Epidyolex is the first cannabis-based drug to receive the green light from regulatory agencies in the UK and EU.
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 the 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 years and over.
The authorization means that the drug, a strawberry-flavored oral cannabidiol solution of plant origin which is taken twice a day and without psychotropic effects, can be distributed throughout Europe. Up to 50000 children and young adults in Europe suffer from one of the two syndromes.
GW COO Chris Tovey said the company was in talks with the UK's National Institute for Health and Care Excellence (NICE) about making the drug available. GW is in parallel discussions with other European countries. The drug is already on the market in the United States, where around 15 young people have been treated.
Tovey said, “The response [in the United States] has been overwhelmingly positive. He said he was very optimistic that NICE would agree to fund Epidyolex through the NHS (UK Social Security). “We hope for a relatively quick decision from NICE and hope to make it available to UK patients in the coming months. "
A high cost drug
However, NICE had refused Sativex, GW's cannabis-based multiple sclerosis drug, due to its high price, much to the chagrin of patients. The cost of Epidyolex could be a complication as it gets reimbursed by the NHS. In the United States, this treatment costs $ 35000 per year.
Justin Gover, CEO of GW, said: “Approval of Epidyolex marks an important milestone, providing patients and their families with the first in a new class of epilepsy drugs and the first and only drug approved by the EMA for the treatment of two serious and potentially fatal forms of childhood epilepsy. "
Many patients with LGS or Dravet experience multiple seizures on a daily basis and do not respond to many of the other treatments available. Both syndromes have high death rates, and many patients die before the age of 20.
A hope for patients and their families
Combined with other anti-epileptic drugs, Epidyolex significantly reduced the frequency of seizures in patients with GAS and Dravet syndrome.
Over 150 patients have already been treated with the drug in the UK under a special program. “We know families are desperate,” Tovey said.
A change in UK law last year made it possible to legally prescribe 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 from authorities in the UK.
However, doctors are reluctant to prescribe cannabis-based medicines in the UK, due to the lack of clinical evidence of their benefits and safety. This could change after 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 one channeled through a rigorous, EMA-authorized clinical trial program."
Elinor Ben-Menachem, professor of neurology and epilepsy at the Sahlgren Academy, University of Gothenburg, said: “LGS and Dravet syndrome are two of the most serious and difficult to treat forms of the disease. childhood epilepsy, and few patients achieve adequate seizure control. The EMA 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 approval in Switzerland and Israel. She is currently conducting further clinical trials to treat other forms of epilepsy with this drug.