These three conditions are rare and severe forms of epilepsy that cause multiple seizure types, which are generally resistant to treatment.
“Living with LGS, Dravet syndrome or TSC is very difficult. It means enduring recurring seizures every day. This presents a challenging and isolating experience for individuals living with these conditions to experience and places a great deal of stress on caregivers who witness the seizures,” says Laura Dickson, president of the Canadian Epilepsy Alliance. “This approval encourages hope and optimism for this community. The priority now becomes ensuring those living with these conditions have equitable and timely access to promising treatment options.”
Epidiolex is a prescription medicine, administered as an oral solution. Cannabidiol, the active ingredient in Epidiolex, is a cannabinoid that naturally occurs in the Cannabis sativa L. plant.
“While there are many anti-seizure medications available for different epilepsies, as rare and severe forms of childhood-onset epilepsies, LGS, Dravet syndrome and TSC have limited approved therapies and are often treatment-resistant. This makes these conditions extremely difficult to treat,” says Linda Huh, paediatric epileptologist at BC Children’s Hospital and the University of British Columbia. “The Health Canada approval of Epidiolex is significant news for a patient population with a critical unmet need, providing us with a robustly studied treatment option.”
The Health Canada approval was based on results from five double-blind, randomized, placebo-controlled Phase 3 clinical trials, with a total of 939 LGS, Dravet syndrome, or TSC patients enrolled, making it one of the largest global clinical trial programs to date in rare refractory epilepsy syndromes.
“The approval of Epidiolex is an important development for individuals living with specific rare epilepsies, their families, and clinicians across Canada, providing a new treatment option for those living with LGS, Dravet syndrome or TSC,” said Paul Petrelli, General Manager of Jazz Pharmaceuticals Canada Inc. “This decision affirms the potential of cannabinoid-based medicines for those living with epilepsy. It also reaffirms Jazz’s commitment to bringing forward new therapeutic options for Canadians living with rare and debilitating neurological conditions.”
The onset of LGS typically occurs between ages of three to five 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. The estimated prevalence LGS is approximately three to 28 in 100,000 people.
Dravet syndrome is a severe infantile-onset and highly treatment-resistant developmental and epileptic encephalopathy frequently associated with genetic mutations in the sodium channel gene SCN1A. Research suggests the condition affects between one in 20,000 to one in 40,000 people. Three to eight per cent of children who have their first seizure by 12 months old may have Dravet syndrome.
TSC is a condition that causes mostly benign tumours to grow in vital organs of the body, including the brain, skin, heart, eyes, kidneys and lungs, and in which, epilepsy is one of the most common neurological manifestations. TSC affects as many as one million individuals worldwide, and more than 60 per cent of individuals with TSC do not achieve seizure control with standard treatments.
This decision represents the authorization of Jazz Canada’s first approved cannabinoid-based medicine and underlines the company’s commitment to developing, researching, and commercializing regulatory approved cannabinoid-based medicines.
Following the Health Canada approval, Jazz will work with the relevant stakeholders in Canada, to secure reimbursement for eligible patients.
In addition to Canada, Jazz has received approval for the cannabidiol oral solution Epidiolex (also known as Epidyolex in the European Union) for the treatment of seizures associated with LGS, Dravet Syndrome or TSC, for certain patient populations, in 36 countries other countries, including the U.S., EU, Great Britain, Australia, New Zealand, Israel and Switzerland.