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 Pharmaceuticals (JAZZ) presented nine company-sponsored Epidiolex® (cannabidiol) posters at the American Epilepsy Society 2024 Annual Meeting. The presentations include real-world evidence from multiple studies showcasing the drug's effectiveness in treating rare epilepsies.
Key findings include: improvements in behavioral symptoms from the EpiCom trial; positive outcomes from the BECOME-TSC survey where 89% of caregivers plan to continue treatment; and results from the BECOME-LTC survey showing 85% of nurses reported reduced seizure frequency, with 49% noting over 50% reduction. The data also demonstrated Epidiolex's effectiveness in treating tuberous sclerosis complex (TSC), with median seizure reductions of 51%-87% in focal seizures and 44%-87% in total seizures.
Jazz Pharmaceuticals (JAZZ) ha presentato nove poster sponsorizzati dall'azienda riguardanti Epidiolex® (cannabidiolo) durante l'Annual Meeting della American Epilepsy Society 2024. Le presentazioni includono evidenze della vita reale da diversi studi che mostrano l'efficacia del farmaco nel trattamento delle epilessie rare.
I risultati principali includono: miglioramenti nei sintomi comportamentali dal trial EpiCom; esiti positivi dal sondaggio BECOME-TSC in cui l'89% dei caregiver intende continuare il trattamento; e risultati dal sondaggio BECOME-LTC che mostrano come l'85% degli infermieri abbia riferito una riduzione della frequenza delle crisi, con il 49% che ha notato una riduzione superiore al 50%. I dati hanno anche dimostrato l'efficacia di Epidiolex nel trattamento della complessità della tuberosa sclerosi (TSC), con riduzioni medie delle crisi del 51%-87% nelle crisi parziali e del 44%-87% nelle crisi totali.
Jazz Pharmaceuticals (JAZZ) presentó nueve carteles patrocinados por la empresa sobre Epidiolex® (cannabidiol) en la Reunión Anual de la Sociedad Americana de Epilepsia 2024. Las presentaciones incluyen evidencia del mundo real de varios estudios que demuestran la efectividad del fármaco en el tratamiento de epilepsias raras.
Los hallazgos clave incluyen: mejoras en los síntomas comportamentales del ensayo EpiCom; resultados positivos de la encuesta BECOME-TSC donde el 89% de los cuidadores planea continuar el tratamiento; y resultados de la encuesta BECOME-LTC que muestran que el 85% de las enfermeras informaron una reducción en la frecuencia de las crisis, con el 49% indicando una reducción de más del 50%. Los datos también demostraron la efectividad de Epidiolex en el tratamiento de la complejidad de la esclerosis tuberosa (TSC), con reducciones medianas de crisis del 51%-87% en las crisis focales y del 44%-87% en las crisis totales.
재즈 제약회사 (JAZZ)는 2024년 미국 간질 학회 연례 회의에서 에피디오렉스® (칸나비디올)에 대한 아홉 개의 회사 후원 포스터를 발표했습니다. 발표 내용은 이 약물이 희귀 간질 치료에 대한 효과를 보여주는 여러 연구의 실제 증거를 포함하고 있습니다.
주요 발견으로는: EpiCom 시험에서 행동 증상의 개선; BECOME-TSC 설문조사 결과 89%의 보호자가 치료를 계속할 계획이라고 응답; 그리고 BECOME-LTC 설문조사 결과 85%의 간호사가 발작 빈도의 감소를 보고했으며, 49%는 50% 이상의 감소를 나타냈습니다. 데이터 또한 에피디오렉스가 결절 경화증 복합체 (TSC) 치료에 효과적임을 보여주었으며, 초점성 발작에서 51%-87%의 중간 발작 감소와 전체 발작에서 44%-87%의 감소를 기록했습니다.
Jazz Pharmaceuticals (JAZZ) a présenté neuf posters parrainés par l'entreprise sur Epidiolex® (cannabidiol) lors de la Réunion Annuelle de la Société Américaine de l'Épilepsie 2024. Les présentations incluent des preuves du monde réel issues de plusieurs études montrant l'efficacité du médicament dans le traitement des épilepsies rares.
