Jazz Pharmaceuticals plc (Jazz Pharmaceuticals) operates as biopharmaceutical company.
The company is dedicated to developing life-changing medicines for people with serious diseases - often with limited or no therapeutic options. The company has a diverse portfolio of marketed medicines, including leading therapies for sleep disorders and epilepsy, and a growing portfolio of cancer treatments. The company’s patient-focused and science-driven approach powers pioneering research and development...
Jazz Pharmaceuticals plc (Jazz Pharmaceuticals) operates as biopharmaceutical company.
The company is dedicated to developing life-changing medicines for people with serious diseases - often with limited or no therapeutic options. The company has a diverse portfolio of marketed medicines, including leading therapies for sleep disorders and epilepsy, and a growing portfolio of cancer treatments. The company’s patient-focused and science-driven approach powers pioneering research and development advancements across its robust pipeline of innovative therapeutics in oncology and neuroscience.
The company's strategy for growth is rooted in executing commercial launches and ongoing commercialization initiatives, advancing robust research and development (R&D) programs, and delivering impactful clinical results, effectively deploying capital to strengthen the prospects of achieving its short- and long-term goals through strategic corporate development, and delivering strong financial performance. The company focuses on patient populations with high unmet needs.
The company’s lead marketed products, listed below are approved in countries around the world to improve patient care.
Neuroscience
Xywav (calcium, magnesium, potassium, and sodium oxybates) oral solution is a product approved by the U.S. Food and Drug Administration (FDA) in July 2020 and launched in the U.S. in November 2020 for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients seven years of age and older with narcolepsy. It was also approved by the FDA in August 2021 for the treatment of idiopathic hypersomnia (IH) in adults and launched in the U.S. in November 2021. Xywav contains 92% less sodium than Xyrem. Additionally, Xywav is approved in Canada for the treatment of cataplexy in patients with narcolepsy.
Epidiolex (cannabidiol) oral solution is a product approved by the FDA and launched in the U.S. in 2018 by GW. It 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 or older. In the EU and Great Britain, (where it is marketed as Epidyolex), it is approved for adjunctive treatment of seizures associated with LGS or DS, in conjunction with clobazam (EU and Great Britain only), in patients two years of age and older, and for adjunctive treatment of seizures associated with TSC in patients two years of age and older.
Oncology
Rylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn) is a product approved by the FDA in June 2021 and launched in the U.S. in July 2021 for use as a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LBL) in adults and pediatric patients aged one month or older who have developed hypersensitivity to E. coli-derived asparaginase. In September 2023, the European Commission granted marketing authorization under the trade name Enrylaze. This therapy is also approved in markets, including Great Britain, Canada, and Switzerland.
Zepzelca (lurbinectedin), a product approved by FDA in June 2020 under FDA's accelerated approval pathway and launched in the U.S. in July 2020 for the treatment of adult patients with metastatic SCLC with disease progression on or after platinum-based chemotherapy; in Canada, Zepzelca received conditional approval in September 2021 for the treatment of adults with Stage III or metastatic SCLC, who have progressed on or after platinum-containing therapy.
Ziihera (zanidatamab-hrii) is a product that the company received FDA approval for in November 2024 under the FDA’s accelerated approval pathway. It was launched in the U.S. in December 2024 for the treatment of adults with previously treated, unresectable, or metastatic HER2-positive (IHC 3+) BTC, as detected by an FDA-approved test.
In 2024, consistent with the company’s strategy, it continued to focus on research and development activities within its neuroscience and oncology therapeutic areas.
Commercialized Products
Neuroscience
Sleep Products: The company is the global leader in the development and commercialization of oxybate therapy for patients with sleep disorders. Xyrem was approved by FDA in 2002 for treating EDS and cataplexy in narcolepsy. In 2020, the company received FDA approval for Xywav for the treatment of cataplexy or EDS, in patients seven years of age and older with narcolepsy. In 2021, Xywav became the first and only therapy approved by FDA for the treatment of IH in adults. Xywav is an oxybate therapy that contains 92% less sodium than Xyrem. Xywav has become a standard of care for patients with narcolepsy and IH.
Xywav: In July 2020 FDA approved Xywav for the treatment of cataplexy and EDS in patients seven years of age and older with narcolepsy. Narcolepsy is a chronic, debilitating neurological disorder characterized by EDS and the inability to regulate sleep-wake cycles normally. Since there is no cure for narcolepsy and long-term disease management is needed. There are five primary symptoms of narcolepsy, including EDS, cataplexy, disrupted nighttime sleep, sleep-related hallucinations, and sleep paralysis. While patients with narcolepsy may not experience all five symptoms, EDS, an essential symptom of narcolepsy, is present in all narcolepsy patients and is characterized by chronic, pervasive sleepiness, as well as sudden irresistible and overwhelming urges to sleep (inadvertent naps and sleep attacks). Narcolepsy may affect many areas of life, including limiting a patient’s education and employment opportunities, and may lead to difficulties at work, school, or in daily life activities, such as driving, operating machinery, or caring for children. Patients with narcolepsy may also suffer from significant medical comorbidities in both adult and pediatric populations, including increased psychiatric comorbidity (including anxiety disorders and mood disorders), intentional self-injury, attempted suicide, suicide-related mortality, other sleep disorders (such as periodic limb movement disorder), cardiovascular disorders (such as hypertension, coronary atherosclerosis, acute myocardial infarction, and congestive heart failure), and metabolic disorders (such as obesity, metabolic syndrome, and diabetes).
