FDA Approves Epidiolex For Tuberous Sclerosis Complex Seizures

The cannabis-focused Cambridge-based GW Pharmaceuticals (and its U.S. subsidiary Greenwich Biosciences, Inc.) has received FDA approval to treat Tuberous Sclerosis Complex seizures with Epidiolex. The approval applies to TSC patients one year of age and older and also extends the age range of Lennox-Gastaut syndrome and Dravet syndrome patients to one year of age and older.

“Since Epidiolex is already available to patients by physician’s prescription, patients with TSC can immediately access the medication. This label expansion, including the expansion of the age range in all approved indications, further demonstrates that the FDA process can continue to enable broader patient access to appropriately tested regulatory approved cannabinoid medicines,” says GW Pharmaceuticals CEO, Justin Gover. “It also provides hope for these patients and their families and is yet another important milestone for Epidiolex as a first-in-class antiepileptic drug.”

“It is a new tool in the toolbox for physicians and could meet a significant unmet need,” says Elizabeth Thiele, M.D., Ph.D., Director of the Herscot Center for Tuberous Sclerosis Complex at Massachusetts General Hospital. “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 GW Pharmaceuticals study was a 16-week, randomized, double-blind, placebo-controlled trial of 224 patients with tuberous sclerosis complex. In the study, of the 148 patients who received cannabidiol 48 percent of patients who took the recommended Epidiolex dose showed a seizure reduction, compared to 24 percent with a placebo. The treatment effect was seen within 8 weeks and remained consistent throughout the trial.

“One of the most challenging and frustrating aspects of TSC are seizures that cannot be effectively controlled by existing medications,” says Kari Luther Rosbeck, Tuberous Sclerosis Alliance President and CEO. “New treatment options are desperately needed, and this approval adds another option for those impacted by this difficult disease.” 

Tuberous Sclerosis Complex is a disease that manifests as tumors in vital organs such as the brain, heart, lungs, kidneys, skin, and eyes. It is one of the most common causes of epilepsy. TSC affects nearly one million worldwide and approximately 50,000 people in the United States.