New marijuana-based pharmaceutical drug approved by FDA
What is Epidiolex?
Epidiolex is an oral liquid-based drug made from the cannabidiol (CBD) portion of cannabis. Compared to the tetrahydrocannabinol (THC) portion, CBD is not psychoactive, so taking this drug would not result in a high for the user.
CBD acts on different parts of the nervous system compared with THC. According to the Centers for Disease and Control (CDC), THC and CBD both may be useful for nausea caused by cancer treatments. Neither marijuana-derived substances, however, have been medically approved since they both fall under the Schedule 1 drug classification, which refers to substances that are usually illegal, often abused and have no proven medical benefit, according to the Drug Enforcement Administration.
Why was it approved?
The drug was approved by the FDA on Monday after a review of four large studies that looked at Epidiolex for the treatment of Lennox-Gastaut syndrome and Dravet syndrome in both adults and children.
The first major study looked at the effect of Epidiolex for Lennox-Gastaut syndrome was also published in The New England Journal of Medicine in May. In the study, the researchers enrolled 225 patients, ages 2 to 55 (all of whom had two or more seizures per day) to two different doses of Epidiolex or a placebo that contained no medicine. The trial lasted 14 weeks. The average seizure frequency dropped by 41.9 percent in the higher dose group and by 37.2 percent in the lower dose group and was down 17.2 percent in the placebo group (an inactive placebo often has a medicinal effect).
It is known as the potential for CBD in seizure treatment for years, and it is expected a response.
The second major study was published in The New England Journal of Medicine in May, and it looked at the effect of Epidiolex for Dravet syndrome. Researchers enrolled 120 children and young adults to compare Epidiolex to standard anti-epileptic treatment. After 14 weeks, the average seizure frequency dropped by at least half in 43 percent of people who took Epidiolex and 5 percent of the people taking the medication became seizure-free.
Both Dravet Syndrome and Lennox-Gastaut syndrome are diagnosed by a clinical exam and EEG by a doctor, ideally a doctor who specializes in brain conditions for children, known as a pediatric neurologist. Dravet Syndrome is confirmed with a genetic test.
What are the side effects of this medication?
In the trial, the main side effect was liver toxicity, which appeared as increased liver enzymes.
Others may have felt more sedated, nauseous or experienced diarrhea or vomiting, but these were not life-limiting symptoms.
There is some research that indicates that CBD specifically could interact with other medications such as statins, and increases the amount of those medications in the blood.
