RARE Daily

NICE Recommends GW Pharmaceuticals’ Epidyolex for Rare Epileptic Seizures

November 10, 2019

Photo: Marijuana plant

The UK’s National Institute for Health and Care Excellence (NICE) has recommended that GW Pharmaceuticals’ rare disease therapies, Epidyolex (cannabidiol) oral solution and Sativex (nabiximols), receive routine reimbursement from NHS England.

Plant-derived cannabis-based cannabidiol oral solution is recommended as an adjunctive therapy for seizures associated with Lennox Gastaut syndrome (LGS) or Dravet syndrome, in conjunction with clobazam, for patients two years of age and older. Nabiximols, reviewed as part of NICE’s evaluation of cannabis-based medicinal products, has been considered cost effective for the treatment of spasticity due to multiple sclerosis.

“This is a momentous occasion for UK patients and families who have waited for so many years for rigorously tested, evidenced and regulatory approved cannabis-based medicines to be reimbursed by the NHS,” said Chris Tovey, GW’s chief operating officer. “This is proof that cannabis-based medicines can successfully go through extensive randomized placebo-controlled trials and a rigorous NICE evaluation process to reach patients.”

Lennox-Gastaut syndrome (LGS) typically occurs between ages of 3 to 5 years and can be caused by a number of conditions, including brain malformations, severe head injuries, central nervous system infections and genetic neuro-degenerative or metabolic conditions. In up to 30 percent of patients, no cause can be found. Patients with LGS commonly have multiple seizure types including drop and convulsive seizures, which frequently lead to falls and injuries, and non-convulsive seizures. Resistance to anti-epileptic drugs is common in patients with LGS. Most patients with LGS experience some degree of intellectual impairment, as well as developmental delays and aberrant behaviors.

Dravet syndrome is a severe infantile-onset and highly treatment-resistant epileptic encephalopathy frequently associated with genetic mutations in the sodium channel gene SCN1A. Initial seizures are often body temperature related, severe, and long-lasting. Over time, patients with Dravet syndrome often develop multiple types of seizures, which can be life threatening. Additionally, the majority of patients will develop moderate to severe intellectual and development disabilities and require lifelong supervision and care.

“This is a significant moment for adults and children with the most difficult to treat epilepsies,” said Rhys Thomas, consultant neurologist at the Royal Victoria Hospital in Newcastle.

“We welcome the addition of cannabidiol oral solution as a new medicine to add to the Dravet syndrome treatment armamentarium. Dravet syndrome is a devastating condition and having a new treatment option offers potential new hope to patients and their families searching for better seizure control,” said Galia Wilson, chair, Dravet Syndrome UK. “Many families come to us asking about the potential of cannabis-based medicines, particularly cannabidiol, and we are thrilled that one is now available on the NHS.”

When added to other anti-epileptic therapies, GW’s cannabidiol oral solution significantly reduced the frequency of seizures in patients with LGS and Dravet syndrome. The most common adverse reactions that occurred in patients treated with the medicine were somnolence, decreased appetite, diarrhea, pyrexia, fatigue and vomiting. GW’s development program represents the only well-controlled clinical evaluation of a cannabinoid medication for patients with refractory epilepsy.

GW’s cannabidiol oral solution was approved by the European Medicines Agency and received marketing authorization in September 2019 under the trade name Epidyolex as an adjunctive therapy for seizures associated with LGS or Dravet syndrome, in conjunction with clobazam, for patients two years of age and older.

The inclusion of nabiximols in NICE guidelines comes as part of the comprehensive evaluation of its clinical and cost-effectiveness. Nabiximols has been approved by medicines regulators in more than 25 countries around the world.

Author: Rare Daily Staff

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