Jazz Pharmaceuticals receives Health Canada approval for Epidiolex® (cannabidiol oral solution) for the treatment of seizures associated with three rare forms of epilepsy

MISSISSAUGA, ON, Nov. 23, 2023 /CNW/ - Jazz Pharmaceuticals Canada Inc. today announced that Epidiolex^® (cannabidiol oral solution), has been approved by Health Canada for use as adjunctive therapy for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome, or tuberous sclerosis complex (TSC) in patients 2 years of age and older.¹

These three conditions are rare and severe forms of epilepsy that cause multiple seizure types, which are generally resistant to treatment.^2,3,4

"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.¹ 

"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.¹

"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.⁵ The estimated prevalence LGS is approximately three to 28 in 100,000 people.⁶

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.⁷ Research suggests the condition affects between one in 20,000 to one in 40,000 people.⁷ Three to eight per cent of children who have their first seizure by 12 months old may have Dravet syndrome.⁷

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,⁸ and more than 60 per cent of individuals with TSC do not achieve seizure control with standard treatments.⁴

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.

For more information, please refer to the product monograph for Epidiolex located here.

About Jazz Pharmaceuticals plc

Jazz Pharmaceuticals plc (NASDAQ: JAZZ) is a global biopharmaceutical company whose purpose is to innovate to transform the lives of patients and their families. We are dedicated to developing potentially life-changing medicines for people with serious diseases – often with limited or no therapeutic options. We have a diverse portfolio of marketed medicines and novel product candidates, from early to late-stage development, in neuroscience and oncology. Within these therapeutic areas, we are identifying new options for patients by actively exploring small molecules and biologics, and through innovative delivery technologies and cannabinoid science. Jazz is headquartered in Dublin, Ireland and has employees around the globe, serving patients in nearly 75 countries.

About Epidiolex^® (cannabidiol oral solution)

Epidiolex^® (cannabidiol oral solution) is indicated for use as adjunctive therapy for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC) in patients 2 years of age and older.¹ It was approved by Health Canada on November 15, 2023.¹ 

Epidiolex (known as Epidyolex in the European Union) has also received approval for the adjunct treatment of seizures associated with LGS, Dravet Syndrome or TSC, for certain patient populations, in 36 countries, including the U.S., EU, Great Britain., Australia, New Zealand, Israel and Switzerland.

About Lennox-Gastaut syndrome (LGS)

Lennox-Gastaut syndrome (LGS) is a severe and treatment-resistant rare epilepsy syndrome with no singular cause. 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.⁵ Patients with LGS commonly have multiple seizure types including drop and convulsive seizures, which frequently lead to falls and injuries, and are predisposed to comorbidities such as prolonged seizures called status epilepticus.⁵ In addition, patients with LGS are at high-risk for SUDEP (sudden unexpected death in epilepsy)⁹ and head/bodily injuries due to falls associated with "drop" seizures.⁵ The the estimated prevalence LGS is approximately three to 28 in 100,000 people.⁶ The peak age for onset is between three to five years of age with extreme incidence occurring in the first and tenth years of life.¹⁰

About Dravet syndrome

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 SCN1A⁷. Onset of Dravet syndrome occurs during the first year of life in previously healthy and developmentally normal infants. Risk of premature death including SUDEP (sudden unexpected death in epilepsy) is elevated in patients with Dravet syndrome.¹¹ Adequate seizure control remains a significant concern for many patients despite currently available anti-seizure treatment options. Reports suggest that Dravet syndrome affects between one in 20,000 to one in 40,000 people and that three to eight per cent of children who have their first seizure by 12 months old may have Dravet syndrome.⁷

About Tuberous sclerosis complex (TSC)

