Jazz Pharmaceuticals Provides Update on Cannabidiol Oral Solution Phase 3 Trial in Japan in Treatment-Resistant Epilepsies

Rhea-AI Summary

Jazz Pharmaceuticals (Nasdaq: JAZZ) announced top-line results from a Phase 3 trial in Japan evaluating cannabidiol oral solution (Epidiolex/Epidyolex) for treating seizures associated with Lennox-Gastaut syndrome, Dravet syndrome, and tuberous sclerosis complex. The trial did not meet its primary efficacy endpoint of reducing seizure frequency compared to baseline in Japanese pediatric patients. However, numeric improvements were observed in primary and secondary endpoints, and no new safety signals were identified.

Despite not meeting the primary endpoint, Jazz remains confident in Epidyolex's clinical profile, citing its success in five previous Phase 3 trials involving over 900 patients globally. The company plans to continue collecting data and engage with Japanese regulatory authorities regarding a potential new drug application.

Positive

• Numeric improvements observed in primary and secondary endpoints
• No new safety signals identified in the trial
• Confidence in overall clinical profile of Epidyolex based on previous global trials
• Continuing to collect data in Japanese patients for potential new drug application

Negative

• Phase 3 trial did not meet primary efficacy endpoint in Japanese pediatric patients
• Potential delay in Japanese market entry due to trial results

Insights

Medical Research Analyst

The Phase 3 trial results for cannabidiol oral solution in Japan are mixed. While the primary efficacy endpoint wasn't met, there were numeric improvements in primary and secondary endpoints. This suggests potential benefits, albeit not statistically significant. The safety profile aligns with previous trials, which is reassuring.

The company's decision to continue data collection and engage with regulators indicates confidence in the drug's potential. However, the failure to meet the primary endpoint could pose challenges for regulatory approval. The $1.7 billion global sales of Epidiolex in 2022 underscores the drug's importance to Jazz's portfolio, making the Japanese market important for expansion.

Investors should monitor upcoming regulatory discussions and additional data from the ongoing trial, as these will be critical in determining the drug's future in Japan.

Financial Analyst

The trial results present a mixed outlook for Jazz Pharmaceuticals. While not meeting the primary endpoint is disappointing, the company's commitment to the Japanese market is evident. The global success of Epidiolex/Epidyolex, with $1.7 billion in 2022 sales, highlights its significance to Jazz's portfolio.

Investors should consider the potential impact on future revenue projections if Japanese approval is delayed or denied. However, the company's diverse portfolio and global presence provide some cushion. The stock may experience short-term volatility as the market digests this news.

Long-term prospects remain tied to the broader epilepsy market and Jazz's ability to navigate regulatory challenges in Japan. Keep an eye on upcoming catalysts, including regulatory discussions and additional trial data.

Neurology Specialist

The trial results, while not meeting statistical significance, show promising trends in seizure reduction. This is particularly important given the treatment-resistant nature of LGS, DS and TSC. The safety profile consistency with previous trials is reassuring for long-term use.

The unmet need in Japan for effective epilepsy treatments cannot be overstated. Even modest improvements can significantly impact patients' quality of life. The company's commitment to further data collection and regulatory engagement is commendable.

From a clinical perspective, the numeric improvements observed warrant further investigation. It's possible that longer treatment duration or subgroup analyses might reveal more pronounced benefits. The ongoing trial data will be important in fully understanding cannabidiol's potential in the Japanese population.

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

FAQ

What were the results of Jazz Pharmaceuticals' Phase 3 trial for cannabidiol oral solution in Japan?

The Phase 3 trial in Japan for cannabidiol oral solution (Epidiolex/Epidyolex) did not meet its primary efficacy endpoint of reducing seizure frequency in Japanese pediatric patients with treatment-resistant epilepsies. However, numeric improvements were observed in primary and secondary endpoints, and no new safety signals were identified.

Will Jazz Pharmaceuticals (JAZZ) still pursue approval for Epidyolex in Japan despite the trial results?

Yes, Jazz Pharmaceuticals plans to continue collecting data in Japanese patients and engage with regulatory authorities in Japan regarding a potential new drug application (JNDA) for Epidyolex, despite the Phase 3 trial not meeting its primary endpoint.

What epilepsy conditions was the Jazz Pharmaceuticals (JAZZ) Phase 3 trial in Japan targeting?

The Phase 3 trial in Japan was evaluating cannabidiol oral solution for seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), and tuberous sclerosis complex (TSC) in Japanese pediatric patients.

How many patients were involved in Jazz Pharmaceuticals' (JAZZ) Phase 3 trial for Epidyolex in Japan?

The Phase 3 trial in Japan included 62 patients aged 1 to 18 years old, evaluating the efficacy and safety of cannabidiol oral solution for treatment-resistant epilepsies.