FDA approves Epidiolex for Tuberous Sclerosis Complex

The FDA has approved Epidiolex for the treatment of seizures associated with tuberous sclerosis complex (TSC) in patients aged one year and above

The rare disease is a leading cause of genetic epilepsy, affecting around 1 in 6,000 people.

The condition causes tumours to grow in the brain and other parts of the body including the eyes, heart and lungs.

Justin Gover, CEO of GW Pharmaceuticals, said:

“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.”

The CBD medicine’s efficacy in treating TSC seizures was demonstrated in a randomised, double-blind, placebo-controlled trial, with 148 out of 224 participants treated with the drug.

Seizure frequently was reduced significantly more for the Epidiolex patients over the 16-week treatment period than in the placebo group.

The most common side effects experienced by those in this group were diarrhoea, fever, vomiting, decreased appetite, sleepiness and elevated liver enzymes.

The approval was welcomed by Tuberous Sclerosis Alliance president and CEO Kari Luther Rosbeck , who called it a ‘tremendous step forward.’

Rosbeck said:

“[O]ur community applauds this positive development.

“One of the most challenging and frustrating aspects of TSC are seizures that cannot be effectively controlled by existing medications.

“New treatment options are desperately needed, and this approval adds another option for those impacted by this difficult disease.”

Epidiolex (marketed as Epidyolex in the UK) was approved by the FDA for the treatment of Lennox-Gastaut syndrome and Dravet syndrome in 2018.