GW Pharmaceuticals receives approval for the reimbursement of Epidyolex® (cannabidiol) for the treatment of seizures associated with Tuberous sclerosis complex in Wales

GW Pharmaceuticals receives approval for the reimbursement of Epidyolex^® (cannabidiol) for the treatment of seizures associated with Tuberous sclerosis complex in Wales

Wales becomes the first UK nation to fund Epidyolex^® (cannabidiol) for Tuberous sclerosis complex (TSC)

TSC causes epilepsy in up to 85% of patients, and up to 60% of those patients do not respond to standard anti-seizure medicines^1,2,3

DUBLIN, 15 December 2021 – Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced Epidyolex^® (cannabidiol), developed by GW Pharmaceuticals (“GW”), a world leader in discovering, developing and delivering regulatory approved cannabis-based medicines, has been recommended by the All Wales Medicines Strategy Group (AWMSG) for use within NHS Wales for adjunctive therapy of seizures associated with tuberous sclerosis complex (TSC), for patients two years of age and older.

This decision sees Wales become the first country in the UK to make GW’s cannabidiol available for routine reimbursement for TSC. Having already received reimbursement for Lennox-Gastaut Syndrome (LGS) and Dravet Syndrome, this decision means that all three conditions for which Epidyolex is licenced are now reimbursed in Wales.

It follows the authorisation of GW’s cannabidiol 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 in August 2021.

“This is an extremely important decision for patients, their families and clinicians in Wales, where there is a need for additional treatment options for those suffering with this difficult to treat condition. We are delighted that the AWMSG has recommended the use of a rigorously tested, evidenced, regulatory approved cannabis-based medicines for seizures associated with TSC; the first UK nation to recommend use in all three of the medicine’s licensed indications,” said Chris Tovey, executive vice president, chief operating officer and managing director, Europe & International at Jazz Pharmaceuticals. “This decision demonstrates that cannabis-based medicines can go through extensive randomised placebo-controlled trials and a rigorous assessment by the AWMSG to reach patients with significant unmet need.”

Louise Fish, CEO of Tuberous Sclerosis Association, said: “We are delighted with this decision in Wales to reimburse Epidyolex for tuberous sclerosis complex. Between 3,700 and 11,000 people in the UK, including in Wales, are living with TSC, and eight out of ten people living with TSC have epilepsy. To date, TSC-related epilepsy has often been difficult to manage with conventional epilepsy drugs and therefore new treatment options are desperately needed. We hope that other areas of the UK will follow Wales’ lead and make this new option available to patients who could benefit from this treatment.”

ADDITIONAL INFORMATION

About GW Pharmaceuticals

GW Pharmaceuticals (GW), a subsidiary group 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 world first, potentially life changing, cannabis-based medicines. Our continued dedication has resulted in the treatment of thousands of patients with our medicines around the world. In 2021, GW was 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 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 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. For more information, please visit www.jazzpharma.com and follow @JazzPharma on Twitter.

About Epidiolex^®/Epidyolex^® (cannabidiol)

Epidiolex^®/Epidyolex^® (cannabidiol), the first prescription, plant-derived cannabis-based medicine approved by the U.S. Food and Drug Administration (FDA) for use in the U.S. and the European Commission (EC) for use in the European Union, 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. Cannabidiol has 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. cannabidiol has received Orphan Drug Designation from the European Medicines Agency (EMA) for the treatment of seizures associated with LGS, Dravet syndrome, and TSC.

About Tuberous Sclerosis Complex (TSC)

Tuberous Sclerosis Complex (TSC) is a rare genetic condition that has an estimated prevalence in Europe of 10 in 100,000.⁶ 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 leading cause of genetic epilepsy.^4,6 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. It is associated with an increased risk of autism and intellectual disability.⁵ The severity of the condition can vary widely. In some children the disease is very mild, while others may experience life-threatening complications.^7,8

1.       Nabbout R, Belousova E, Benedik MP, et al. Epilepsy in tuberous sclerosis complex: Findings from the TOSCA Study. Epilepsia Open. 2019 Mar; 4(1): 73–84.

2.       Boston Children’s Hospital. https://www.childrenshospital.org/conditions-and-treatments/conditions/t/tuberous-sclerosis-tsc/symptoms-and-causes. Accessed March 2021.

3.       Chu-Shore CJ, Major P, Camposano S, Muzykewicz D, Thiele EA. The natural history of epilepsy in tuberous sclerosis complex. Epilepsia 2010;51(7):1236–41.

4.       NIH Tuberous Sclerosis Fact Sheet. https://www.ninds.nih.gov/Disorders/Patient-Caregiver-Education/Fact-Sheets/Tuberous-Sclerosis-Fact-Sheet.

5.       Tuberous Sclerosis Association website. https://tuberous-sclerosis.org/information-and-support/what-is-tsc/. Accessed August 2021

6.       Prevalence and incidence of rare diseases: Bibliographic data. https://www.orpha.net/orphacom/cahiers/docs/GB/Prevalence_of_rare_diseases_by_alphabetical_list.pdf

7.       TS Alliance Website. https://www.tsalliance.org/. Accessed August 2021

8.       de Vries PJ, Belousova E, Benedik MP, et al. TSC-associated neuropsychiatric disorders (TAND): findings from the TOSCA natural history study. Orphanet J Rare Dis. 2018;13(1):157.