Jazz Pharmaceuticals to Present Extensive New Data and Real-World Evidence Highlighting Epidiolex® Outcomes in Treatment-Resistant Epilepsies at the American Epilepsy Society 2025 Annual Meeting
New interim results from the EpiCom trial, a prospective evaluation of behavioral outcomes in patients with tuberous sclerosis complex, suggest improvements in non-seizure outcomes
Eight abstracts, including four late-breaking abstracts, underscore Jazz's continued commitment to the epilepsy community and advancing the comprehensive treatment for rare forms of epilepsy
DUBLIN, Dec. 5, 2025 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that eight abstracts, including four that are late-breaking, will be presented on Epidiolex® (cannabidiol) at the American Epilepsy Society (AES) 2025 Annual Meeting, being held December 5-9, 2025, in Atlanta, Georgia.
Notable data include new results from the EpiCom trial, an ongoing Phase 3b/4 study evaluating behavioral and other non-seizure outcomes after initiation of adjunctive Epidiolex treatment in participants with tuberous sclerosis complex (TSC)-associated seizures. Notably, the prespecified 6-month intermediate analysis of EpiCom demonstrated promising reductions in the TSC–Associated Neuropsychiatric Disorders Self-Report Quantified Checklist (TAND-SQ) and Aberrant Behavior Checklist (ABC) subscale scores, as well as in the severity of behavioral problems reported by caregivers and clinicians after 26 weeks of Epidiolex treatment.
"We are proud to share extensive research at AES 2025, including findings from the innovative EpiCom trial which utilized novel endpoints focused on individualized outcomes, including each participant's respective most problematic behavior as identified by caregivers and clinicians to ensure our research addresses the priorities of those we serve," said Jessa Alexander, Ph.D., neuroscience therapeutic area head, global medical and scientific affairs of Jazz Pharmaceuticals. "Data from EpiCom and real-world insights continue to expand our understanding of the complex realities faced by patients living with rare epilepsy disorders while further underscoring Epidiolex's ability to contribute to meaningful, holistic management."
Additional highlights at AES 2025 include:
A late-breaking poster featuring results from a Phase 1 clinical study evaluating bidirectional pharmacokinetic (PK) drug-drug interactions between Epidiolex and cenobamate, which did not find PK interactions between cannabidiol and cenobamate parent molecules when concomitantly administered at clinically relevant dosages.
A late-breaking poster showcasing preclinical data that demonstrates a novel synergistic pharmacodynamic interaction between Epidiolex and cenobamate in effectiveness in the mouse maximal electroshock seizure model of acute generalized seizures.
Two presentations highlight real-world effectiveness of Epidiolex using data from the U.S. Optum® Market Clarity Database (an integrated electronic health record and claims database). One late-breaking poster features data that shows Epidiolex initiation in CBD-naïve patients with Dravet Syndrome (DS), LGS or TSC is associated with reduced polypharmacy and healthcare resource utilization (HCRU) at 12 months, with further reductions among early-line Epidiolex initiators. The other poster highlights data that indicates Epidiolex initiation also reduces antiseizure medication cycling at 12 months, as well as decreased polypharmacy and HCRU in both pediatric and adult patients.
A poster presentation featuring post-hoc analysis from the Expanded Access Program demonstrates Epidiolex treatment is associated with reductions in convulsive and total seizure frequency in patients with developmental and epileptic encephalopathies (DEEs) and rare epilepsy syndromes. The comparative effectiveness analysis confirms the superiority of Epidiolex in reducing seizure frequency from baseline compared to an external placebo control arm.
A poster presentation highlighting a subgroup analysis of Epidiolex pivotal clinical trial data showing participants with Lennox-Gastaut syndrome (LGS) diagnoses, which are secondary to a genetic DEE, experienced consistent efficacy and safety as compared to the overall LGS pivotal trial populations.
A late-breaking poster presentation on data from the Epilepsy Learning Healthcare System (ELHS), an Epilepsy Foundation-sponsored initiative, highlights its potential to provide real-world treatment insights beyond clinical trials and claims data.
All AES 2025 abstracts are available online at the following link: aesnet.org/education/annual-meeting/aes-abstract-search.
