Advisors to the Food and Drug Administration have found a generally favorable benefit-risk profile for cannabidiol oil solution in the treatment of two forms of severe pediatric seizure disorders. The drug is under expedited review by the agency.
If the FDA supports this recommendation, cannabidiol oral solution would be the first cannabis-based medication approved in the United States.
“At this point, this is a spectacular advance,” said committee member John Mendelson, MD, chief medical officer of Ria Health, San Francisco.
Fellow committee member Mark Green, MD, concurred. “It is clearly an honor to be making a decision based on science and public interest, rather than political discussion,” said Dr. Green, professor of neurology, anesthesiology, and rehabilitation medicine at Icahn School of Medicine, Mt Sinai, New York.
Patients with LGS taking cannabidiol oral solution at 20 mg/kg/day in two clinical trials saw a 42%-44% reduction in drop seizure frequency over a 14-week treatment period (P = .0047 and P = .0135, compared to placebo). A 50% reduction of drop seizure frequency was seen in 40% and 44% of LGS patients in the two clinical trials.
For patients with DS, 20 mg/kg/day of cannabidiol oral solution resulted in a 39% decrease in convulsive seizure frequency during a 14-week treatment period (P less than .05). There was a numeric, but not statistically significant, increase in the number of DS patients who saw a 50% reduction in convulsive seizure frequency on this dose.
Sustained efficacy for both seizure disorders has been seen during an extended open-label follow-on study.