Jazz Pharmaceuticals

This is a two-stage Phase 2 trial evaluating the efficacy and safety of ONC206 in participants with Pheochromocytoma and Paraganglioma (PCPG).

Cannabidiol oral solution (CBD-OS) is approved in the US 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. This study will assess the efficacy and safety of CBD-OS in participants aged 12 to 75 years for the treatment of focal-onset seizures (FOS).Show more

The BIOMEDE 2.0 study is the second stage of the BIOMEDE multi-arm, multistage rolling programme (adaptive platform protocol). It is a multicenter, randomized, open-label, controlled phase-3 trial evaluating efficacy of ONC201 in comparison with everolimus (primary objective based on internal comparison) and subsequently to historical controls. Two treatment groups will be compared. A switch between treatment groups is allowed after confirmation of the disease progression (real-time central review blinded to the treatment arm allocation). Study treatment will be continued until centrally confirmed disease progression (either radiologically or histologically), unacceptable toxicity or consent withdrawal. The final conclusion of the trial will be successful for ONC201, if ONC201 is found significantly superior to everolimus in terms of centrally-reviewed PFS (Progression-free survival) from randomization (internal comparison) either overall, considering ND-DMG and DIPG-patients together, or in the subgroup of ND-DMG patients alone. In other cases, Everolimus will remain the standard arm unless it appears associated with an excess of toxicity compared to ONC201 which could then be discussed as a new standard.Show more

The purpose of this study is to find out if a drug called lurbinectedin (the "study drug") is safe and effective at treating people with recurrent or relapsed solid tumors, including Ewing sarcoma.

This study focuses on the therapeutic relevance of the endocannabinoid (eCB) system for the treatment of Fragile-X syndrome (FXS), the primary hereditary cause of autism spectrum disorder (ASD). Most individuals with FXS have moderate to severe intellectual disability (ID), and caregivers are mainly concerned about aggressive behavior and anxiety problems. Since FXS individuals have a normal lifespan, the overall lifetime cost for the Canadian society of a single case is estimated at $1.2 to $4.7 millions reaching $18 billions for all FXS cases. There is no cure for FXS, as all clinical trials so far have been unsuccessful.FXS is caused by transcriptional silencing of the Fragile X mental retardation protein (FMR1) gene, making FXS a simple model to study ASD and ID pathophysiological mechanisms. Of those, neuronal hyperexcitability is largely recognized as a core deficit in FXS, and a critical therapeutic target for the disorder. Using transcranial magnetic stimulation (TMS) in FXS patients, our team provided the first direct evidence of Gamma-aminobutyric acid (GABA) receptor a (GABAa) dysfunctions in humans with this disorder and showed that this inhibitory deficit is linked with cortical hyperexcitability (PMID: 31748507). Concurrent lines of evidence suggest that stimulation of the endocannabinoid (eCB) system with the administration of Cannabidiol (CBD) could upregulate GABAergic function and correct inhibitory deficits presumed responsible for the neuropsychiatric phenotype of FXS. CBD has been shown to increase GABA concentration levels in the brains of healthy individuals, an effect that could help correct the hyperexcitability typically found in FXS. Thus, this trial aims to define the therapeutic potential of the eCB system for FXS, by measuring the impacts of oral CBD administration on the principal inhibitory neurotransmitter system of FXS patients, and the severity of the clinical phenotype.Show more

This phase II trial determines if the combination of ONC201 with different drugs is effective for treating participants with diffuse midline gliomas (DMGs). Despite years of research, little to no progress has been made to improve outcomes for participants with DMGs, and there are few treatment options. This trial will utilize an adaptive platform design in that the different treatment arms for each cohort will be opened and closed based on ongoing preclinical investigation as well as evolving outcome data from the trial. Novel agents will be continuously added to this study as pre-clinical data emerge to suggest additive or synergistic activity when combined ONC201. Should a novel agent not have an RP2D at the time of incorporation into this study, a phase 1 lead-in will be performed prior to initiation of combination therapy (via study amendment).Show more

This phase I trial studies the effects and best dose of ONC206 alone or in combination with radiation therapy in treating patients with diffuse midline gliomas that is newly diagnosed or has come back (recurrent) or other recurrent primary malignant CNS tumors. ONC206 is a recently discovered compound that may stop cancer cells from growing. This drug has been shown in laboratory experiments to kill brain tumor cells by causing a so called "stress response" in tumor cells. This stress response causes cancer cells to die, but without affecting normal cells. ONC206 alone or in combination with radiation therapy may be effective in treating newly diagnosed or recurrent diffuse midline gliomas and other recurrent primary malignant CNS tumors.Show more

The trial is a randomized, open-label phase II study comparing CPX-351 vs conventional intensivechemotherapy in patients with newly diagnosed de novo AML and intermediate- or adverse-risk genetics

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 has 6 FDA-approved drugs with a rare disease use that we track, as of November 2026.

Xyrem (sodium oxybate), approved 2002: Narcolepsy Type 1.

Xywav (calcium, magnesium, potassium, and sodium oxybates), approved 2020: Narcolepsy Type 1.

Modeyso (dordaviprone), approved 2025: Diffuse Intrinsic Pontine Glioma.

Ziihera (zanidatamab), approved 2024: Cholangiocarcinoma.

Epidiolex (cannabidiol), approved 2018: Dravet Syndrome, Lennox-Gastaut Syndrome, Tuberous Sclerosis Complex.

Vyxeos (daunorubicin and cytarabine (liposomal)), approved 2017: Acute Myeloid Leukemia.

Still in development: ABS-1230 (Phase 2).

Source: the FDA's openFDA label database, matched to the rare diseases on Trial Friend; the list updates automatically and can include older products.

Yes, for 6 of its FDA-approved rare disease medicines that we track, each checked on Jazz Pharmaceuticals' own website.

Xyrem: JazzCares (Jazz Pharmaceuticals), 1-833-533-5299. Offers copay help with private insurance, a free medicine program, a short-term supply while insurance is decided and insurance help. Checked November 10, 2026.

Xywav: JazzCares (Jazz Pharmaceuticals), 1-833-533-5299. Offers copay help with private insurance, a free medicine program, a short-term supply while insurance is decided and insurance help. Checked November 10, 2026.

Modeyso: JazzCares for MODEYSO Patient Support Program, 1-844-302-2737. Offers copay help with private insurance, a free medicine program, a short-term supply while insurance is decided and insurance help. Checked October 12, 2026.

Ziihera: JazzCares, 1-833-533-5299. Offers copay help with private insurance, a free medicine program and insurance help. Checked October 12, 2026.

Epidiolex: JazzCares for EPIDIOLEX, 1-833-426-4243. Offers copay help with private insurance, a free medicine program, a short-term supply while insurance is decided and insurance help. Checked November 2, 2026.

Vyxeos: JazzCares for Vyxeos, 1-833-533-5299. Offers copay help with private insurance, a free medicine program and insurance help. Checked October 19, 2026.

Medicare and Medicaid: drugmaker copay cards are limited to private insurance, so people with government coverage usually look to a free medicine program or a charity fund, both on the help paying page.

Eligibility is decided by each program, so call the number listed before a prescription is filled.