Cataplexy, the sudden loss of muscle tone with retained consciousness, can be one of the most debilitating symptoms of narcolepsy. Cataplexy is present in approximately 70% of patients with narcolepsy. Cataplexy can range from slight weakness or a drooping of facial muscles to the complete loss of muscle tone resulting in postural collapse. It may also impair a patient’s vision or speech. Cataplexy is often triggered by strong emotions, such as laughter, anger or surprise. Cataplexy can severely impair a patient’s quality of life and ability to function.
The company’s commercial efforts are focused on educating patients and physicians about the lifelong impact of high sodium intake, and how the use of Xywav enables them to address what is a modifiable risk factor. When patients transition from Xyrem to Xywav, Xywav treatment is initiated at the same dose and regimen (gram for gram) and titrated as needed based on efficacy and tolerability.
The company views the adoption of Xywav in narcolepsy as a positive indication that physicians and patients appreciate the benefits of a low-sodium oxybate option. In June 2021, FDA recognized seven years of ODE for Xywav in narcolepsy through July 21, 2027 (which was subsequently extended to January 21, 2028). Nevertheless, Lumryz, a fixed-dose, high-sodium oxybate, was approved by FDA on May 1, 2023 for the treatment of cataplexy or EDS in adults with narcolepsy and was launched in the U.S. market in June 2023. FDA continues to recognize seven years of ODE for Xywav in narcolepsy. In connection with granting ODE for Xywav, FDA stated that ‘Xywav is clinically superior to Xyrem by means of greater safety because Xywav provides a greatly reduced chronic sodium burden compared to Xyrem.’ FDA's summary also stated that ‘the differences in the sodium content of the two products at the recommended doses will be clinically meaningful in reducing cardiovascular morbidity in a substantial proportion of patients for whom the drug is indicated.’ FDA has also recognized that the difference in sodium content between Xywav and Lumryz is likely to be clinically meaningful in all patients with narcolepsy and that Xywav is safer than Lumryz in all such patients. Lumryz has the same sodium content as Xyrem. Xywav is the only approved oxybate therapy that does not carry a warning and precaution related to high sodium intake.
On August 12, 2021, FDA approved Xywav for the treatment of IH in adults. Xywav remains the first and only FDA-approved therapy to treat IH. The company initiated the U.S. commercial launch of Xywav for the treatment of IH in adults on November 1, 2021. In January 2022, FDA recognized seven years of ODE for Xywav in IH through August 12, 2028. IH is a debilitating neurologic sleep disorder characterized by chronic EDS (the inability to stay awake and alert during the day resulting in the irrepressible need to sleep or unplanned lapses into sleep or drowsiness), severe sleep inertia, and prolonged and non-restorative nighttime sleep. Although there are overlapping clinical features with other conditions, including narcolepsy, IH has its own specific diagnostic criteria. IH can significantly affect social, educational and occupational functioning.
The company has agreements in place for Xywav with all three major pharmacy benefit managers, or PBMs, in the U.S. To date the company had entered into agreements with various entities and had achieved benefit coverage for Xywav in both narcolepsy and IH indications for approximately 90% of commercial lives.
The company has seen adoption of Xywav in narcolepsy since its launch in 2020 and increasing adoption in IH since its launch in 2021.
Xyrem: Xyrem was approved in the U.S. for the treatment of cataplexy in adult patients with narcolepsy in 2002 and was approved for the treatment of EDS in adult patients with narcolepsy in 2005. In 2018, Xyrem was also approved in the U.S. for the treatment of cataplexy or EDS in pediatric patients seven years of age and older with narcolepsy.
Xywav and Xyrem REMS: The company’s marketing, sales and distribution of Xywav and Xyrem in the U.S. are subject to a risk evaluation and mitigation strategy, or REMS, which is required by FDA to mitigate the risks of serious adverse outcomes resulting from inappropriate prescribing, abuse, misuse and diversion of Xywav and Xyrem. The Xywav and Xyrem REMS has the same requirements for both products. Under this REMS, all of the Xywav and Xyrem sold in the U.S. must be dispensed and shipped directly to patients or caregivers through a central pharmacy.
The company has exclusive agreements with Express Scripts Specialty Distribution Services, Inc., or ESSDS, the central pharmacy for Xywav and Xyrem, to distribute Xywav and Xyrem in the U.S. and provide patient support services related to Xyrem since 2002. In 2022, the company entered into new agreements with ESSDS with a two-year term. The company’s agreements with ESSDS, which expire on December 31, 2025, may be terminated by either party at any time without cause on 180 days’ prior written notice to the other party.
