Jazz Pharmaceuticals to Showcase New Real-World Evidence Reinforcing Epidiolex® (cannabidiol) Benefits and Broad-Spectrum Efficacy in Treatment-Resistant Epilepsies at the American Epilepsy Society 2024 Annual Meeting
JAZZ 12.06.2024
Data from nine abstracts to be presented, including first data from the EpiCom Trial, a prospective evaluation of behavioral outcomes in patients with tuberous sclerosis complex, which suggests improvements in severity of behavioral symptoms
Real-world data from the BECOME (BEhavior, COgnition and More with Epidiolex®) surveys, which show outcomes reported by long-term care facility nurses and tuberous sclerosis complex caregivers
For
Data presented at the meeting includes updated analyses of real-world data from the BECOME-TSC (BEhavior, COgnition, and More withEpidiolex) caregiver survey, which characterizes and quantifies seizure and non-seizure outcomes in patients with epilepsy and tuberous sclerosis complex (TSC) treated withEpidiolex. An additional presentation showcases data from the BECOME-LTC survey, which evaluated the perspectives of nurses who care for patients with epilepsy in long-term care (LTC) facilities and group homes, reporting improvements in seizure frequency and in certain non-seizure outcomes associated withEpidiolextreatment. Further, the first presentation of data from the EpiCom trial, a prospective, interventional trial evaluating the impact of adjunctiveEpidiolexon TSC-associated neuropsychiatric disorders (TAND), revealed improvements in behavioral symptom severity following treatment initiation.
"Our real-world data presentations at AES 2024, including novel findings from the BECOME-LTC, BECOME-TSC and EpiCom studies, demonstrate the meaningful impact ofEpidiolexin the treatment of patients with rare epilepsies," said
Data highlights include:
- A prespecified three-month analysis of the EpiCom trial reporting TSC-associated neuropsychiatric disorders (TAND)-associated outcomes demonstrated improvements after initiating adjunctiveEpidiolextreatment in the severity of behavioral problems in patients with TSC as reported by the TAND Self-Report Quantified Checklist and Aberrant Behavior Checklist.
- Two updated analyses of real-world outcomes from the BECOME-TSC study showed that, of 55 caregivers who completed the survey, 89% planned to continueEpidiolextreatment for their loved one. The most important stated reasons for continuingEpidiolexincluded seizure and non-seizure benefits such as reduced seizure frequency and severity/duration, as well as TAND-related improvements in cognition and language/communication.
- Results from the BECOME-LTC (BEhavior, COgnition, and More withEpidiolexin the Long-Term Care Setting) survey found that, among 102 nurses surveyed, 85% reported a reduction in overall frequency of any seizure type afterEpidiolexinitiation, with 49% reporting a greater than 50% reduction. Improvements were also observed across different seizure subtypes as well as in non-seizure outcomes, with nurses reporting improvements in emotional functioning, sleep, cognitive abilities, ability to communicate, and physical functioning.
- Results from CARE-EpiC (Caregiver Analysis of Real-worldEpidiolexin Epilepsy Context), a cross-sectional caregiver survey, demonstrated reduced caregivers' need for additional support of their dependents' physical, emotional, and behavioral care afterEpidiolexinitiation and characterized improvements in their dependents' well-being as well as caregivers' experiences.
- A subgroup analysis evaluating treatment outcomes in patients with TSC (TSC group) versus other types of focal epilepsy (non-TSC group) treated withEpidiolexin the
U.S. Expanded Access Program, foundEpidiolexhas similar effectiveness in TSC and other focal epilepsies, regardless of focal epilepsy type, further reinforcing the clinical profile ofEpidiolexas a broad-spectrum agent. In the TSC group,Epidiolexwas associated with a median reduction from baseline of 51%–87% in focal seizures and 44%–87% in total seizures. In the non-TSC group,Epidiolexwas also associated with a median reduction from baseline of 46%–75% in focal and 46%–74% in total seizures.
Epidiolexis indicated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome or TSC in patients one year of age and older. All AES 2024 abstracts are available online at the following link:https://aesnet.org/education/annual-meeting/aes-abstract-search.
