Cannabidiol (Epidyolex) approved in Scotland for individuals living with Dravet Syndrome, a life-limiting epilepsy

7 September 2020. Dravet Syndrome UK welcomes today’s decision by the Scottish Medicines Consortium to make cannabidiol (Epidyolex) available via NHS Scotland for patients living with Dravet Syndrome, a rare and catastrophic form of epilepsy.

Dravet Syndrome affects around one in 15,000 people in the UK. It is characterised by treatment-resistant seizures (90% of individuals are resistant to current treatments), intellectual disabilities and a spectrum of other difficulties. Sadly, around 15% of individuals with the condition die before their 20th birthday due to uncontrolled or unexpected seizures.

Today’s approval means that Scottish families living with the Dravet Syndrome have the opportunity to try a much-needed new therapeutic option that may reduce the frequency and severity of seizures and, as a result, improve quality of life for all the family.

Jenny’s son, Jacob (aged 5), was one of the first to receive Epidyolex in England. “We have seen so many positives from it, including a reduction in seizures”, she says. “We’re so delighted that families in Scotland now have the chance to find out if Epidyolex will make a difference to them like it has for Jacob”.

Making the patient voice heard

As the only independent UK charity dedicated to improving the the lives of those affected by this catastrophic condition, Dravet Syndrome UK (DSUK) has been working with families and the Scottish Medicines Consortium (SMC) to make the patient voice heard during the approval process.

Galia Wilson, Chair and Trustee of DSUK, says: “SMC gave DSUK the opportunity to convey how much new medicines, such as cannabidiol, are desperately needed by families living with Dravet Syndrome. The constant worry of potentially fatal seizures is both terrifying and exhausting for parents/carers - their children need 24/7 monitoring. Even small improvements in seizure control can mean significant benefits in quality of life, both for patients and their families. 

We’re thrilled that SMC has recognised this and welcome their approach to giving patient groups a stronger voice in decision-making, especially around rare conditions such as Dravet Syndrome”.

Cannabis-based medicines 

This decision marks the first time that a plant-derived cannabis-based medicine has been approved in Scotland for use on the NHS. At the current time, cannabidiol is only approved for use in patients with Dravet Syndrome, who are aged 2 years or older and must always be prescribed in combination with another medicine, clobazam. 

Galia Wilson adds: “Many families come to us asking about the potential of cannabis-based medicines. Importantly, cannabidiol (also known as Epidyolex) has safety and efficacy data to support its use, and is approved by a medicines regulator.  

Epidyolex may not be suitable for all individuals with Dravet Syndrome, and responses to the treatment vary. That said, we’re delighted that patients in Scotland now have to opportunity to find out if Epidyolex makes a difference for them”.

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Notes for editors:

About Dravet Syndrome

Dravet Syndrome (DS) is a rare, life-long, life-limiting, catastrophic disorder that places a huge burden on children/adults with the condition, their parents/carers and the entire family.  

DS affects around one in every 15,000 people in the UK. As a spectrum disorder, DS is complex and unpredictable. The key features of DS are treatment-resistant seizures, intellectual disability, autism, behavourial problems, and difficulties with speech, mobility, feeding and sleep. In around 85 % of cases, Dravet Syndrome is caused by a mutation in a gene known as SCN1A.

Seizure-related premature mortality is a major issue in DS. Prolonged, recurrent seizures usually start in the first year of life. As the condition progresses, other seizure types occur. 15% of individuals with DS die of SUDEP (Sudden Unexpected Death in Epilepsy) or status epilepticus before their 20th birthday. Given the high seizure burden, high risk of mortality and associated comorbidities, many children/adults with DS require 24-hour supervision, and additional family support or home care is likely to be required.

This combination of treatment-resistant seizures, debilitating comorbidities and the requirement for 24-hour monitoring causes DS to have a catastrophic impact, not only on health-related quality of life but overall quality of life - for the person with the condition, their parents/carers and the entire family. 

See https://www.dravet.org.uk/about-dravet-syndrome/ for more information.

About Dravet Syndrome UK

Dravet Syndrome UK (DSUK) is an independent charity dedicated to improving the lives of those affected by Dravet Syndrome through support, education and medical research. We do this by:

• Supporting families affected by Dravet Syndrome emotionally, practically and financially. 
• Raising awareness and understanding of Dravet Syndrome among medical professionals
• Funding medical research to increase understanding of Dravet Syndrome, improve its management, work towards better outcomes and to hopefully one day find a cure

See www.dravet.org.uk for more information. 

About Epidyolex (cannabidiol)

Epidyolex is made from a natural molecule called cannabidiol (CBD), found in the cannabis plant. While Epidyolex is made from a plant it has been through the same rigorous testing and quality standards process as all other licensed medicines and as a result is licensed and regulatory-approved.

Epidyolex is used in combination with clobazam and other antiepileptic medicines to treat seizures that occur with two rare conditions, called Dravet syndrome and Lennox-Gastaut syndrome.  It can be used in adults, adolescents and children of at least 2 years of age.

Contact / Interviews:

If you would like further information or to arrange interviews please contact info@dravet.org.uk.