Epidiolex: The company acquired Epidiolex (Epidyolex outside the U.S.) in May 2021 as part of its GW Acquisition, which expanded its growing neuroscience business with a global, high-growth childhood-onset epilepsy franchise. Epidiolex was approved in the U.S. in June 2018 for the treatment of seizures associated with two rare and severe forms of epilepsy, Lennox-Gastaut syndrome (LGS) and Dravet syndrome (DS), in patients two years of age and older, and subsequently approved in July 2020 for the treatment of seizures associated with TSC in patients one year of age and older. In September 2019, the EC granted marketing authorization under the trade name Epidyolex for use as adjunctive therapy of seizures associated with LGS or DS, in conjunction with clobazam, for patients two years of age and older. Epidyolex is launched in all five key European markets: United Kingdom, Germany, Italy, Spain and France. The clobazam restriction is limited to the EU and Great Britain. Epidyolex was also approved for adjunctive therapy in seizures associated with TSC for patients 2 years of age and older in the European Union (EU) in April 2021 and Great Britain in August 2021 and is approved for this indication in other markets. Epidiolex/Epidyolex is also approved in markets including Switzerland, Israel, Canada, and Australia.
LGS and DS are severe childhood-onset, drug-resistant epilepsy syndromes. LGS and DS affect approximately 35,000-50,000 and approximately 10,000 individuals in the U.S., respectively. TSC is a rare genetic disorder that causes non-malignant tumors to form in many different organs and is a leading cause of genetic epilepsy. TSC affects approximately 50,000 individuals in the U.S. Epidiolex has received ODE to treat seizures associated with LGS and DS through 2025 and TSC through 2027.
Oncology
Rylaze: Rylaze was approved by FDA in June 2021 under the Real-Time Oncology Review, or RTOR, program, and was launched in the U.S. in July 2021, for use as a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia, or ALL, and lymphoblastic lymphoma, or LBL, in pediatric and adult patients one month and older who have developed hypersensitivity to E. coli-derived asparaginase. Rylaze is the only recombinant Erwinia asparaginase manufactured product that maintains a clinically meaningful level of serum asparaginase activity throughout the entire intended course of treatment. The company developed Rylaze to address the needs of patients and health care providers for an Erwinia asparaginase with reliable supply.
The initial approved recommended dosage of Rylaze was for an intramuscular, or IM, administration of 25 mg/m2 every 48 hours. In November 2022, FDA approved a supplemental Biologics License Application, or sBLA, for a Monday/Wednesday/Friday 25/25/50 mg/m2 IM dosing schedule. In September 2023, the EC granted marketing authorization for JZP458 (Rylaze) under the trade name Enrylaze and the company has initiated a rolling launch in Europe.
Zepzelca: The company acquired U.S. development and commercialization rights to Zepzelca in early 2020, and launched six months thereafter, with an indication for the treatment of patients with SCLC with disease progression on or after platinum-based chemotherapy. Zepzelca is an alkylating drug that binds guanine residues within DNA. This triggers a cascade of events that can affect the activity of DNA binding proteins, including some transcription factors, and DNA repair pathways, resulting in disruption of the cell cycle and eventual cell death. The company’s education and promotional efforts are focused on SCLC-treating physicians. The company is continuing to raise awareness of Zepzelca across academic and community cancer centers.
The company’s exclusive U.S. development and commercialization rights to Zepzelca were acquired through an exclusive license agreement it entered into with Pharma Mar, S.A., or PharmaMar, in 2019. In 2020, the company entered into an amendment to the license agreement with PharmaMar to expand the company’s exclusive license to include rights to develop and commercialize Zepzelca in Canada.
Zepzelca was granted orphan drug designation for adults with metastatic small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy by the FDA in August 2018. In December 2019, PharmaMar submitted an NDA to the FDA for accelerated approval of Zepzelca for relapsed SCLC based on data from a Phase 2 trial, and in February 2020, the FDA accepted the NDA for filing with priority review. In June 2020, the FDA granted accelerated approval of Zepzelca for the treatment of adult patients with metastatic SCLC with disease progression on or after platinum-based chemotherapy. Zepzelca is approved based on response rate and duration of response.In collaboration with Roche, the company has an ongoing Phase 3 pivotal clinical trial for Zepzelca for use as maintenance therapy in first-line extensive-stage SCLC in combination with Tecentriq (atezolizumab) following induction therapy with carboplatin, etoposide, and Tecentriq. In October 2024, the company announced positive top-line results from the trial, showing a statistically significant and clinically meaningful benefit for Zepzelca and atezolizumab in combination in the first-line maintenance setting. The company plans to submit a supplemental New Drug Application (sNDA) in the first half of 2025 to support approval of this combination in the first-line maintenance setting. In addition, the company’s licensor PharmaMar is conducting a confirmatory trial in second-line SCLC. This is a three-arm trial comparing Zepzelca as either monotherapy or in combination with irinotecan to the investigator's choice of irinotecan or topotecan. Data from either the first-line maintenance trial of Zepzelca in combination with Tecentriq or the PharmaMar trial could serve to confirm the clinical benefit of Zepzelca and support full approval in the U.S.
