Epidyolex gains approval for seizures associated with tuberous sclerosis complex

Epidyolex, the CBD-based medicine, has been approved for use in the UK as a treatment for seizures associated with tuberous sclerosis complex (TSC).

Approval from the Medicines and Healthcare products Regulatory Agency (MHRA) means Epidyolex can now be used as an adjunctive treatment of seizures associated with TSC for patients two years of age and older.

Tuberous Sclerosis Complex (TSC) is a rare genetic condition. It is estimated that there are between 3,700 and 11,000 patients in the UK living with TSC. TSC causes non-cancerous growths to develop around the body, such as the brain, eyes, heart and skin. Problems caused by TSC are a result of the growths in different organs.

TSC can cause epilepsy in up to 85 percent of those with the condition and up to 60 percent do not respond to standard anti-seizure medicines.

Epidyolex is also used in addition to clobazam, to treat patients from age two with Lennox-Gastaut syndrome or Dravet syndrome. The drug was developed by UK firm GW Pharmaceuticals, which was acquired by Dublin-based Jazz Pharmaceuticals in May.

Chris Tovey, executive vice president, chief operating officer and MD, Europe & International, at Jazz Pharmaceuticals said:

“We are delighted by today’s decision from the MHRA for this new indication, which represents a significant step forward for TSC patients in the UK, many of whom may benefit from this new treatment.

Read more: CBD may reduce side effects of anti-seizure medications

Welcome news for patients

“The UK is a significant country for us for many reasons, and we are proud to be able to offer medicine, that has been developed and is manufactured here, to even more patients across the UK.

He added: “This authorisation, expanding the label, is further proof of our continued commitment to the UK and the pioneering research and development into regulatory approved cannabis-based medicines we have conducted here.”

Tuberous Sclerosis Association (TSA) chief executive Louise Fish said: “One in every two people living with TSC-related epileptic seizures has difficult to treat epilepsy that does not respond to traditional anti-epileptic drugs.

“We are excited to see this new medicine approved by the MHRA, which gives people living with TSC and their families hope.
“We desperately need further options for clinicians who are managing seizures associated with TSC, and we, therefore, welcome this decision by the MHRA.”

Studies

The approval is based on data from a positive phase three safety and efficacy study which evaluated 25 mg/kg/day of GW’s cannabidiol. The study met its primary goal which was the reduction in seizure frequency when comparing cannabidiol and a placebo.

It recorded a seizure reduction of 49 percent in patients taking cannabidiol 25 mg/kg/day compared with 27 percent for placebo. The safety profile was consistent with findings from other studies with no new risks identified.

Read more: How medical cannabis can improve migraine symptoms.