Epidiolex Receives New Indication for Rare Genetic Disorder

July 31, 2020 – The U.S. FDA has approved a new indication for Epidiolex® (cannabidiol) oral solution, manufactured by GW Pharmaceuticals. The drug is now indicated to treat seizures associated with tuberous sclerosis complex (TSC) in patients who are at least one year old.

TSC is a rare, inherited genetic disorder that is also known as Bourneville syndrome. It leads to the growth of benign tumors throughout the body, including in the brain, heart, kidneys, and lungs. TSC can cause various problems such as seizures, developmental delays, skin changes, neurological issues, behavioral issues, and intellectual disability.

In a clinical study, patients who received Epidiolex had a significantly greater reduction from baseline in seizure frequency compared to those who received a placebo. The effect was seen within eight weeks of starting treatment and remained consistent throughout the 16-week treatment period of the study.

Recommended initial dosing under the new indication is 2.5mg/kg of body weight taken twice daily by mouth. The dose should be increased on a weekly basis by 2.5mg/kg twice daily, as tolerated, to a recommended maintenance dose of 12.5mg/kg twice daily. Dose modification is recommended for patients who have moderate to severe hepatic impairment.

Epidiolex first received FDA approval in 2018 and is also indicated to treat seizures associated with Lennox-Gastaut syndrome or Dravet syndrome.