Ziihera: The company acquired exclusive development and commercialization rights to Ziihera in 2022 through a licensing agreement with a subsidiary of Zymeworks, providing development and commercialization rights to zanidatamab across all indications in the U.S., Europe, Japan, and all other territories except for those Asia/Pacific territories previously licensed by Zymeworks. The term of the license agreement extends on a licensed product-by-licensed product and country-by-country basis until the expiration of the royalty term for such licensed product in such country. The company has the right to terminate the license agreement at will upon a specified notice period, and either party can terminate the license agreement for the other party’s uncured material breach or bankruptcy.
Ziihera is a bispecific HER2-directed antibody that binds to two extracellular sites on HER2. Binding of zanidatamab-hrii with HER2 results in internalization, leading to a reduction of the receptor on the tumor cell surface. In the U.S., Ziihera is indicated for the treatment of adults with previously treated, unresectable, or metastatic HER2-positive (IHC 3+) BTC, as detected by an FDA-approved test. Ziihera was launched in December 2024.
Research and Development Progress
The company's research and development activities encompass all stages of development and include clinical testing of new product candidates, as well as activities related to clinical improvements of, or additional indications or new clinical data for, its existing marketed products. The company also has active preclinical programs for novel therapies, including neuroscience and precision medicines in oncology. The company is increasingly leveraging its growing internal research and development function, and it has also entered into collaborations with third parties for the research and development of innovative early-stage product candidates. Additionally, the company has supported investigator-sponsored trials that are anticipated to generate additional data related to its products. The company seeks out investment opportunities to support the development of early- and mid-stage technologies in its therapeutic areas and adjacencies. The company has a number of licensing and collaboration agreements with third parties, including biotechnology companies, academic institutions, and research-based companies and institutions, related to preclinical and clinical research and development activities in hematology, precision oncology, as well as in neuroscience.
Oncology
Zanidatamab: Zanidatamab is a HER2-targeted bispecific antibody that can simultaneously bind two non-overlapping epitopes of HER2, known as biparatopic binding. Zanidatamab currently being evaluated in multiple clinical trials as a treatment for patients with HER2-expressing cancers. In November 2024, the company announced that zanidatamab had been approved by the FDA for the treatment of adults with previously treated, unresectable, or metastatic HER2-positive (IHC 3+) BTC, as detected by an FDA-approved test. The company has an ongoing Phase 3 randomized clinical trial evaluating zanidatamab in combination with chemotherapy plus or minus tislelizumab as a first-line treatment for HER2-expressing GEA, an ongoing Phase 2 trial examining zanidatamab in combination with chemotherapy in first-line patients with HER2-expressing metastatic GEA, an ongoing Phase 3 trial examining zanidatamab in first-line patients with HER2-positive BTC. There are also multiple ongoing clinical trials exploring zanidatamab in breast cancer and other HER2-expressing tumor types.
Zepzelca. The company’s development plan for Zepzelca continues to progress.
In collaboration with Roche, the company has an ongoing Phase 3 pivotal clinical trial in use as maintenance therapy in first-line extensive-stage SCLC of Zepzelca in combination with Tecentriq (atezolizumab). In October 2024, the company announced positive top-line results from the trial showing a statistically significant and clinically meaningful benefit for Zepzelca and atezolizumab in combination in the first-line maintenance setting. In addition, the company’s licensor PharmaMar is conducting a confirmatory trial in second-line SCLC. This is a three-arm trial comparing Zepzelca as either monotherapy or in combination with irinotecan to investigator's choice of irinotecan or topotecan. Data from either the first-line maintenance trial of Zepzelca in combination with Tecentriq or the PharmaMar trial could serve to confirm clinical benefit of Zepzelca and support full approval in the U.S.
The company has an ongoing Phase 4 observational study to collect real world safety and outcome data in adult Zepzelca monotherapy patients with SCLC who progress on or after prior platinum-containing chemotherapy.
JZP815: JZP815 is a pan-RAF kinase inhibitor that targets specific components of the MAPK pathway that, when activated by oncogenic mutations, can be a frequent driver of human cancer. In October 2022, the company enrolled its first patient in a Phase 1 study to investigate the safety, dosing, and initial antitumor activity of JZP815 in participants with advanced or metastatic solid tumors harboring alterations in the MAPK pathway.
JZP898: JZP898 is a differentiated, conditionally activated IFNa INDUKINE molecule. The company acquired rights to JZP898 from Werewolf in 2022 under an exclusive, worldwide, royalty-bearing license to develop, manufacture and commercialize Werewolf's investigational WTX-613, now referred to as JZP898. In November 2023, the company enrolled its first patient in a Phase 1 study to investigate the safety, tolerability, pharmacokinetics, immunogenicity and preliminary antitumor activity of JZP898 both as a monotherapy and in combination with pembrolizumab in adults with advanced/metastatic solid tumors.
Neuroscience
Epidiolex: The company has an ongoing Phase 3 trial of Epidiolex for LGS, DS, and TSC in Japan.
