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Jazz Pharma to present new research findings on Epidyolex

The company has shared new findings on its role in treating seizures related to rare forms of epilepsy.



Jazz Pharma to present new research findings on Epidiolex

The company behind the cannabis-based treatment, Epidyolex, has shared new findings on its role in treating seizures related to rare forms of epilepsy.

Jazz Pharmaceuticals will share new research on its CBD-based drug, Epidyolex/Epidiolex at the upcoming American Epilepsy Society (AES) annual meeting from 2-6 December in Nashville, US.

Data from six abstracts is expected to be presented, including new long-term Phase 3 results and two real-world analyses of US caregiver-reported seizure and non-seizure outcomes in patients prescribed the medication.

Irish firm, Jazz Pharmaceuticals, acquired GW Pharmaceuticals the UK producer of Epidyolex last year for USD$7.2million.

Epidyolex is a pharmaceutical formulation of highly-purified cannabidiol (CBD),which can be prescribed on the NHS for seizures associated with Lennox-Gastaut Syndrome (LGS) and Dravet Syndrome (DS) in patients two years and older.

It is the first prescription, plant-derived cannabis-based medicine approved by the US Food and Drug Administration (FDA).

New research

Experts from the company will present the findings from a global survey of caregivers of patients with LGS or DS, which aimed to quantify seizure and non-seizure outcomes of the treatment.

The survey explores the effects of CBD in paediatric compared to adult patients. 

According to the paper’s abstract, the primary analysis showed a ‘substantial proportion’ of caregivers reported improvements in seizure frequency and severity.

Jazz Pharmaceuticals has also supported research into the development of a tool to improve the diagnosis of LGS, known as the Refractory Epilepsy Screening Tool for LGS patients (REST-LGS)

The condition is frequently undiagnosed in the adult population, but identifying adults likely to meet criteria for LGS, but with no formal diagnosis, could improve access to care, says the paper’s abstract. 

REST-LGS indicated that over half of the individuals involved in the study, without a previous LGS diagnosis, should be referred to an epilepsy centre for further diagnostic evaluation.

‘Continually striving’

Kelvin Tan, MB BCh, MRCPCH, senior vice president and chief medical officer of Jazz Pharmaceuticals, commented: “We understand the significant, lifelong implications of severe, childhood-onset epilepsy on both patients and their families. We continually strive to improve patient outcomes and are proud to present data that includes caregiver-reported insight into Epidyolex’s effects on seizure and non-seizure outcomes in both children and adults living with Lennox-Gastaut Syndrome and Dravet Syndrome.

“In addition, we are sharing long-term Phase 3 trial findings which demonstrate Epidyolex’s ability to reduce tuberous sclerosis complex-related seizures over the course of three years of treatment – further underscoring that Epidyolex is an important treatment option in an area with significant unmet patient need.”

Seizures in Tuberous Sclerosis Complex

The company has also been investigating the use of Epidyolex for seizures associated with Tuberous Sclerosis Complex (TSC), having received approval from the Medicines and Healthcare products Regulatory Agency (MHRA) for its use in patients aged two and older, last year.

Final data from a Phase 3 trial suggests that long-term, add-on Epidyolex treatment was ‘well tolerated and demonstrated sustained reductions in TSC-associated seizures’ in patients.

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