BRIEF—FDA approves Epidiolex for rare genetic disease

The US Food and Drug Administration on Friday approved Epidiolex (cannabidiol) [CBD] oral solution for the treatment of seizures associated with tuberous sclerosis complex (TSC) in patients one year of age and older.

Manufactured by UK company GW Pharmaceuticals and distributed in the USA by its subsidiary Greenwich Biosciences, Epidiolex is a plant-derived cannabinoid prescription medicine and the only current FDA-approved form of cannabidiol (CBD).

In 2018, the FDA approved the drug to treat people two years of age and older with Lennox-Gastaut syndrome (LGS) or Dravet syndrome. For full-year 2019, Epidiolex generated sales of $296.4 million.

TSC is a rare genetic disease that causes non-cancerous (benign) tumors to grow in the brain and other parts of the body like the eyes, heart, kidneys, lungs, and skin. TSC usually affects the central nervous system and can result in a combination of symptoms including seizures, developmental delay, and behavioral problems, although the signs and symptoms of the condition, as well as the severity of symptoms, vary widely. TSC affects about 1 in 6,000 people.