Les principaux résultats incluent : des améliorations des symptômes comportementaux provenant de l'essai EpiCom ; des résultats positifs de l'enquête BECOME-TSC où 89 % des aidants prévoient de poursuivre le traitement ; et des résultats de l'enquête BECOME-LTC montrant que 85 % des infirmières ont signalé une diminution de la fréquence des crises, dont 49 % notent une réduction de plus de 50 %. Les données ont également démontré l'efficacité d'Epidiolex dans le traitement du complexe de la sclérose tubéreuse (TSC), avec des réductions médianes des crises de 51 %-87 % dans les crises focales et de 44 %-87 % dans les crises totales.
Jazz Pharmaceuticals (JAZZ) hat auf dem Jahresmeeting der American Epilepsy Society 2024 neun unternehmenseigene Poster zu Epidiolex® (Cannabidiol) präsentiert. Die Präsentationen beinhalten reale Evidenz aus mehreren Studien, die die Wirksamkeit des Medikaments bei der Behandlung seltener Epilepsien zeigen.
Wichtige Ergebnisse sind: Verbesserungen in den Verhaltenssymptomen aus der EpiCom-Studie; positive Ergebnisse aus der BECOME-TSC-Umfrage, bei der 89% der Betreuer planten, die Behandlung fortzusetzen; und Ergebnisse aus der BECOME-LTC-Umfrage, die zeigen, dass 85% der Krankenschwestern eine reduzierte Anfallsfrequenz berichteten, wobei 49% eine Reduktion von über 50% angaben. Die Daten zeigten auch die Wirksamkeit von Epidiolex bei der Behandlung des tuberösen Sklerose-Komplexes (TSC), mit mittleren Anfallreduktionen von 51%-87% bei fokalen Anfällen und 44%-87% bei totalen Anfällen.
- 85% of nurses reported reduction in overall seizure frequency after Epidiolex initiation
- 89% of caregivers in BECOME-TSC study plan to continue Epidiolex treatment
- 51%-87% median reduction in focal seizures for TSC patients
- 44%-87% median reduction in total seizures for TSC patients
- None.
Insights
The new real-world evidence for Epidiolex demonstrates significant clinical value across multiple treatment-resistant epilepsy types. The 89% continuation rate reported in the BECOME-TSC study and 85% seizure reduction rate in the BECOME-LTC survey strongly validate the drug's effectiveness in real-world settings.
Most notably, the EpiCom trial data shows promising behavioral improvements beyond seizure control, addressing a critical unmet need in TSC patients. The subgroup analysis revealing 51-87% reduction in focal seizures for TSC patients reinforces Epidiolex's broad-spectrum efficacy profile.
These comprehensive datasets substantially strengthen Epidiolex's market position in rare epilepsies and could drive increased adoption among healthcare providers, particularly in long-term care facilities where the nurse-reported outcomes are especially compelling.
This robust clinical data presentation strengthens Jazz Pharmaceuticals' competitive position in the rare epilepsy market. The real-world evidence demonstrating both seizure control and behavioral benefits creates multiple value propositions for payers and healthcare providers.
The positive outcomes in long-term care settings are particularly significant for market expansion, as this represents a substantial untapped opportunity. The high continuation rates and strong efficacy data across multiple patient populations should support sustained revenue growth for Epidiolex, which has become a key growth driver for Jazz since its acquisition of GW Pharmaceuticals.
The expanded evidence base supporting benefits beyond seizure control could help defend against future competition and support premium pricing.
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 with Epidiolex) caregiver survey, which characterizes and quantifies seizure and non-seizure outcomes in patients with epilepsy and tuberous sclerosis complex (TSC) treated with Epidiolex. 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 with Epidiolex treatment. Further, the first presentation of data from the EpiCom trial, a prospective, interventional trial evaluating the impact of adjunctive Epidiolex on 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 of Epidiolex in the treatment of patients with rare epilepsies," said Sarah Akerman, MD, head of neuroscience global medical and scientific affairs of Jazz Pharmaceuticals. "These findings increase our understanding of Epidiolex's benefits beyond seizure control, addressing unmet needs across a range of epilepsy syndromes for people living with rare epilepsies and demonstrating reproducibility and consistency of effect across different populations."
