A medicinal cannabis product that can treat two rare but severe forms of childhood epilepsy has been approved by European regulators.
Epidyolex can now be given out by doctors in the UK and other European countries, if they believe it will help their patients.
But the NHS is not recommended to use the product, an oral solution of cannabidiol (CBD), due to concerns over its long-term effectiveness and cost.
Epidyolex does not contain tetrahydrocannabinol (THC), a component of cannabis which causes a ‘high’.
It has been proven successful in controlling seizures in children with two forms of epilepsy that are resistant to most treatment – Dravet and Lennox-Gastaut syndromes.
Health watchdogs today have ruled against approving a CBD-based drug, Epidyolex, for children with rare forms of epilepsy on the NHS
Ley Sander, medical director at Epilepsy Society and professor of neurology at University College London, said: ‘These are both severe childhood epilepsies which can be very debilitating.
‘This new drug will bring hope for some families and European approval feels like a positive step.
‘There is evidence to show that pharmaceutical grade CBD, under the trade name Epidyolex, is effective in reducing seizures in some children with Dravet syndrome and Lennox-Gastaut.’
WHAT CAN EPIDYOLEX TREAT?
Epidyolex is a treatment for children with Lennox-Gastaut syndrome (LGS) or Dravet syndrome, from the age of two.
Dravet syndrome is a severe form of epilepsy that usually begins within the first year of life, affecting around 600 people in England.
Around one in five people with Dravet syndrome will die because of their condition, the majority before 10 years of age.
Lennox-Gastaut syndrome is another severely debilitating form of epilepsy diagnosed in childhood, affecting up to 4,000 people in England.
Around five per cent of people with Lennox-Gastaut syndrome will die prematurely because of their condition.
The National Institute for Health and Care Excellence (NICE) has not approved Epidyolex for the NHS in draft guidance.
It said last month there was not enough evidence Epidyolex was a cost-effective or that it worked long term.
Epidyolex is produced by GW Pharma, one of the largest companies in the cannabidiol pharmaceutical market.
Some GPs have already been prescribing it on compassionate grounds, funded by GW Pharma.
Mr Sander said: ‘It is important that the pharmaceutical industry continues to work with the medical advisory body to ensure that drugs are cost-effective and that its long-term effects are clear.
‘It is important that the medical profession, regulatory and advisory bodies remain level-headed in ensuring that the medications prescribed to people in the UK are rigorously tested and economically viable.
‘If a drug holds promise in treating a condition, it is important that it is available for all those who require it and that it does not become another victim of a postcode lottery.’
GW Pharma said the next step will be to secure a recommendation from NICE.
Justin Gover, chief executive of GW, said: ‘The approval of Epidyolex marks a significant milestone, offering patients and their families the first in a new class of epilepsy medicines.
‘We believe patients and physicians deserve access to rigorously-tested and evaluated cannabis-based medicines, manufactured to the highest standards and approved by medicines regulators, and we are delighted to be the first to offer this solution to the epilepsy community.’
Some parents of ill children say THC is the component in cannabis that helps their children’s condition the most.
Charlotte Caldwell, mother of Billy Caldwell, said medicinal cannabis containing THC gave her epileptic son the ‘right to life’.
She had seven bottles of medicinal cannabis confiscated at Heathrow Airport customs on June 11 2018, causing a row over the medicinal status of the oil.
The Home Office granted Ms Caldwell an emergency licence for the product that was calming Billy’s seizures.
Following this, a landmark law change gave specialist NHS doctors permission to legally prescribe medicinal cannabis from November 1.
Mr Sander said: ‘Medicinal cannabis, however, still remains a medical minefield and there are many hurdles ahead.’
Only two patients – both of whom are children – are believed to have been given a prescription on the NHS.
NICE last month prompted outrage by deciding to rule against prescribing the drug for some conditions.
In draft guidelines, NICE said drugs containing THC – the psychoactive compound – should not be given to patients with multiple sclerosis or chronic pain.
And it couldn’t decide whether or not to approve it for children with rare forms of epilepsy because of ‘a lack of evidence’.
WHAT IS EPIDYOLEX?
Epidyolex is a pharmaceutical liquid form of cannabidiol, an active ingredient in marijuana.
Cannabis contains around 400 different chemicals. The one most people know is tetrahydrocannabinol, or THC, which produces the characteristic ‘high’ from the drug.
CBD does not have any psychoactive effects.
In epilepsy it is thought to work by blocking the abnormal electric signals that can trigger seizures.
Epidyolex is already being prescribed to NHS patients as an unlicensed medicine, funded on compassionate grounds by the manufacturer GW Pharma.
CBD-based products are available to buy over the counter in the UK.
Unless licensed as a medicine, companies aren’t allowed to make explicit health claims.
The UK government’s Medicines and Healthcare products Regulatory Agency monitors unauthorised health claims for CBD.
To make health claims, manufacturers have to get products licensed through the European Medicines Agency, which Epidyolex is in the process of doing.
Epidyolex has been found to reduce seizures.
For example, in 2015, a study presented at the American Epilepsy Society’s annual meeting showed patients with epilepsy had their seizures reduced by a median of 45 per cent after three months of Epidyolex treatment with the regular drug regimen.
The New York University Langone Medical Center study included 261 participants with an average age of 11, and nine per cent of patients reported being seizure-free after treatment.
Another study led by Dr Michael Oldham, formerly of the University of California, explored the long-term efficacy of Epidyolex.
He followed a subset of 25 children – with an average age of nine. The cannabidiol and usual drug regimen resulted in a 50 per cent reduction in seizures for 10 participants.
However, Epidyolex can have serious side effects and doesn’t work for everybody.