Data from nine abstracts to be presented,
including real-world outcomes of Epidiolex®
(cannabidiol) in treatment-resistant focal epilepsies
Presentations include data on U.S.
caregiver-reported seizure and non-seizure outcomes in patients
with tuberous sclerosis complex prescribed Epidiolex
DUBLIN, Dec. 1, 2023
/PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today
announced that nine company-sponsored presentations, including five
late-breaking abstracts, will be shared at the 2023 American
Epilepsy Society (AES) annual meeting, being held December 1-5 in Orlando, Florida.
Late-breaking presentations include a post-hoc analysis
examining real-world outcomes of Epidiolex®
(cannabidiol) in treatment-resistant focal epilepsies within the
Expanded Access Program (EAP), as well as a post-hoc analysis of
the GWPCARE6 Open-Label Extension (OLE) trial evaluating the
effectiveness of Epidiolex as add-on therapy against focal
seizures in tuberous sclerosis complex (TSC). Two additional
late-breaking abstracts will feature interim results from the
BECOME-TSC (BEhavior, COgnition, and More with Epidiolex)
Survey of caregivers of patients with TSC. These abstracts will
report seizure and non-seizure outcomes with real-world use of
Epidiolex in TSC and further expand the results of the
original BECOME survey conducted among caregivers of people with
Dravet syndrome (DS) or Lennox-Gastaut syndrome (LGS) that were
presented at the 2022 AES meeting.
"Multiple presentations at the 2023 AES meeting continue to
build the scientific evidence in support of Epidiolex and
improve understanding around the treatment's full impact," said
Kelvin Tan, MBBCh, MRCPCH, senior
vice president and chief medical officer of Jazz Pharmaceuticals.
"In addition to seizure outcomes, the BECOME studies are also
designed to assess non-seizure benefits of Epidiolex, such
as cognition and executive function, emotional and social function,
and language and communication. We are determined to not only
address seizure outcomes for patients living with rare epilepsies,
but also improve the day-to-day lives for both patients and their
loved ones."
Data highlights at the 2023 AES annual meeting include:
- A post-hoc analysis of patients diagnosed with
treatment-resistant focal epilepsy within the EAP that demonstrated
Epidiolex was associated with a sustained reduction in
focal-onset seizures through 144 weeks. Notably, a second post-hoc
analysis investigated the effectiveness and safety results of
Epidiolex in adult patients from the EAP which found similar
outcomes among patients with treatment-resistant epilepsies,
including focal seizures. The EAP was an initiative launched in
2014 to provide add-on Epidiolex to patients with
treatment-resistant epilepsy at 35 U.S. epilepsy centers.
- A post-hoc analysis of the GWPCARE6 OLE trial evaluating the
effectiveness of Epidiolex as add-on therapy against focal
seizures in TSC. At least 50% reduction was reported by the
majority of patients across focal seizure types through 144 weeks.
Additionally, responder rates for focal seizure subtypes were
consistent with the overall focal seizure responder rates, and the
safety profile observed in the analysis was consistent with that
observed in its overall clinical development program.
- Interim results of the BECOME-TSC survey, in which the majority
of caregivers reported patient improvements in overall seizure
frequency and severity, as well as improvements in patients'
cognition, emotional functioning, and communication domains.
The 2023 AES 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
|
Lead
Author
|
Poster Number / Date
& Time (ET)
|
Late-Breaking
Abstracts
|
Real-World Outcomes of
Cannabidiol (CBD) in Treatment-Resistant Focal Epilepsies:
Experience From the Expanded Access Program (EAP)
|
A. D. Patel
|
Poster Number:
2.493
Session Date/Time:
Sunday, December 3rd, 12:00-2:00 PM
|
Long-Term Effectiveness
of Cannabidiol Against Focal Seizures in Tuberous Sclerosis
Complex: Results From the GWPCARE6 Open-Label Extension
Trial
|
J.Y. Wu
|
Poster Number:
2.498
Session Date/Time:
Sunday, December 3rd, 12:00-2:00 PM
|
Caregiver-Reported
Nonseizure Outcomes with Real-World Use of Cannabidiol (CBD) in
Tuberous Sclerosis Complex (TSC): Interim Results From the
BECOME-TSC Survey
|
S. Wilson
|
Poster Number:
2.499
Session Date/Time:
Sunday, December 3rd, 12:00-2:00 PM
|
Caregiver-Reported
Seizure Outcomes with Real-World Use of Cannabidiol (CBD) in
Tuberous Sclerosis Complex (TSC): Interim Results from the
BECOME-TSC Survey
|
M.K. Koenig
|
Poster Number:
2.503
Session Date/Time:
Sunday, December 3rd, 12:00-2:00 PM
|
Cannabidiol and Sodium
Valproate Demonstrate Pharmacodynamic Synergism in an Acute Mouse
Model of Generalised Seizures
|
R.R. Rohini
|
Poster Number:
3.479
Session Date/Time:
Monday, December 4th,
12:00-1:45 PM
|
Abstracts
|
Efficacy and Safety of
Cannabidiol (CBD) for Seizures Associated With Tuberous Sclerosis
Complex (TSC) in Pediatric and Adult Patients From GWPCARE6: A
Phase 3 Trial With Open-Label Extension (OLE)
|
E.A. Thiele
|
Poster Number:
3.266
Session Date/Time:
Monday, December 4th, 12:00-1:45 PM
|
Change in Antiseizure
and Anxiolytic Medications Pre- and Post-Cannabidiol
Initiation
|
G. Fang
|
Poster Number:
3.287
Session Date/Time:
Monday, December 4th, 12:00-1:45 PM
|
Long-term Effectiveness
of Cannabidiol (CBD) Against Focal-Onset Seizures in
Treatment-Resistant Epilepsies (TRE)
|
Y.D. Park
|
Poster Number:
3.291
Session Date/Time:
Monday, December 4th, 12:00-1:45 PM
|
Real-world Safety and
Effectiveness of Cannabidiol (CBD) in Adults With
Treatment-Resistant Epilepsies: Long-term Results From the United
States Expanded Access Program (EAP)
|
J.P.
