biosciences.
The NDA submission and ongoing FDA review represented the culmination of 10 years of extensive preclinical and clinical research studying CBD, a cannabis plant component that lacks euphoric properties. This research includes three Phase 3 clinical trials involving approximately 500 patients suffering from LGS and DS, 2 rare, severe and difficult-to-treat childhood-onset epilepsies.
Clinical Program Takes Shape
The launching point to these patient trials was an FDA-authorized Expanded Access Program (EAP), initiated as a series of individual investigator-sponsored requests and started in 2013. The program grew to over 1,100 children and adults suffering the most difficult-to-treat forms of epilepsy who had exhausted all other treatment options and had no therapeutic alternatives. This made it the largest EAP, or Compassionate Use Program, of its kind.
“Our clinical program took shape from the EAP,” notes Kathryn Nichol, Senior Director, Medical Affairs at Greenwich Biosciences. “On the basis of the preclinical data in animal models of seizures and the initial EAP data, we worked closely with the FDA and moved quickly to initiate the largest randomized controlled clinical studies to date in LGS and Dravet.”
Results of the company’s groundbreaking study in Dravet syndrome were published in the New England Journal of Medicine in May 2017. The results of a controlled study in LGS were published in the Lancet in January 2018 and the New England Journal of Medicine in May 2018.
Behind the Data: An Enduring Commitment to
The company’s clinical trials on the adjunctive use of CBD to treat some of the most severe and difficult-to-treat forms of rare, childhood-onset epilepsy are built upon its foundation of pioneering leadership in cannabinoid science. That legacy spans nearly 2 decades of scientific research covering the evaluation of 14 cannabinoids in preclinical studies that have been detailed in over 80 independent peer-reviewed publications.
“Our footprint in cannabinoid science is part of the fiber of our company,” says Nichol. “We have an enduring commitment to studying cannabinoids from the ground up, including all the unique cannabinoids the plant contains and how they interact with different systems of the body. This has enabled us to better understand where a singular cannabinoid might have a therapeutic application, and, in other disease states, where a combination of cannabinoids might have therapeutic benefit.”
She added, “We have built upon the science underlying our extensive cannabinoid platform to study CBD and have worked tirelessly to bring forward a consistent and well-characterized pharmaceutical formulation for FDA review.”
Bringing Hope to Patients
Nichol is deeply touched by the patients she meets and is inspired to work on the potential treatment options that will address their significant unmet needs.
“With treatment-resistant epilepsy, it is such an arduous journey for patients, as well as their families and caregivers, to try to achieve seizure control,” says Nichol. “Most patients have tried a number of medications, dietary therapies, or surgery to achieve seizure control.”
Nichol continued, “I was struck by a recent survey from the Dravet Syndrome Foundation, showing that roughly 80 percent of caregivers sleep with their children out of fear the child will have a seizure and they will not be there to help them. This is a patient community very much in need of new ways to gain seizure control for those suffering.”
“I am very proud of the fact that our clinical and scientific research has yielded important data on the efficacy and safety profile of CBD and offers patients hope for a potential new treatment option.”