DUBLIN, Aug. 22,
2024 /PRNewswire/ -- Jazz Pharmaceuticals plc
(Nasdaq: JAZZ) today announced top-line results from the Phase 3
open-label, single-arm trial in Japan evaluating the safety and efficacy of
cannabidiol oral solution (marketed as
Epidiolex®/Epidyolex® globally) as an
adjunctive treatment for seizures associated with Lennox-Gastaut
syndrome (LGS), Dravet syndrome (DS) or tuberous sclerosis complex
(TSC). The trial did not meet the primary efficacy endpoint of a
pre-specified percentage change in indication-associated seizure
frequency during the treatment period (up to 16 weeks) compared to
baseline in Japanese pediatric patients; however, numeric
improvements were observed in the primary and several secondary
endpoints. No new safety signals were observed in the trial.
"We are confident in the overall clinical profile of
Epidyolex, which has been established in five Phase 3
clinical trials in more than 900 patients. We believe the totality
of the Epidyolex global data, including the findings from
this trial, supports advancement of the program in Japan," said Rob
Iannone, M.D., M.S.C.E., executive vice president, global
head of research and development of Jazz Pharmaceuticals. "We
are continuing to collect data in Japanese patients and plan to
engage with regulatory authorities in Japan regarding a potential new drug
application (JNDA). We recognize the significant unmet need for
patients in Japan living with rare
epilepsies and thank the investigators, patients and caregivers who
are involved in this trial."
About the Phase 3 Trial
The Phase 3 open-label,
single-arm clinical trial investigates the safety and efficacy of
cannabidiol oral solution (GWP42003-P) for the treatment of
seizures associated with LGS, DS or TSC in Japanese pediatric
patients. The trial includes a pre-specified primary efficacy
outcome measuring the percentage change in indication-associated
seizure frequency during the treatment period (up to 16 weeks)
compared to baseline in 62 patients ≥ 1 to ≤ 18 years of age. The
trial design includes a treatment period consisting of an initial
2-week titration period followed by a 14-week maintenance period;
and a period evaluating safety for up to 52 weeks of treatment. The
trial remains ongoing to collect efficacy and safety data in
pediatric and adult Japanese patients.
About Cannabidiol
Cannabidiol, 100 mg/mL oral
solution, a prescription, plant-derived cannabis-based medicine
approved by the U.S. Food and Drug Administration (FDA) for use in
the U.S., and the European Medicines Agency (EMA) for use in the
European Union, is an oral solution which contains highly purified
cannabidiol (CBD). In the U.S., cannabidiol, under the tradename
Epidiolex, is indicated for the treatment of seizures
associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome or
tuberous sclerosis complex (TSC) in patients one year of age and
older. Cannabidiol has also received approval in the European
Union, under the tradename Epidyolex, for adjunctive use in
conjunction with clobazam to treat seizures associated with LGS and
Dravet syndrome in patients two years and older, and for adjunctive
use to treat seizures associated with TSC, in patients two years of
age and older.
About Jazz Pharmaceuticals
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, 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.
Caution Concerning Forward-Looking Statements
This
press release contains forward-looking statements, including, but
not limited to, statements related to potential timing of data
availability and regulatory engagement related to the Phase 3
clinical trial in Japan and other
statements that are not historical facts. These forward-looking
statements are based on Jazz Pharmaceuticals' current plans,
objectives, estimates, expectations and intentions and inherently
involve significant risks and uncertainties. Actual results and the
timing of events could differ materially from those anticipated in
such forward-looking statements as a result of these risks and
uncertainties, which include, without limitation, risks and
uncertainties associated with pharmaceutical product development,
and other risks and uncertainties affecting Jazz Pharmaceuticals
and its development programs, including those described from time
to time under the caption "Risk Factors" and elsewhere in Jazz
Pharmaceuticals plc's Securities and Exchange Commission filings
and reports (Commission File No. 001-33500), including Jazz
Pharmaceuticals' Annual Report on Form 10-K for the year ended
December 31, 2023, as supplemented by
our Quarterly Report on Form 10-Q for the quarter ended
March 31, 2024, and future filings
and reports by Jazz Pharmaceuticals. Other risks and uncertainties
of which Jazz Pharmaceuticals is not currently aware may also
affect Jazz Pharmaceuticals' forward-looking statements and may
cause actual results and the timing of events to differ materially
from those anticipated. The forward-looking statements herein are
made only as of the date hereof or as of the dates indicated in the
forward-looking statements, even if they are subsequently made
available by Jazz Pharmaceuticals on its website or otherwise. Jazz
Pharmaceuticals undertakes no obligation to update or supplement
any forward-looking statements to reflect actual results, new
information, future events, changes in its expectations or other
circumstances that exist after the date as of which the
forward-looking statements were made.
Contacts:
Media:
Kristin
Bhavnani
Head of Global Corporate Communications
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
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SOURCE Jazz Pharmaceuticals plc