Szaflarski
|
Poster Number:
3.293
Session Date/Time:
Monday, December 4th, 12:00-1:45 PM
|
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,
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, and the Food &
Nutrition Services of the Israel Ministry of Health for use in
Israel, is an oral solution which
contains highly purified cannabidiol. 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 received Orphan Drug Designation from the
U.S. FDA for the treatment of seizures associated with LGS, DS and
TSC. 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. Epidyolex has received Orphan Drug Designation from
the European Medicines Agency (EMA) for the treatment of seizures
associated LGS, DS and TSC. A Phase 3 study is ongoing in
Japan evaluating cannabidiol in
patients with LGS, DS and TSC.
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
Hepatocellular Injury:
EPIDIOLEX can cause
dose-related transaminase elevations. Concomitant use of valproate
and elevated transaminase levels at baseline increase this risk.
Transaminase and bilirubin levels should be obtained prior to
starting treatment, at one, three, and six 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.
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 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 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 refer to the EPIDIOLEX full Prescribing Information
for additional important information here.
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. Please visit www.jazzpharmaceuticals.com for more
information.
Media Contact:
Kristin
Bhavnani
Head of Global Strategic Brand Engagement
Jazz Pharmaceuticals plc
CorporateAffairsMediaInfo@jazzpharma.com
Ireland +353 1 637 2141
U.S. +1 215 867 4948
Investors:
Andrea N.
Flynn, Ph.D.
Vice President, Head, Investor Relations
Jazz Pharmaceuticals plc
InvestorInfo@jazzpharma.com
Ireland +353 1 634 3211
U.S. +1 650 496 2717
References
1 TS Alliance. What is TSC?
https://www.tsalliance.org/about-tsc/what-is-tsc/. Accessed
November 2023.
2 National Institute of Neurological Disorders and
Stroke. Tuberous Sclerosis Complex.
https://www.ninds.nih.gov/health-information/disorders/tuberous-sclerosis-complex.
Accessed November 2023.
3 TS Alliance. What is tuberous sclerosis complex?
https://www.tsalliance.org/. Accessed November 2023.
4 Kingswood JC, d'Augères GB, Belousova E, et al.
TuberOus SClerosis registry to increase disease Awareness (TOSCA) -
baseline data on 2093 patients. 2017;12(1):2.
5 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.
6 Tuberous Sclerosis Alliance. Diagnosis, Surveillance
and Management for Healthcare
Professionals. https://www.tscalliance.org/healthcare-professionals/diagnosis/.
Accessed October 2023.
7 Jeong A, Wong M. Systemic disease manifestations
associated with epilepsy in tuberous sclerosis complex.
Epilepsia. 2016;57(9):1443-1449.
8 Chu-Shore CJ, Major P, Camposano S, Muzykewicz D,
Thiele EA. The natural history of epilepsy in tuberous sclerosis
complex. Epilepsia. 2010;51(7):1236-1241.
9 Kwan P, Brodie MJ. Early identification of
refractory epilepsy. N Engl J Med. 2000;342(5):314-319.
10 French JA. Refractory epilepsy: clinical
overview. Epilepsia. 2007;48 Suppl 1:3-7.
11 Epilepsy Foundation. Dravet Syndrome.
https://www.epilepsy.com/what-is-epilepsy/syndromes/dravet-syndrome.
Accessed November 2023.
12 National Organization for Rare Disorders,
Inc. Lennox-Gastaut Syndrome: Signs & Symptoms.
https://rarediseases.org/rare-diseases/lennox-gastaut-syndrome/.
Accessed November 2023.
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