A full list of
Presentation Title | Presenting Author | Poster Number / Date & Time (PT) |
Cannabidiol has antiseizure and antitumor effects inpreclinical models of tuberous sclerosis complexthrough mTORC1-independent mechanisms | Poster Number: 1.052Session Date/Time: Saturday, | |
Caregiver-Reported Nonseizure Outcomes withReal-World Use of Cannabidiol (CBD) in Tuberous | Poster Number: 1.433Session Date/Time: Saturday, | |
Caregiver-Reported Real-World Use of Cannabidiol(CBD) and Effects on Seizures and CaregiverBurden: Results From the | Poster Number: 1.434Session Date/Time: Saturday, | |
Caregiver-Reported Seizure Outcomes with Real-World Use of Cannabidiol (CBD) in Tuberous | Poster Number: 1.435Session Date/Time: Saturday, | |
Effects of Cannabidiol (CBD) on AudiogenicSeizures in the DBA/1 Mouse Model of SuddenUnexpected Death in Epilepsy (SUDEP) | Poster Number: 2.354Session Date/Time: Sunday, | |
Real-World Outcomes of Cannabidiol (CBD) in | Poster Number: 2.369Session Date/Time: Sunday, | |
Poster Number: 2.377Session Date/Time: Sunday, | ||
Clinical Characteristics and Treatment Patterns inPatients with Dravet Syndrome and Lennox-GastautSyndrome Who Are Prescribed Cannabidiol (CBD) | Poster Number: 2.406Session Date/Time: Sunday, | |
Nurse-Reported Outcomes of Cannabidiol (CBD)Treatment in the Long-Term Care (LTC) Setting:Results From the | A. Fowler | Poster Number: 2.411Session Date/Time: Sunday, |
About
About Dravet SyndromeDravet 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).11Additionally, 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 SyndromeLennox-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 5.12More 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/Epidyolexis a prescription, plant-derived cannabis-based medicine administered as an oral solution which contains highly purified cannabidiol (CBD). Cannabidiol, the active ingredient inEpidiolex, is a cannabinoid that naturally occurs in theCannabis sativaL. plant. The precise mechanisms by whichEpidiolexexerts its anticonvulsant effect in humans are unknown. Epidiolexwas approved by the
Important Safety Information & Indications
CONTRAINDICATION: HYPERSENSITIVITY
EPIDIOLEX (cannabidiol) oral solution is contraindicated in patients with a history of hypersensitivity to cannabidiol or any ingredients in the product.
WARNINGS & PRECAUTIONS
Hepatic Injury:EPIDIOLEX can cause dose-related transaminase elevations. Concomitant use of valproate and elevated transaminase levels at baseline increase this risk. Obtain transaminase and bilirubin levels prior to starting treatment, at 1, 3, and 6 months after initiation of treatment, and periodically thereafter, or as clinically indicated. Resolution of transaminase elevations occurred with discontinuation of EPIDIOLEX, reduction of EPIDIOLEX and/or concomitant valproate, or without dose reduction. For patients with elevated transaminase levels, consider dose reduction or discontinuation of EPIDIOLEX or concomitant medications known to affect the liver (e.g., valproate or clobazam). Dose adjustment and slower dose titration is recommended in patients with moderate or severe hepatic impairment. Consider not initiating EPIDIOLEX in patients with evidence of significant liver injury. There have been postmarketing reports of cholestatic or mixed patterns of liver injury. Elevated ammonia levels were reported in some patients with transaminase elevations; most taking concomitant valproate, clobazam, or both. Consider discontinuation or dose adjustment of valproate or clobazam if ammonia is elevated.
Somnolence and Sedation:EPIDIOLEX can cause somnolence and sedation that generally occurs early in treatment and may diminish over time; these effects occur more commonly in patients using clobazam and may be potentiated by other CNS depressants.
Suicidal Behavior and Ideation:Antiepileptic drugs (AEDs), including EPIDIOLEX, increase the risk of suicidal thoughts or behavior. Inform patients, caregivers, and families of the risk and advise them to monitor and report any signs of depression, suicidal thoughts or behavior, or unusual changes in mood or behavior. If these symptoms occur, consider if they are related to the AED or the underlying illness.
Withdrawal of Antiepileptic Drugs:As with most AEDs, EPIDIOLEX should generally be withdrawn gradually because of the risk of increased seizure frequency and status epilepticus.
ADVERSE REACTIONS:The most common adverse reactions in patients receiving EPIDIOLEX (≥10% and greater than placebo) include transaminase elevations; somnolence; decreased appetite; diarrhea; pyrexia; vomiting; fatigue, malaise, and asthenia; rash; insomnia, sleep disorder and poor-quality sleep; and infections. Hematologic abnormalities were also observed.
PREGNANCY:EPIDIOLEX should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. Encourage women who are taking EPIDIOLEX during pregnancy to enroll in the EPIDIOLEX Pregnancy Surveillance Program and the North American Antiepileptic Drug (NAAED) Pregnancy Registry.
DRUG INTERACTIONS:Strong inducers of CYP3A4 and CYP2C19 may affect EPIDIOLEX exposure. EPIDIOLEX may affect exposure to CYP2C19 substrates (e.g., clobazam, diazepam, stiripentol), orally administered P-gp substrates, or other substrates (see full Prescribing Information). Consider dose reduction of orally administered everolimus, with appropriate therapeutic drug monitoring, when everolimus is combined with EPIDIOLEX. A lower starting dose of everolimus is recommended when added to EPIDIOLEX therapy. Concomitant use of EPIDIOLEX and valproate increases the incidence of liver enzyme elevations. Pneumonia was observed more frequently with concomitant use of EPIDIOLEX and clobazam. Dosage adjustment of EPIDIOLEX or other concomitant medications may be necessary.
INDICATIONS:EPIDIOLEX (cannabidiol) oral solution is indicated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC) in patients 1 year of age and older.
Please read the EPIDIOLEX full Prescribing Information for additional important informationhere.
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