JZP441: JZP441 is a potent, highly selective oral orexin-2 receptor agonist with potential application for the treatment of narcolepsy, IH, and other sleep disorders. In May 2022, the company announced that it had entered into a licensing agreement with Sumitomo to acquire exclusive development and commercialization rights in the U.S., Europe, and other territories for DSP-0187, now referred to as JZP441. Sumitomo initiated a Phase 1 trial in Japan in November 2021 to evaluate the safety, tolerability, and pharmacokinetics of JZP441 in healthy volunteers. In November 2022, the company initiated a Phase 1 development program to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of JZP441 in sleep-deprived healthy volunteers. In November 2023, it announced that the study achieved proof of concept in healthy volunteers based on the Maintenance of Wakefulness Test and that the program was being paused as the company analyzed safety findings related to visual disturbances and cardiovascular effects; no liver toxicity signals were observed. Following additional review of the trial findings and input from the FDA, the company plans to initiate a small Phase 1b trial of JZP441 in narcolepsy Type 1 patients in 2025. It expects data from this trial will further its understanding of JZP441 and orexin-2 receptor agonism, providing learnings that could inform potential future development efforts.
The company is also pursuing early-stage activities related to the development of JZP324, an extended-release low sodium oxybate formulation that could provide a clinically meaningful option for patients with serious sleep disorders.
Preclinical
Through third parties, the company is also pursuing oncology and neuroscience preclinical and clinical research and development activities under a number of licensing and collaboration agreements, including with:
XLp for rights to use XLp’s PASylation technology to extend the plasma half-life of selected asparaginase product candidates;
Redx for preclinical collaboration activities related to the Ras/Raf/MAP kinase pathway program that the company purchased from Redx;
The company is also evaluating the use of its CombiPlex delivery technology platform in a number of therapeutic formulations and combinations in oncology as part of its internal oncology research and development activities; and
Autifony to collaborate on discovering and developing drug candidates targeting two different ion channel targets associated with neurological disorders.
Commercialization Activities
The company has direct Jazz commercial operations in the U.S., Europe, Australia, and Canada, along with a network of commercial distributors that represent its commercial interests in other key markets across the globe. In the U.S., the company's products are commercialized through a number of teams, including a team of experienced, trained sales professionals who provide education and promote Xywav, Epidiolex, Zepzelca, Rylaze, Ziihera, Vyxeos, and Defitelio to healthcare providers in the appropriate specialties for each product. In addition, the company has a team that interacts with payors and institutions to ensure access and coverage for the products, as well as Xyrem, and a team that distributes the products throughout the U.S. healthcare system (wholesalers, pharmacies, hospitals, and community and academic institutions) and provides patient services.
In Canada and in approved markets in Europe and Australia where the company commercializes Defitelio and Vyxeos, it has a field force of hematology sales specialists. In markets where these products are either not approved or are unable to be promoted under local regulation, the company has medical affairs personnel responsible for responding to medical information requests and for providing information consistent with local treatment protocols with respect to such products. In certain European markets and Australia, the company has a sales team and a team of medical science liaisons supporting its commercialization of Epidiolex. In addition, the company directly markets Xywav, Xyrem, and Rylaze in Canada.
Other commercial activities include marketing-related services, pricing and access, industry analytics and insights, distribution services, and commercial support services. The company employs third-party vendors, such as advertising agencies, market research firms, and suppliers of marketing and other sales support-related services, to assist with its commercial activities. Additionally, the company provides reimbursement and patient assistance support for its U.S. markets.
The company intends to scale the size of its sales force as appropriate to effectively reach its target audience in the specialty markets in which it currently operates. It promotes Zepzelca, Rylaze, Vyxeos, Defitelio, and Ziihera to many hematology and oncology specialists who operate in the same hospitals and outpatient clinical sites.
Customers
In the U.S., Xywav and Xyrem are sold to one certified specialty pharmacy, ESSDS, that ships Xywav and Xyrem directly to patients. Additionally, in the U.S., Epidiolex is sold to specialty pharmacies, wholesalers, and specialty distributors. Defitelio is sold to hospital customers through subsidiary specialty distributors of McKesson. Zepzelca, Rylaze, Vyxeos, and Ziihera are sold to customers through subsidiary specialty distributors of McKesson, Cencora, and Cardinal. The company has distribution services agreements made in the ordinary course of business with McKesson, Cencora, and Cardinal, and a pharmacy services agreement with ESSDS.
In certain countries in Europe, Defitelio and Vyxeos are sold pursuant to marketing authorizations. The company distributes these products through Durbin PLC, a U.K.-based wholesaler and distributor, and O&M Movianto Nederland BV, its centralized European logistics services provider, to hospitals and local wholesalers in Europe where it markets these products directly, and in other markets in Europe and elsewhere where it does not market these products directly, to local distributors and wholesalers. In certain countries in Europe, Epidiolex is also sold pursuant to marketing authorizations. The company distributes Epidiolex through a variety of wholesalers and distributors. In countries where there is no marketing authorization, Epidiolex is available pursuant to named patient programs, temporary use authorizations, or similar authorizations in accordance with local regulations controlling the medical use of unapproved products.
The company commercializes and distributes Xyrem in Canada for the treatment of cataplexy in patients with narcolepsy. Xyrem is also sold in certain countries outside the U.S. and Canada by UCB under the UCB license. In July 2024, the parties agreed to terminate the UCB license. UCB has up to two years from the execution of the termination agreement to withdraw the marketing authorization for Xyrem in all applicable territories.
