Epidyolex is the first cannabis drug to receive green light from regulatory bodies in the United Kingdom and the European Union.
The first cannabis-based drug for childhood epilepsy is expected to be available soon in the UK and the rest of Europe after its UK manufacturer, GW Pharmaceuticals, receives green light from European authorities.
GW, based in Cambridge, has declared that the European Medicines Agency (EMA) and the European Commission had approved Epidyolex for seizures associated with two rare and severe forms of epilepsy, Lennox-Gastaut syndrome (LGS) and Dravet syndrome for patients aged two and over.
The authorization means that the drug, an oral solution of cannabidiol scented with strawberry of plant origin that is taken twice a day and without psychotropic effect, can be distributed throughout Europe. Up to 50000 children and young adults in Europe suffer from one of two syndromes.
GW's Chief Operating Officer, Chris Tovey, said the company was in talks with the UK's National Institute for Health and Care Excellence (NICE) about the availability of the drug. GW is in parallel discussion with other European countries. The drug is already on the market in the United States, where about 15 000 young people have been treated.
Tovey said, "The reactions [to the US] have been incredibly positive." He said he was very optimistic that NICE would agree to fund Epidyolex via the NHS. "We are hoping for a relatively quick decision from NICE and we hope to make it available to UK patients in the coming months."
A high cost drug
However, NICE had refused Satifx, GW's cannabis-based multiple sclerosis drug, because of its high price, much to the chagrin of patients. The cost of Epidyolex could be a complication as part of its reimbursement by the NHS. In the US, this treatment costs 35000 dollars a year.
GW Chief Executive Officer Justin Gover said: "The approval of Epidyolex marks a milestone, offering patients and their families the first of a new class of epilepsy drugs and the first and only EMA approved drug for the treatment of two serious and life-threatening forms of childhood epilepsy. "
Many patients with LGS or Dravet suffer from multiple seizures daily and do not respond to many of the other treatments available. Both syndromes have high mortality rates and many patients die before the age of 20 years.
A hope for patients and their families
Combined with other antiepileptic drugs, Epidyolex significantly reduced the frequency of seizures in patients with ILS and Dravet syndrome.
More than 150 patients have already been treated with this drug in the UK as part of a special program. "We know that families are desperate," said Tovey.
A change to the British law last year allowed for the legal prescription of medicinal cannabis. The change was made after the parents of Alfie Dingley and Billy Caldwell, who suffer from treatment-resistant epilepsy, have publicly demanded access to cannabis oil in the United Kingdom.
However, doctors are reluctant to prescribe cannabis-based drugs in the UK because of the lack of clinical evidence of their benefits and safety. This could change after the European approval of Epidyolex.
Isabella Brambilla, President of the European Federation of Dravet syndrome, said: "We are very pleased that patients now have access to a much needed new treatment option, and an option delivered through a rigorous and approved EMA-approved clinical trial program."
Elinor Ben-Menachem, professor of neurology and epilepsy at the Sahlgren Academy of Gothenburg University, said: "LGS and Dravet syndrome are two of the most serious and difficult to treat forms of the disease. epilepsy, and few patients manage to adequately control their seizures. EMA's approval of Epidyolex will bring hope to patients and their families, with the potential to better control seizures and improve quality of life. "
GW has also applied for registration in Switzerland and Israel. She is currently conducting other clinical trials on the treatment of other forms of epilepsy with this drug.