First Cannabis-based Drug approved by the FDA
For the first time, the US Food and Drug Administration approved a cannabis-based drug. This past April Epidiolex was recommended for approval by an advisory committee. The deadline for approval or denial was this week, and on Monday the announcement was made.
The twice-daily oral solution is approved for use in patients 2 and older. Epidiolex is approved to treat two types of epileptic syndromes:
- Lennox-Gastaut Syndrome is a form of epilepsy with multiple types of seizures that begin in early childhood, usually between the ages of 3 and 5. It is also linked to learning problems, intellectual disabilities, and compromised motor skills.
- Dravet Syndrome is a rare genetic dysfunction of the brain that begins in the first year of life. It’s associated with fever-related seizures, poor language development and motor skills, hyperactivity, and trouble relating to others. It has a high mortality rate with patients sometimes dying before the age of 10.
“Because of the adequate and well-controlled clinical studies that supported this approval, prescribers can have confidence in the drug’s uniform strength and consistent delivery,” FDA Commissioner Dr. Scott Gottlieb said in a statement Monday. “This is an important medical advance.” The approval was based on the results of three Phase III clinical trials published in The New England Journal of Medicine and the Lancet.
Epidiolex is the “first pharmaceutical formulation of highly-purified, plant-based cannabidiol (CBD), and the first in a new category of anti-epileptic drugs,” according to a statement from GW Pharmaceuticals, the UK-based biopharmaceutical company responsible for the breakthrough.
Cannabidiol is one of more than 80 active cannabinoid chemicals, and unlike tetrahydrocannabinol, or THC, it does not produce a high. The FDA has approved synthetic versions of some cannabinoid chemicals found in the marijuana plant for other purposes, including cancer pain relief, however this is the first and only FDA-approved cannabidiol medicine to treat two severe childhood epilepsies. Justin Gover, chief executive officer of GW Pharmaceuticals described the approval in a simple statement, “it is a historic milestone and validation of the science of cannabinoid medication.” Epidiolex will become available in the Fall, however cost information has not yet been made available and will be announced at a later date.