Marketed Products
Xywav: Xywav is manufactured at the company's Athlone facility. Xywav, like Xyrem, is a Schedule III controlled substance in the U.S. The API of Xywav are the calcium, magnesium, potassium, and sodium salts of gamma-hydroxybutyric acid (as gamma-hydroxybutyric acid is the API for Xyrem), which are Schedule I controlled substances in the U.S. As a result, Xywav and Xyrem are subject to regulation by the DEA under the CSA, and its manufacturing and distribution are highly restricted. Quotas from the DEA are required in order to manufacture or procure calcium, magnesium, potassium, and sodium salts of gamma-hydroxybutyric acid in the U.S.
Xyrem: Xyrem is manufactured by the company in its Athlone facility and by Patheon under the Patheon Agreement entered into with Patheon in 2015. The company manufactures Xyrem in its Athlone facility for most of its U.S. commercial supply and relies on Patheon to supply Xyrem for other markets, though it is not required to purchase Xyrem exclusively from Patheon. The current term of the Patheon Agreement will expire in December 2026, subject to further automatic two-yearly extensions if Patheon is then providing manufacturing services for any product, unless either party provides prior notice of termination. In addition, the company may terminate the Patheon Agreement for any reason upon 12 months’ prior written notice.
Siegfried supplies sodium oxybate, the API of Xyrem, to Patheon and the company's Athlone facility. Although Siegfried has been the company's only supplier of sodium oxybate since 2012, the company has the right to purchase a portion of its worldwide requirements of sodium oxybate from other suppliers. The agreement with Siegfried expires in April 2027, subject to automatic three-year extensions until either party provides advance notice of its intent to terminate the agreement. During the term of the agreement and, under certain circumstances, for 18 months after the agreement terminates, Siegfried is not permitted to manufacture sodium oxybate for any other company.
Epidiolex: Epidiolex/Epidyolex is manufactured by the company in its Kent Science Park facility in the U.K. Epidiolex is a pharmaceutical formulation comprising highly purified plant-derived CBD. The company cultivates its cannabinoid plants in the U.K. under highly controlled and standardized conditions.
Zepzelca: Zepzelca is manufactured by Simtra Biopharma Solutions (Simtra). The current term of the agreement with Simtra will expire in December 2025 and will then be subject to automatic two-year extensions, unless either party provides advance notice of its intent to terminate the agreement. PharmaMar retains manufacturing rights for the API for U.S. and Canadian commercial supply of Zepzelca.
Rylaze: Rylaze is manufactured by Patheon Pharmaceuticals Inc., together with its affiliates (Patheon), and the API of Rylaze is manufactured by AGC Biologics A/S. The initial term of the agreement with Patheon will expire in December 2025 and will then be subject to automatic two-year extensions, unless either party provides advance notice of its intent to terminate the agreement. The initial term of the agreement with AGC Biologics A/S will expire in October 2026 and will then be subject to automatic three-year extensions, unless either party provides advance notice of its intent to terminate the agreement.
Vyxeos: Vyxeos is manufactured by Simtra, which is a sole source supplier from a single site location, using the company's CombiPlex technology platform. CombiPlex products represent formulations with increased manufacturing complexities associated with producing drug delivery vehicles encapsulating two or more drugs that are maintained at a fixed ratio and, in the case of Vyxeos, two drugs that are co-encapsulated in a freeze-dried liposomal format. The company's manufacturing agreement with Simtra expires in August 2025, subject to automatic three-year renewal terms, unless either party provides advance notice of its intent to terminate the agreement. While other contract manufacturers may be able to produce Vyxeos, the proprietary technology that supports the manufacture of Vyxeos is not easily transferable. The marketing authorization in the EU for Vyxeos also requires the company to comply with certain manufacturing-related post-approval commitments.
Defitelio: The company is its own sole supplier of and believes that it is the sole worldwide producer of defibrotide API. It manufactures defibrotide API from porcine DNA in a single facility located in Villa Guardia, Italy. Patheon processes defibrotide API into its finished vial form under a specific product agreement entered into under a separate agreement with Patheon. Patheon is the sole provider of the company's commercial and clinical supply of Defitelio.
Ziihera: Ziihera is manufactured by WuXi Biologics (Hong Kong) Limited at their manufacturing site in WuXi, China. The initial term of the agreement with WuXi will expire in March 2029 and will then be subject to automatic three-year extensions, unless either party provides advance notice of its intent to terminate the agreement. The company has the right to appoint an alternate contract manufacturer at its discretion.
Patents and Proprietary Rights
The company owns a portfolio of U.S. and non-U.S. patents and patent applications and have licensed rights to a number of issued patents and patent applications. The company’s owned and licensed patents and patent applications cover or relate to its products and product candidates, including certain formulations, used to treat particular conditions, distribution methods and methods of administration, drug delivery technologies and delivery profiles and methods of making and use. Patents extend for varying periods according to the date of the patent filing or grant and the legal term of patents in the various countries where patent protection is obtained. The patent laws of non-U.S. countries differ from those in U.S., and the degree of protection afforded by non-U.S. patents may be different from the protection offered by U.S. patents. In addition to patents, the company’s products and product candidates are in some instances protected by various regulatory exclusivities.
