Jazz Pharmaceuticals subsidiary GW Pharmaceuticals has announced it has received approval in the UK for the use of Epidyolex to manage seizures associated with tuberous sclerosis complex (TSC).
Tuberous sclerosis complex is a rare genetic disease causing benign tumours to grow in the brain and on other vital organs. Among its many impacts where the condition is severe are seizures and impaired intellectual development. The seizures are often resistant to conventional therapies.
Usually diagnosed in early childhood, the number of people living with TSC in the UK is estimated between 3,700 and 11,000.
GW Pharmaceuticals’ cannabidiol-based Epidyolex (known as Epidiolex in the USA) is already in use in the UK for seizures associated with Lennox Gastaut syndrome (LGS) and Dravet syndrome. In September last year, the Australian Therapeutic Goods Administration (TGA) approved it here for the same indications.
The UK Medicines and Healthcare Products Regulatory Agency (MHRA) has now also approved it for TSC as an adjunct therapy to be used in conjunction with clobazam (a benzodiazepine class medication), for patients two years of age and older.
“The UK is a significant country for us for many reasons, and we are proud to be able to offer a medicine, that has been developed and is manufactured here, to even more patients across the UK,” said Jazz Pharmaceuticals’ Chris Tovey.
This development follows approval in all 27 European Union countries as well as Norway, Iceland, Liechtenstein and Northern Ireland.
As for its effectiveness, a Phase 3 safety and efficacy study evaluating cannabidiol indicated a seizure reduction of 49% in patients taking 25 mg/kg/day compared with 27% for placebo.
Epidyolex is crazy expensive stuff, but GW will now work with relevant stakeholders in the UK, including the National Institute for Health and Care Excellence (NICE), to secure reimbursement for eligible patients.
In the USA, Epidiolex was the first CBD medicine to be approved USA’s Food and Drug Administration (FDA), for seizures associated with Lennox-Gastaut syndrome and Dravet syndrome.
In May this year, Jazz completed the acquisition of GW Pharmaceuticals for a total value of approximately USD $7.2 billion , or $6.8 billion net of GW cash.