Jazz Pharmaceuticals receives NICE recommendation for the reimbursement of Epidyolex® (cannabidiol) for the treatment of seizures associated with tuberous sclerosis complex in England

LONDON, Jan. 31, 2023 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that Epidyolex^® (cannabidiol,100 mg/mL oral solution), developed by GW Pharmaceuticals ("GW"; now part of Jazz Pharmaceuticals plc), a world leader in discovering, developing and delivering regulatory approved cannabinoid-based medicines, has been recommended for reimbursement by National Institute for Health and Care Excellence (NICE) for adjunctive therapy of seizures associated with tuberous sclerosis complex (TSC), for patients two years of age and older.[1]

Following earlier decisions by the Scottish Medicines Consortium, Northern Ireland's Strategic Planning and Performance Group and the All Wales Medicines Strategy Group this recommendation now ensures all eligible UK patients who may benefit from this medicine will be able to receive reimbursed access to Epidyolex^® from 1 March 2023.  

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,[2] and in which epilepsy is one of the most common neurological features[3] – and can severely impact the lives of these patients. TSC can be diagnosed in infancy but many children are not diagnosed until later in childhood when their seizures begin and other symptoms appear.[4] It is estimated that between 3,700 and 11,000 people in the UK live with TSC.[5]

Dr. Pooja Takhar, Joint Chief Executive at Tuberous Sclerosis Association (TSA), said "The TSA is delighted with this decision, along with people living with TSC and their families. TSC is a very difficult to manage condition, with common issues including epilepsy in eight out of ten people. Up to half of the people with TSC-related epilepsy are unable to manage their seizures with standard anti-seizure medication, leading to a massive unmet need for new treatment options. This underlines why we are so pleased that this medicine will now be available on the NHS in England, improving lives in the TSC community."

"We welcome NICE's recommendation which provides appropriate patients across the UK, who are living with TSC, a difficult to treat condition, access to a new treatment option. This is an important milestone not only for those living with TSC but also for their families, carers and clinicians," said Simon Newton, General Manager at Jazz Pharmaceuticals. "This demonstrates the importance of randomised clinical trials and regulatory approval in providing reimbursed access to cannabinoid-based medicines to patients who may benefit."

Jazz's cannabinoid-based medicine has been approved for use in the UK in three conditions. In July 2019[6], it was approved by the European Medicines Agency (EMA) and received marketing authorisation under the trade name Epidyolex as an adjunctive therapy for seizures associated with Lennox Gastaut syndrome or Dravet syndrome, in conjunction with clobazam, for patients two years of age and older. In August 2021,[7] it was approved by the UK Medicines and Healthcare Products Regulatory Agency (MHRA) for use as an adjunctive treatment of seizures associated with TSC, for patients two years of age and older.

NOTES TO EDITORS

Please note that, in relation to this Jazz media announcement, no honorarium was provided to the Tuberous Sclerosis Association.

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 GW Pharmaceuticals

GW Pharmaceuticals (GW), now part of Jazz Pharmaceuticals plc, has established a world-leading position in cannabinoid science and medicine. Founded over two decades ago in response to significant unmet patient need, patients remain our key focus and improving their quality of life, our motivation. GW's pioneering work has led to the regulatory approval of cannabinoid-based medicines. Our continued dedication has resulted in the treatment of thousands of patients with our medicines around the world. In 2021, GW received the Queens Award for Enterprise in Innovation in recognition of its innovative and ground-breaking work in cannabinoid science and medicines. GW was acquired by Jazz Pharmaceuticals in May 2021.