The patents, patent applications and regulatory exclusivities that relate to the company’s marketed products include:
Xywav: The company has 15 U.S. patents that relate to Xywav. These patents expire from 2033 to 2041. In addition, the company has patent applications that relate to Xywav for use in additional indications that would, if issued, expire between 2040 and 2041. Xywav has been granted ODE by the FDA to treat narcolepsy through January 2028 (inclusive of six months of pediatric exclusivity), and to treat IH through August 2028. Some of the company's Xywav patents have been subject to patent litigation with the companies that filed ANDAs seeking to market a generic version of Xywav. For example, the company received notices in June 2021 and February 2023, that Lupin and Teva, respectively, filed ANDAs for generic versions of Xywav.
Xyrem. The company has seven issued patents in the U.S. relating to Xyrem listed in the Orange Book. The company's patents relate to a DDI between Xyrem and divalproex sodium. In October 2018, due to the FDA’s grant of pediatric exclusivity, an additional six months was added to the original expiration dates of all of the company's Orange Book-listed patents that existed at that time. As a result, the company's Orange Book-listed patents have periods of exclusivity between December 2022 (with an additional six months for pediatric exclusivity), and September 2033. Some of the company's Xyrem patents have been subject to patent litigation with the companies that filed ANDAs seeking to market a generic version of Xyrem, including challenges through the IPR procedures of the PTAB. The PTAB dismissed some of the IPR petitions. However, in July 2018, the United States Court of Appeals for the Federal Circuit upheld on appeal PTAB decisions finding that six patents associated with the Xywav and Xyrem REMS, and three claims of a seventh REMS patent, were unpatentable. As a result, the company will not be able to enforce patents or claims that the PTAB found unpatentable. Although the company has settled all patent litigation against the ten companies that filed ANDAs, additional companies may challenge the company's U.S. patents for Xyrem in the future.
The European Patent Office issued a method of administration patent relating to the DDI between Xyrem and divalproex sodium that expires in 2034.
Epidiolex: The company’s patent portfolio relating to the use of CBD in the treatment of epileptic encephalopathies includes 32 issued U.S. patents listed in the Orange Book. These patents claim the use of CBD for the treatment of convulsive, drop, and atonic seizures associated with both LGS and DS, an oral composition of CBD, as well as the use of CBD in combination with clobazam, and the teaching that dose adjustment may be needed when concomitantly prescribed. The patents currently listed in the Orange Book will expire between 2035 and 2041. The company has filed corresponding patent applications in many jurisdictions worldwide, including Europe, the U.K., Canada, Japan, Mexico, Australia, and New Zealand. The USPTO has granted two patents based on data that demonstrates that Epidiolex provides a benefit over synthetic CBD in an animal model of epilepsy, which will expire in 2039 and which are listed in the Orange Book. Epidiolex has received ODE to treat seizures associated with LGS and DS through 2025, and TSC through 2027. Some of the company's Epidiolex patents have been subject to patent litigation with the companies that filed ANDAs seeking to market a generic version of Epidiolex. In November and December 2022, ten companies sent the company notices that they had filed ANDAs seeking approval to market a generic version of Epidiolex.
Zepzelca: In December 2019, the company entered into an exclusive license agreement with PharmaMar pursuant to which it obtained exclusive U.S. development and commercialization rights to Zepzelca. In October 2020, the company entered into the amended license agreement which expanded its exclusive license to include rights to develop and commercialize Zepzelca in Canada. The company has a portfolio of in-licensed U.S. and Canadian patents for lurbinectedin relating to compositions, methods of use, and processes. For example, one Orange Book listed U.S. patent (expiring in 2029, with granted patent term extension) covers a genus of compounds, including lurbinectedin, and use in treating various cancers. A request for extension (CSP) has also been filed in Canada. Zepzelca has also been granted ODE for the treatment of adults with metastatic SCLC with disease progression on or after platinum-based chemotherapy until 2027, and new chemical entity exclusivity until 2025 in the U.S.
Rylaze: In 2016, the company obtained worldwide rights from Pfenex, including Pfenex’s patent rights relating to Rylaze, to develop and commercialize multiple early-stage hematology product candidates, including a license to two U.S. process patents relating to Rylaze, with respective expirations in 2026 and 2038. Pfenex has been acquired by Ligand. Rylaze has been granted orphan drug designation for the treatment of patients with ALL or LBL. The company has two patent application families relating to dosing regimens. One covers the dosing regimen (25mg/m2 intramuscularly every 48 hours), while the other covers various dosing regimens of interest. If issued, these would expire in 2040 and 2042, respectively. Another patent application relating to formulations of asparaginase would expire in 2042, if issued.
Vyxeos: The company has a portfolio of U.S. and ex-U.S. patents and patent applications for Vyxeos and the CombiPlex technology platform relating to various compositions and methods of making and use. These include seven U.S. patents covering Vyxeos compositions and methods of use expiring between 2025 and 2032, and two U.S. patents covering CombiPlex (which also cover Vyxeos) expiring in January 2027. These patents are listed in the Orange Book. The FDA granted Vyxeos ODE for the treatment of adults with newly diagnosed t-AML or AML-MRC, which expired in August 2024. In March 2021, the FDA approved an expanded label for Vyxeos for the treatment of t-AML or AML-MRC in pediatric patients 1 year and older. In addition, Vyxeos was granted OME by the EC until August 2028, ten years from its EC approval for the treatment of adults with newly diagnosed t-AML or AML-MRC. Vyxeos was approved by Health Canada for the treatment of adults with newly diagnosed t-AML or AML-MRC in April 2021. Vyxeos was approved in March 2024 in Japan and designated as an orphan drug.
