Long-Term Data for Cannabidiol in Lennox-Gastaut Syndrome
Expert perspective on the open-label extension trial that analyzed long-term safety and efficacy with cannabidiol in patients with Lennox-Gastaut syndrome.
Anup Patel, MD
PUBLISHED June 28, 2020
Anup Patel, MD: We were going to present our long-term data of the open-label study for patients with Lennox-Gastaut syndrome at the annual American Academy of Neurology meeting, and unfortunately, we couldn’t do that in person. But it’s still very important information that we have to share, as it relates to the open-label study.
We now have data up to 170 weeks for patients who were in the open-label trial. These are patients who have Lennox-Gastaut syndrome. They were participating in the randomized, double-blind, placebo-controlled trial and then went into the open-label study. We’ve been able to follow them, both from an efficacy and safety standpoint. As far as results go, we didn’t learn about any new adverse events or effects that were seen in these patients, and the efficacy—as it relates to both the primary and secondary end points of drop atonic seizures and all seizures—did not decrease. It was maintained at the same high level that was seen during the pivotal trials all the way up through 170 weeks. The important take-home messages for folks are that efficacy can remain for patients on Epidiolex, or cannabidiol, for Lennox-Gastaut syndrome, and the adverse effect profiles are very similar, without any new ones cropping up later in treatment.
The important thing that the data tell us about safety and efficacy is efficacy is maintained throughout the course of being on cannabidiol and the adverse effect profile was also very similar to what was seen in the pivotal trials. Most patients will have some sort of adverse event, which is seen in all epilepsy medication trials. The more common ones, as they relate to this medicine, are the same, with diarrhea being number 1. That was seen in over 20% of the patients. As far as efficacy goes, specifically for primary end points, we were still able to attain a decrease in median seizures in the 40% to 60% range, which is very significant.