The FDA approved the CBD solution following the results of a randomized, double-blind, placebo-controlled trial.
The US Food and Drug Administration (FDA) has approved Epidiolex cannabidiol (CBD) oral solution to treat seizures associated with tuberous sclerosis complex in patients at least 1 year old.
The CBD solution was previously approved to treat seizures associated with 2 rare and severe forms of epilepsy, Lennox-Gastaut syndrome and Dravet syndrome. Now, the oral solution can be used for tuberous sclerosis complex, a rare genetic disease that causes benign tumors to grow in the brain and other parts of the body. The condition affects the central nervous system and may results. In symptoms including seizures, developmental delay, and behavioral problems.
“The FDA continues to believe the drug approval process represents the best way to make new medicines, including any drugs derived from cannabis, available to patients in need of appropriate medical therapy such as the treatment of seizures associated with these rare conditions,” Douglas Throckmorton, MD, deputy center director for regulatory programs in the FDA’s Center for Drug Evaluation and Research, said in a statement. “This paradigm ensures new therapies are safe, effective, and manufactured to a high quality that provides uniform and reliable dosing for patients.”
FDA’s approval of Epidiolex came following the results of a randomized, double-blind, placebo-controlled trial of 224 patients. Of those included, 148 patients received Epidiolex. Investigators measured the change from baseline in seizure frequency. Patients treated with Epidiolex had a significantly greater reduction in the frequency of seizures during the treatment period than patients who received placebo. The benefits were seen within 8 weeks and remained consistent during the 16-week study period.
Common side effects included diarrhea, elevated liver enzymes, decreased appetite, sleepiness, fever, and vomiting. A patient Medication Guide will be dispensed with Epidiolex to describe important information about the drug’s uses and risks. Like other drugs used to treat epilepsy, the most serious risks may include an increase in suicidal thoughts and behavior or thoughts of self-harm.
“The agency is committed to supporting rigorous scientific research on the potential medical uses of cannabis-derived products and working with product developers who are interesting in bringing patients safe and effective, high-quality products,” Throckmorton said.