Cannabidiol for Treatment of TSC Conditions Approved

“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.” Louise Fish,

GW Pharmaceuticals has received approval from the UK Medicines and Healthcare Products Regulatory Agency (MHRA) for a new indication of its EPIDYOLEX® cannabidiol as an adjunctive treatment of seizures associated with tuberous sclerosis complex (TSC), for patients two years of age and older. The condition causes mostly benign tumours to grow in vital organs of the body, including the brain, skin, heart, eyes, kidneys and lungs, and in which epilepsy is the most common neurological feature.

This is the fourth approved indication of this cannabis-based medicine in the UK for the company, which is now part of Jazz Pharmaceuticals plc. In 2019 it received marketing authorisation for use associated with Lennox Gastaut syndrome (LGS) or Dravet syndrome, in conjunction with clobazam, for patients two years of age and older; and was also recommended by the UK’s National Institute for Health and Care Excellence (NICE) to receive routine reimbursement from NHS England.

Step forward for TSC

“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,” said Chris Tovey, Executive Vice President, Chief Operating Officer and Managing Director, Europe & International at Jazz Pharmaceuticals. “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. 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.”

 Commenting on the MHRA approval of GW’s cannabidiol, 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.”

 The approval was made through the European Commission Decision Reliance Procedure (ECDRP) and is based on data from a positive Phase 3 safety and efficacy study evaluating 25 mg/kg/day of the medicine. It is now available in England, Scotland and Wales following all 27 countries of the European Union, alongside Norway, Iceland and Liechtenstein (including Northern Ireland).

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