Defitelio. The unique process of deriving defibrotide from porcine DNA is extensive and uses both chemical and biological processes that rely on complex characterization methods. The company has U.S. and non-U.S. patents and patent applications relating to various compositions, methods of use, and methods of characterization, with the issued patents expiring at various times between 2021 and 2035. Three U.S. patents are listed in the Orange Book. In December 2024, the FDA accepted an ANDA for a generic version of Defitelio (defibrotide sodium). The company has yet to receive notice of this ANDA filing. Defibrotide has also been granted orphan drug designation by the Korean Ministry of Food and Drug Safety to treat and prevent veno-occlusive disease (VOD), by the Commonwealth of Australia-Department of Health for the treatment of VOD, and by the European Commission (EC) for the prevention of acute Graft-versus-Host Disease. The company acquired the rights to defibrotide for the treatment and prevention of VOD in North America, Central America, and South America from Sigma-Tau Pharmaceuticals, Inc. in 2014.
Ziihera: In 2022, the company obtained a license from Zymeworks BC Inc., which included rights to a portfolio of U.S. and ex-U.S. patents and patent applications, including compositions and methods of using Ziihera. The portfolio contains a U.S. composition of matter patent relating to Ziihera, which expires in 2034 (excluding any adjustments or extensions).
The patents and/or patent applications that relate to the company’s product candidates include:
JZP815: Through a collaboration agreement and an asset purchase agreement with Redx in 2019, the company acquired a portfolio of the U.S. and non-U.S. patents and patent applications, including rights relating to compositions and methods of using JZP815. The portfolio contains a U.S. composition of matter patent relating to JZP815, which expires in 2035 (excluding any adjustments or extensions).
JZP898: Through a license agreement with Werewolf in 2022, the company obtained a license to a portfolio of U.S. and non-U.S. patents and patent applications, including rights relating to compositions and methods of using JZP898. The portfolio contains a U.S. composition of matter patent relating to JZP898, which, if granted, expires in 2040 (excluding any adjustments or extensions).
Suvecaltamide (JZP385): Through the acquisition of Cavion in 2019, the company obtained a portfolio of U.S. and non-U.S. patents and patent applications, including rights relating to compositions and methods of using suvecaltamide. The portfolio includes a U.S. composition of matter patent relating to suvecaltamide, which expires in 2027, but which may be extended to 2032 depending on regulatory approval. A U.S. patent to the treatment of ET may provide supplemental protection to 2039.
JZP441: Through a license agreement with Sumitomo Pharma Co., Ltd (Sumitomo) in 2022, the company obtained a license to a portfolio of U.S. and non-U.S. patents and patent applications, including rights relating to compositions and methods of using JZP441. The portfolio contains a U.S. composition of matter patent relating to JZP441, which expires in 2040 (excluding any adjustments or extensions).
In addition, the company has rights to a number of trademarks and service marks, and pending trademark and service mark applications, in the U.S. and elsewhere in the world to further protect the proprietary position of its products.
Government Regulation
The company and the third-party suppliers of its products are subject to cGMP, which are extensive regulations governing manufacturing processes, stability testing, recordkeeping, and quality standards as defined by the FDA, the EC, EMA, competent authorities of EU member states, and other regulatory authorities.
The API of Xywav and Xyrem, oxybate salts, are regulated by the DEA as Schedule I controlled substances, and Xywav and Xyrem are regulated as Schedule III controlled substances. Certain product candidates the company is developing contain controlled substances as defined in the CSA.
As Schedule III drugs, Xywav and Xyrem are also subject to DEA and state regulations relating to the importation, manufacturing, storage, distribution and physician prescription procedures, including limitations on prescription refills. In addition, the third parties who perform its clinical and commercial manufacturing, distribution, dispensing and clinical studies for Xywav and Xyrem are required to maintain necessary DEA registrations and state licenses.
The company’s business activities outside of the U.S. are subject to the U.S. Foreign Corrupt Practices Act, or FCPA, and similar anti-bribery or anti-corruption laws, regulations, industry self-regulation codes of conduct and physicians’ codes of professional conduct or rules of other countries in which the company operates, including the U.K. Bribery Act of 2010, or the U.K. Bribery Act.
In particular, the company is subject to the EU General Data Protection Regulation, which imposes penalties up to 4% of annual global revenue, and the California Consumer Privacy Act of 2018.
Defibrotide has also been granted orphan drug designation by the Korean Ministry of Food and Drug Safety to treat and prevent VOD, by the Commonwealth of Australia-Department of Health for the treatment of VOD and by the EC for the prevention of acute Graft-versus-Host Disease.
History
Jazz Pharmaceuticals plc was founded in 2003. The company was incorporated in 2003.
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