First and only FDA-approved CBD product approved for patients age 1 year and older

WASHINGTON — The FDA has expanded the indication for cannabidiol (CBD) oral solution (Epidiolex) to treat seizures in patients ages one year and older with tuberous sclerosis complex (TSC).

In addition to this new indication, the FDA widened the approved age range for the drug’s other indications — treatment of seizures associated with Dravet syndrome and Lennox-Gastaut syndrome — to treat patients one year of age and older. CBD oral solution is the only cannabis-derived drug currently approved by the FDA and the second FDA-approved treatment for seizures associated with TSC.

TSC is a rare genetic disease in which benign tumors grow in the brain and other parts of the body, such as the eyes, heart, kidneys, lungs, and skin. The disease, which impacts roughly 1 in 6,000 people, is responsible for a number of 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. This paradigm ensures new therapies are safe, effective, and manufactured to a high quality that provides uniform and reliable dosing for patients,” said Douglas Throckmorton, MD, deputy center director for regulatory programs in the FDA’s Center for Drug Evaluation and Research, in a statement. “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 interested in bringing patients safe and effective, high quality products.”

This latest approval was based on results from a randomized, double-blind, placebo-controlled trial with a cohort of 224 patients with TSC, 148 of whom received the CBD solution. “In the study, patients treated with [CBD oral solution] had a significantly greater reduction in the frequency of seizures during the treatment period than patients who received placebo. This effect was seen within eight weeks and remained consistent throughout the 16-week treatment period,” the FDA explained.

“Based on previous positive trial results in TSC patients, [CBD oral solution] may become an important treatment option for patients. It is a new tool in the toolbox for physicians and could meet a significant unmet need,” said Elizabeth Thiele, MD, PhD, Director of the Herscot Center for Tuberous Sclerosis Complex at Massachusetts General Hospital, Professor of Neurology at Harvard Medical School and clinical investigator, in a press release from GW Pharmaceuticals plc and its U.S. subsidiary, Greenwich Biosciences, Inc. “Nearly two-thirds of individuals with TSC develop treatment-resistant epilepsy and there is a need for new options that may benefit these patients who often try and fail existing treatments.”

The most common adverse effects associated with CBD oral solution in patients with TSC included diarrhea, elevated liver enzymes, decreased appetite, sleepiness, fever, and vomiting — the FDA noted that side effects across all indications included liver injury, decreased weight, anemia, and increased creatinine.

The FDA added that the drug must be dispensed with a patient Medication Guide outlining the drug’s uses and risks. “As is true for all drugs that currently treat epilepsy, including [CBD oral solution], the most serious risks may include an increase in suicidal thoughts and behavior, or thoughts of self-harm. Patients, their caregivers, and their families should be advised to monitor for any unusual changes in mood or behavior, such as worsening depression, suicidal thoughts or behavior. Patients, caregivers, and families should report behaviors of concern immediately to healthcare providers. [CBD oral solution] also caused liver injury in some patients. Most cases were generally mild, but a risk of rare, but more severe liver injury exists. More severe liver injury can cause nausea, vomiting, abdominal pain, fatigue, anorexia, jaundice, and/or dark urine.”

John McKenna, Associate Editor, BreakingMED™

Cat ID: 34

Topic ID: 82,34,730,34,192,725