Jazz Pharmaceuticals
Jazz Pharmaceuticals works on 22 rare diseases tracked on Trial Friend, including CDKL5 Deficiency Disorder, Cholangiocarcinoma, Chronic Graft-versus-Host Disease and 19 more, with 12 active clinical trials (7 recruiting) and 8 FDA-approved rare disease drugs.
Jazz Pharmaceuticals develops medicines for rare neurological and oncologic conditions. Following its $7.2 billion acquisition of GW Pharmaceuticals in 2021, the company's portfolio includes Epidiolex (cannabidiol) for Dravet syndrome, Lennox-Gastaut syndrome, and tuberous sclerosis complex, Xywav (calcium, magnesium, potassium, and sodium oxybates) for narcolepsy and idiopathic hypersomnia, and oncology products including Zepzelca for small cell lung cancer, Rylaze for acute lymphoblastic leukemia, Vyxeos for acute myeloid leukemia, and Ziihera (zanidatamab-hrii) for previously treated HER2-positive biliary tract cancer and, on its current label, first-line HER2-positive stomach and esophageal adenocarcinoma with chemotherapy. Jazz bought Chimerix in April 2025, and its drug Modeyso (dordaviprone) received FDA accelerated approval on August 6, 2025 for adults and children 1 year and older with diffuse midline glioma that carries an H3 K27M mutation and has grown after earlier treatment.
On September 15, 2026, Jazz completed its acquisition of Actio Biosciences for $820 million upfront plus up to $500 million tied to approval and sales milestones. The deal was for ABS-1230, an oral small molecule that blocks the KCNT1 ion channel, in development for KCNT1-related epilepsy. That is a rare genetic epilepsy affecting roughly 2,500 people in the United States, where children can have dozens or even hundreds of seizures a day and no FDA-approved treatment exists. The ongoing Phase 1b/2a KYRON trial is intended to support a U.S. new drug application. Actio spun out its other programs into a separate, independent company at closing, in which Jazz holds a minority stake.
Jazz Pharmaceuticals Drug Pipeline
Jazz Pharmaceuticals has 12 active clinical trials across 5 development stages, with 7 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline counts the active and recruiting trials listed on this page, from ClinicalTrials.gov. For companies that work outside rare disease, we list only trials for rare diseases we track, so the company's full pipeline may be larger.
Jazz Pharmaceuticals Clinical Trials (12)
Active and recruiting clinical trials sponsored by Jazz Pharmaceuticals, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
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Jazz Pharmaceuticals FDA-Approved Drugs (8)
Medications developed or marketed by Jazz Pharmaceuticals that have received U.S. Food and Drug Administration approval. Drug data is sourced from the openFDA database and includes brand names, generic names, approval dates, and matched rare disease indications. Where a drug treats a condition covered by Trial Friend, the disease name links directly to that disease page.
| Drug Name | Brand Name | Rare Diseases | Approval Date |
|---|---|---|---|
| DEFIBROTIDE SODIUM | DEFITELIO intravenous | — | Mar 30, 2016 |
| CANNABIDIOL | Epidiolex oral | Jun 25, 2018 | |
| DORDAVIPRONE Protease Activator [EPC] | MODEYSO oral | Aug 6, 2025 | |
| ASPARAGINASE ERWINIA CHRYSANTHEMI (RECOMBINANT)-RYWN Asparagine-specific Enzyme [EPC] | Rylaze intramuscular | — | Jun 30, 2021 |
| (DAUNORUBICIN AND CYTARABINE) LIPOSOME Nucleoside Metabolic Inhibitor [EPC] | VYXEOS intravenous | Aug 3, 2017 |
Jazz Pharmaceuticals Trial Locations
Jazz Pharmaceuticals clinical trials are running at 197 sites in 15 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (22)
Diseases targeted by Jazz Pharmaceuticals's clinical trial and drug development programs
CDKL5 deficiency disorder is a severe genetic condition that causes early-onset seizures, usually beginning within the first few months of life, along with significant developmental delays and movemen...
Cholangiocarcinoma is a rare and aggressive cancer that forms in the bile ducts, the thin tubes that carry digestive fluid (bile) from the liver to the small intestine. It can occur inside the liver (...
Chronic graft-versus-host disease is an immune-mediated complication after allogeneic stem cell or bone marrow transplant, in which donor immune cells attack the recipient's tissues. It can affect the...
Desmoplastic small round cell tumor is a rare aggressive sarcoma characterized by EWSR1-WT1 fusion and dense desmoplasia. Most commonly arising in the peritoneum of adolescents and young adults, the c...
Diffuse intrinsic pontine glioma (DIPG) is an aggressive childhood brain tumor that grows in the pons, the part of the brainstem controlling breathing, swallowing, and movement. Because it weaves thro...
Dravet Syndrome is a severe form of epilepsy that typically begins in infancy with prolonged seizures triggered by fever. Most cases are caused by mutations in the SCN1A gene encoding a sodium channel...
Patient Resources
Organizations and resources related to Jazz Pharmaceuticals's rare disease focus areas
Frequently Asked Questions About Jazz Pharmaceuticals
Common questions about Jazz Pharmaceuticals's rare disease programs, clinical trials, and treatments.