JAZZ Pharmaceuticals plc (Nasdaq: JAZZ) today announced that Epidyolex® (cannabidiol,100 mg/mL oral solution), developed by GW Pharmaceuticals (“GW”; now part of Jazz Pharmaceuticals plc), a world leader in discovering, developing and delivering regulatory approved cannabinoid-based medicines, has been recommended for reimbursement by National Institute for Health and Care Excellence (NICE) for adjunctive therapy of seizures associated with tuberous sclerosis complex (TSC), for patients two years of age and older.
Following earlier decisions by the Scottish Medicines Consortium, Northern Ireland’s Strategic Planning and Performance Group and the All Wales Medicines Strategy Group this recommendation now ensures all eligible UK patients who may benefit from this medicine will be able to receive reimbursed access to Epidyolex® from 1 March 2023.
TSC is a condition that 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 one of the most common neurological features – and can severely impact the lives of these patients. TSC can be diagnosed in infancy but many children are not diagnosed until later in childhood when their seizures begin and other symptoms appear. It is estimated that between 3,700 and 11,000 people in the UK live with TSC.
Dr. Pooja Takhar, Joint Chief Executive at Tuberous Sclerosis Association (TSA), said “The TSA is delighted with this decision, along with people living with TSC and their families. TSC is a very difficult to manage condition, with common issues including epilepsy in eight out of ten people. Up to half of the people with TSC-related epilepsy are unable to manage their seizures with standard anti-seizure medication, leading to a massive unmet need for new treatment options. This underlines why we are so pleased that this medicine will now be available on the NHS in England, improving lives in the TSC community.”
“We welcome NICE’s recommendation which provides appropriate patients across the UK, who are living with TSC, a difficult to treat condition, access to a new treatment option. This is an important milestone not only for those living with TSC but also for their families, carers and clinicians,” said Simon Newton, General Manager at Jazz Pharmaceuticals. “This demonstrates the importance of randomised clinical trials and regulatory approval in providing reimbursed access to cannabinoid-based medicines to patients who may benefit.”
Jazz’s cannabinoid-based medicine has been approved for use in the UK in three conditions. In July 2019, it was approved by the European Medicines Agency (EMA) and received marketing authorisation under the trade name Epidyolex as an adjunctive therapy for seizures associated with Lennox Gastaut syndrome or Dravet syndrome, in conjunction with clobazam, for patients two years of age and older. In August 2021, it was approved by the UK Medicines and Healthcare Products Regulatory Agency (MHRA) for use as an adjunctive treatment of seizures associated with TSC, for patients two years of age and older.