A full list of Jazz Pharmaceuticals' presentations follows below:
Cannabidiol Efficacy in Patients With Lennox-Gastaut Syndrome With Developmental
and Epileptic Encephalopathy-Associated Genetic Variants: A Subgroup AnalysisE Thiele
Poster Number: 1.382
Session Date/Time: Saturday
December 6, 12:00-2:00pm
Phase 1, Open-Label, Fixed-Sequence, Bidirectional, Pharmacokinetic Drug-Drug
Interaction Study Between Cannabidiol and Cenobamate in Healthy Adult ParticipantsA Vijan
Poster Number: 1.545
Session Date/Time: Saturday
December 6, 12:00-2:00pm
An Assessment of Acute Pharmacodynamic Drug-Drug Interactions Between
Cannabidiol and Cenobamate in a Mouse Model of Generalized Tonic SeizuresW Hind
Poster Number: 1.546
Session Date/Time: Saturday
December 6, 12:00-2:00pm
A Population Health Study of Patients Prescribed Cannabidiol in the Epilepsy
Learning Healthcare System (ELHS) RegistryKM Farrell
Poster Number: 1.549
Session Date/Time: Saturday
December 6, 12:00-2:00pm
Effectiveness of Cannabidiol in Patients With Rare Epilepsies Compared to External
Placebo Control: A Post-Hoc Analysis From the Expanded Access ProgramY Park
Poster Number: 2.337
Session Date/Time: Sunday
December 7, 12:00-2:00pm
Real-World Polypharmacy and Healthcare Resource Utilization After Early-Line
Treatment With Cannabidiol for Lennox-Gastaut Syndrome, Dravet Syndrome, and
Tuberous Sclerosis ComplexM Navetta
Poster Number: 2.433
Session Date/Time: Sunday
December 7, 12:00-2:00pm
Tuberous Sclerosis Complex (TSC)–Associated Neuropsychiatric Disorders (TAND)
Outcomes Following Adjunctive Cannabidiol (CBD) Treatment: 6-Month Intermediate
Analysis of the EpiCom TrialJ Stevens
Poster Number: 3.351
Session Date/Time: Monday
December 8, 12:00-1:45pm
Real-World Effectiveness of Cannabidiol on Antiseizure Medication Cycling,
Polypharmacy, and Healthcare Resource Utilization: A US Claims AnalysisA Sillah
Poster Number: 3.368
Session Date/Time: Monday
December 8, 12:00-1:45pm
About Tuberous Sclerosis Complex
Tuberous sclerosis complex (TSC) is a rare genetic condition.1 The condition causes mostly benign tumors to grow in vital organs of the body including the brain, skin, heart, eyes, kidneys and lungs2 and is a leading cause of genetic epilepsy.3 People with TSC may experience a variety of seizure types. One of the most common is infantile spasms that typically present in the first year of life; focal (or partial) seizures are also very common.4 TSC is associated with an increased risk of autism and intellectual disability5 and the severity of the condition can vary widely. In some children the disease is very mild, while others may experience life-threatening complications.4 Epilepsy is present in about 85% of patients with TSC and may progress to become intractable to medication.4,6,7 More than 60% of individuals with TSC do not achieve seizure control8 with standard treatments such as antiepileptic drugs, epilepsy surgery, ketogenic diet, or vagus nerve stimulation8 compared to 30%-40% of individuals with epilepsy who do not have TSC who are drug resistant.9,10
About Dravet Syndrome
Dravet syndrome (DS) is a rare genetic condition that appears during the first year of life with frequent fever-related seizures (febrile seizures). Later, other types of seizures typically arise, including myoclonic seizures (involuntary muscle spasms).11 Additionally, status epilepticus, a potentially life-threatening state of continuous seizure activity requiring emergency medical care, may occur. Children with DS typically experience poor development of language and motor skills, hyperactivity and difficulty relating to others.
About Lennox-Gastaut Syndrome
Lennox-Gastaut syndrome (LGS) begins in childhood. It is characterized by multiple types of seizures. People with LGS begin having frequent seizures in early childhood, usually between ages 3 and 5.12 More than three-quarters of affected individuals have tonic seizures, which cause the muscles to contract uncontrollably. Almost all children with LGS develop learning problems and intellectual disability. Many also have delayed development of motor skills such as sitting and crawling. Most people with LGS require help with usual activities of daily living.
About Epidiolex®/Epidyolex® (cannabidiol)
Epidiolex/Epidyolex is a prescription, plant-derived cannabis-based medicine administered as an oral solution which contains highly purified cannabidiol (CBD). Cannabidiol, the active ingredient in Epidiolex, is a cannabinoid that naturally occurs in the Cannabis sativa L. plant. The precise mechanisms by which Epidiolex exerts its anticonvulsant effect in humans are unknown. Epidiolex was approved by the U.S. Food and Drug Administration (FDA) for use in the U.S., the European Commission (EC) for use in Europe, the Medicines and Healthcare products Regulatory Agency (MHRA) for use in Great Britain, the Therapeutic Goods Administration for use in Australia, Swissmedic for use in Switzerland, the Food & Nutrition Services of the Israel Ministry of Health for use in Israel, and the New Zealand Medicines and Medical Devices Safety Authority for use in New Zealand, is an oral solution which contains highly purified cannabidiol (CBD). In the U.S., Epidiolex is indicated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS) or tuberous sclerosis complex (TSC) in patients one year of age and older. Epidiolex 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 DS in patients two years and older, and for adjunctive use to treat seizures associated with TSC, in patients two years of age and older. Epidiolex has received Orphan Drug Designation (ODD) from the U.S. FDA for the treatment of seizures associated with LGS, DS, and TSC. Similarly, Epidyolex received ODD from the European Medicines Agency (EMA) for the same indications. Epidiolex is also being studied in additional forms of epilepsy, including the EpiFOS exploratory study (NCT07233239) in focal-onset seizures.