Data highlights include:
- A prespecified three-month analysis of the EpiCom trial reporting TSC-associated neuropsychiatric disorders (TAND)-associated outcomes demonstrated improvements after initiating adjunctive Epidiolex treatment 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 continue Epidiolex treatment for their loved one. The most important stated reasons for continuing Epidiolex included 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 with Epidiolex in the Long-Term Care Setting) survey found that, among 102 nurses surveyed,
85% reported a reduction in overall frequency of any seizure type after Epidiolex initiation, with49% reporting a greater than50% 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-world Epidiolex in Epilepsy Context), a cross-sectional caregiver survey, demonstrated reduced caregivers' need for additional support of their dependents' physical, emotional, and behavioral care after Epidiolex initiation 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 with Epidiolex in the
U.S. Expanded Access Program, found Epidiolex has similar effectiveness in TSC and other focal epilepsies, regardless of focal epilepsy type, further reinforcing the clinical profile of Epidiolex as a broad-spectrum agent. In the TSC group, Epidiolex was associated with a median reduction from baseline of51% –87% in focal seizures and44% –87% in total seizures. In the non-TSC group, Epidiolex was also associated with a median reduction from baseline of46% –75% in focal and46% –74% in total seizures.
Epidiolex is 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 Jazz Pharmaceuticals' presentations follows below:
Presentation Title | Presenting Author | Poster Number / Date & Time (PT) |
Cannabidiol has antiseizure and antitumor effects in | A.R. Tee | Poster Number: 1.052 |
Caregiver-Reported Nonseizure Outcomes with | D. Samanta | Poster Number: 1.433 |
Caregiver-Reported Real-World Use of Cannabidiol | M. Faithe | Poster Number: 1.434 |
Caregiver-Reported Seizure Outcomes with Real- | D. Krueger | Poster Number: 1.435 |
Effects of Cannabidiol (CBD) on Audiogenic | C.L. Faingold | Poster Number: 2.354 |
Real-World Outcomes of Cannabidiol (CBD) in | E.A. Thiele | Poster Number: 2.369 |
Tuberous Sclerosis Complex (TSC)–Associated | A. van Eeghen | Poster Number: 2.377 |
Clinical Characteristics and Treatment Patterns in | S. Kothare | Poster Number: 2.406 |
Nurse-Reported Outcomes of Cannabidiol (CBD) | A. Fowler | Poster Number: 2.411 |
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 percent of patients with TSC and may progress to become intractable to medication.4,6,7 More than 60 percent 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 percent 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
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 (≥
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 information here.
About Jazz Pharmaceuticals
Jazz Pharmaceuticals plc (Nasdaq: JAZZ) is a global biopharma 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, including leading therapies for sleep disorders and epilepsy, and a growing portfolio of cancer treatments. Our patient-focused and science-driven approach powers pioneering research and development advancements across our robust pipeline of innovative therapeutics in oncology and neuroscience. Jazz is headquartered in Dublin, Ireland with research and development laboratories, manufacturing facilities and employees in multiple countries committed to serving patients worldwide. Please visit www.jazzpharmaceuticals.com for more information.
Contacts:
Media:
Kristin Bhavnani
Head of Global Corporate Communications
Jazz Pharmaceuticals plc
CorporateAffairsMediaInfo@jazzpharma.com
Investors:
Jeff Macdonald
Executive Director, Investor Relations
Jazz Pharmaceuticals plc
InvestorInfo@jazzpharma.com
References: |
_____________ |
1 TSC Alliance. What is TSC? https://www.tscalliance.org/understanding-tsc/what-is-tsc/. Accessed December 2024. |
View original content to download multimedia:https://www.prnewswire.com/news-releases/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-302324979.html
SOURCE Jazz Pharmaceuticals plc
FAQ
What were the key findings of JAZZ's Epidiolex treatment in the BECOME-LTC survey?
What was the seizure reduction rate for TSC patients using Epidiolex (JAZZ)?
What percentage of caregivers plan to continue Epidiolex treatment according to JAZZ's BECOME-TSC study?
