FDA Approves Epidiolex to Treat Tuberous Sclerosis Complex

The Food and Drug Administration has expanded the label for the cannabis-derived pharmaceutical Epidiolex to treat seizures associated with tuberous sclerosis complex (TCS) and allowing the drug to be used by patients one year of age and older.

The GW Pharmaceuticals drug is the only FDA-approved form of CBD derived from the cannabis plant. The medication was initially approved by the agency in 2018 for treating seizures associated with Dravet syndrome – or Lennox-Gastaut syndrome – in patients aged two and older.

GW CEO Justin Gover said the label expansion allows patients with TCS to “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.” – Gover in a press release

Dr. Elizabeth Thiele, director of the Herscot Center for Tuberous Sclerosis Complex at Massachusetts General Hospital, said that nearly two-thirds of TSC patients develop treatment-resistant epilepsy and noted “a need for new options that may benefit these patients who often try and fail existing treatments.”

“Based on previous positive trial results in TSC patients, Epidiolex may become an important treatment option for patients,” she said in a statement. “It is a new tool in the toolbox for physicians and could meet a significant unmet need.”

In the U.S. about 50,000 people suffer from TSC along with approximately 1 million worldwide. About 85 percent of patients experience epilepsy and more than 60 percent do not achieve seizure control.

The drug is also approved in the European Union under the name Epidyolex. Regulators in Europe are currently reviewing the application to expand the label for TSC and lowered age range for patients.