FDA approves cannabis product to treat seizures related to tuberous sclerosis complex
The FDA recently announced its approval of the cannabis-based Epidiolex, an oral solution to treat seizures in patients aged older than 1 year with tuberous sclerosis complex, a rare genetic disease that causes noncancerous tumors to grow in the brain and other parts of the body.
Epidiolex (cannabidiol, GW Pharmaceuticals), the only FDA-approved therapy that contains a purified drug substance derived from cannabis, was approved in 2018 for the treatment of seizures associated with Lennox-Gastaut syndrome and Dravet syndrome — both rare and severe forms of epilsepy, the agency said.
According to the FDA, the efficacy of cannabidiol was established in a 16-week, randomized, double-blind, placebo-controlled trial of 224 patients with tuberous sclerosis complex (TSC). In the study, 148 patients who received cannabidiol had a significantly greater reduction of TSC-related seizures vs. those who received placebo. The treatment effect was seen within 8 weeks and remained consistent throughout the trial. Common adverse events associated with cannabidiol use were diarrhea, elevated liver enzymes, decreased appetite, fever, sleepiness and vomiting, as well as anemia, decreased weight, increased creatinine and liver injury. The most serious risks may include an increase in suicidal thoughts and behavior, or thoughts of self-harm.
Patients who are prescribed cannabidiol must receive a medication guide with information about the drug’s uses and risks, the FDA said.
According to the Tuberous Sclerosis Alliance, TSC is the leading genetic cause of autism and epilepsy. TSC affects approximately 1 million people worldwide, including 50,000 in the United States.
“Up to 85% of those affected by TSC experience seizures at some point in their lifetime,” Tuberous Sclerosis Alliance President and CEO Kari Luther Rosbeck said in a press release. “Unfortunately, existing medications don't always effectively control them. Our organization and the TSC community certainly welcome a new option such as Epidiolex, which is the second FDA-approved drug specific to TSC.”