Tuberous sclerosis complex (TSC) is a rare genetic condition that affects as many as one million individuals worldwide.⁸ The condition causes mostly benign tumours to grow in vital organs of the body including the brain, skin, heart, eyes, kidneys and lungs and is a  genetic cause of epilepsy.⁸ The onset of epilepsy in TSC often occurs in the first year of life with patients initially experiencing predominantly focal seizures or infantile spasms.¹² It is associated with an increased risk of autism spectrum disorder and many children with TSC have a developmental delay, intellectual disability or behavioral problems.³ The severity of the condition can vary widely: in some patients the disease is very mild, while others may experience life-threatening complications.³ 

References

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¹ Epidiolex Product Monograph. Available at: https://pp.jazzpharma.com/pi/epidiolex.ca.PM-en.pdf/ Accessed November 17, 2023.

² Villanueva V, Carreño-Martínez M, Gil-Nagel A, et al. New therapeutic approach in Dravet syndrome and Lennox-Gastaut syndrome with cannabidiol. Rev Neurol 2021;72 (Supl.1):S0-S10. doi: 10.33588/rn.72S01.2021017

³ Boston Children's Hospital. Tuberous Sclerosis Complex (TSC). ChildrensHospital.org. Accessed October 18, 2023. https://www.childrenshospital.org/conditions/tuberous-sclerosis-tsc

⁴ Chu-Shore CJ, Major P, Camposano S, et al. The natural history of epilepsy in tuberous sclerosis complex. Epilepsia. 2010 Jul;51(7):1236-41. Accessed [insert date]. doi: https://doi.org/10.1111/j.1528-1167.2009.02474.x

⁵ National Organization for Rare Disorders. Lennox-Gastaut Syndrome. rarediseases.org. Updated June 5, 2020. Accessed October 18, 2023. https://rarediseases.org/rare-diseases/lennox-gastaut-syndrome/.

⁶ Strzelczyk A, Zuberi SM, Striano P, Rosenow F, Schubert-Bast S. The burden of illness in Lennox-

Gastaut syndrome: a systematic literature review. Orphanet J Rare Dis. Mar 1 2023;18(1):42.

doi:10.1186/s13023-023-02626-4

⁷ Epilepsy Foundation. Dravet Syndrome Fact Sheet. Updated August 24, 2020. Epilepsy.com. Accessed October 18, 2023. https://www.epilepsy.com/what-is-epilepsy/syndromes/dravet-syndrome.

⁸ TSC Alliance. What is TSC? [online]. TSC Alliance website. Available at: https://www.tscalliance.org/about-tsc/what-is-tsc/. Accessed: Accessed October 18, 2023

⁹ LGS Foundation. What is LGS? Fact Sheet. lgsfoundation.org. Accessed October 18, 2023. https://www.lgsfoundation.org/wp-content/uploads/2023/08/LGS-Fact-Sheet-2023-1.pdf.

¹⁰ Epilepsy Ontario. Lennox-Gastaut Syndrome. Accessed October 16, 2023. Further information on Lennox-Gastaut Syndrome syndrome can be found at: https://epilepsyontario.org/lennox-gastaut-syndrome/.

¹¹ National Organization for Rare Disorders. Dravet Syndrome. RareDiseases.org. Updated 2020. Accessed October 18, 2023. https://rarediseases.org/rare-diseases/dravet-syndrome-spectrum.  

¹² Nabbout R, Belousova E, Benedik MP, et al. Epilepsy in tuberous sclerosis complex: findings from the TOSCA Study. Epilepsia Open. 2018;4:73–84. Accessed October 18, 2023.DOI https://doi.org/10.1002/epi4.12286.

SOURCE Jazz Pharmaceuticals Canada Inc

Media Contacts: GCI Canada, Anna Campbell, Vice President, [email protected], [phone]; Jazz Pharmaceuticals, Kristin Bhavani, Head of Strategic Brand Engagement, [email protected], Ireland [phone], U.S. [phone]; Investors: Andrea N. Flynn, Ph.D., Vice President, Head, Investor Relations, Jazz Pharmaceuticals plc, [email protected], Ireland [phone], U.S. [phone]