About Epidiolex®/Epidyolex® (cannabidiol)

Epidiolex®/Epidyolex® (cannabidiol; 100 mg/mL oral solution), a prescription, plant-derived cannabinoid-based medicine approved by the U.S. Food and Drug Administration (FDA) for use in the U.S,[8] and the European Medicines Agency (EMA) for use in the European Union,[9] is an oral solution which contains highly purified cannabidiol (CBD). In the U.S., cannabidiol is indicated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome or Tuberous Sclerosis Complex (TSC) in patients one year of age and older.⁸ Cannabidiol has also received approval in the European Union, under the tradename Epidyolex, for adjunctive use in conjunction with clobazam to treat seizures associated with LGS and Dravet syndrome in patients two years and older, and for adjunctive use to treat seizures associated with TSC, in patients two years of age and older.⁹ 

About Tuberous sclerosis complex (TSC)

Tuberous sclerosis complex (TSC) is a rare genetic condition that has an estimated prevalence in Europe of 12 in 100,000.[10] The condition causes mostly benign tumours to grow in vital organs of the body including the brain, skin, heart, eyes, kidneys and lungs² and is a common genetic cause of epilepsy.[11] The onset of epilepsy in TSC often occurs in the first year of life with patients initially suffering from predominantly focal seizures or infantile spasms.^3,[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.^6,[13] The severity of the condition can vary widely: in some patients the disease is very mild, while others may experience life-threatening complications.⁴

References

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[1] National Institute for Health and Care Excellence. Final Appraisal Document – Cannabidiol for treating seizures caused by tuberous sclerosis complex; Available at: https://www.nice.org.uk/guidance/gid-ta10840/documents/final-appraisal-determination-document. Accessed January 2023.

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

[3] Nabbout R, Belousova E, Benedik MP et al. Epilepsy in tuberous sclerosis complex: findings from the TOSCA Study. Epilepsia Open 2018;4:73–84. Available at: https://doi.org/10.1002/epi4.12286

Accessed: January 2023

[4] National Institute of Neurological Disorders and Stroke. Tuberous Sclerosis Fact Sheet [online]. Available at: https://www.ninds.nih.gov/health-information/disorders/tuberous-sclerosis-complex. Accessed: January 2023

[5] Tuberous Sclerosis Association website. https://tuberous-sclerosis.org/information-and-support/what-is-tsc/ Accessed January 2023

[6] Committee for Medicinal Products for Human Use. Epidyolex European public assessment report. European Medicines Agency. https://www.ema.europa.eu/en/documents/assessment-report/epidyolex-epar-public-assessment-report_en.pdf. Published July 25, 2019. Accessed January 2023

[7] Electronic Medicines Compendium. Epidyolex 100 mg/ml oral solution.  https://www.medicines.org.uk/emc/product/10781/smpc#gref. Published January 1, 2021. Accessed January 2023

[8] Epidiolex [package insert] Carlsbad, CA: Greenwich Biosciences Inc; 2022. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/210365s015lbl.pdf. Accessed: January 2023

[9] Committee for Medicinal Products for Human Use. Epidyolex European Public Assessment Report. SmPC, European Medicines Agency. https://www.ema.europa.eu/en/documents/product-information/epidyolex-epar-product-information_en.pdf. Published September 2022. Accessed: January 2023

[10] Tuberous sclerosis complex: 805 [online]. Prevalence and incidence of rare diseases; Orphanet, 2019; Orpha.net. Available at: https://www.orpha.net/orphacom/cahiers/docs/GB/Prevalence_of_rare_diseases_by_diseases.pdf. Accessed: January 2023

[11] Jozwiak S, Kotulska K, Wong M et el. Modifying genetic epilepsies – Results from studies on tuberous sclerosis complex. Neuropharmacology 2020; 166;107908:3-8. Available at: https://doi.org/10.1016/j.neuropharm.2019.107908. Accessed: January 2023

[12] 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. Available at: https://doi.org/10.1111/j.1528-1167.2009.02474.x Accessed: January 2023

[13] De Ridder J, Lavanga, M, Verhelle, B et al. Prediction of Neurodevelopment in Infants With Tuberous Sclerosis Complex Using Early EEG Characteristics. Frontiers, 2020; 11:582891. Available at: https://doi.org/10.3389/fneur.2020.582891.s001. Accessed: January 2023

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