Dedicated to creating life-changing medicines for people with
limited or no options.

Research and Development

As the field of science has grown, so have our R&D capabilities. We actively explore revolutionary drug options ranging from small molecule advancements to biologics, and novel compounds within hematology/oncology and sleep medicine. This dynamic approach has allowed us to shepherd new medicines to market — from late-stage discovery through clinical development and regulatory approval.

All of these advancements are a reflection of our continued investment in an evolving and growing research and development focus.

Pipeline

PRE-CLINICAL
Oxybate
Once Nightly Formulation

CombiPlex
Solid tumors candidate

CombiPlex
Hem/Onc exploratory activities

Recombinant Crisantaspase-HLE2
ALL/other hematological malignancies

Recombinant Pegaspargase1
Hematological malignancies

Defibrotide
Exploratory activities

Exosome NRAS candidate2
Hematological malignancies

Exosome STAT3 candidate2
Hematological malignancies

Exosome-based candidates2
Solid tumors/ Hematological malignancies

Pan-RAF Inhibitor Program
RAF & RAS mutant tumors

PHASE 1
CPX-351 + gemtuzumab3
R/R AML or HMA Failure
MDS

CPX-351 + venetoclax
Low Intensity Therapy for first-line, unfit AML (Phase 1b)

CPX-3513
Low Intensity Dosing for higher risk MDS

IMGN6321
CD123+ Hematological malignancies5

PHASE 2
Defibrotide
Prevention of aGvHD

Defibrotide4
Treatment of TA-TMA

Defibrotide4
Prevention of CAR-T associated neurotoxicity

CPX-351 + venetoclax3
de novo or R/R AML

CPX-3516
HR-MDS

CPX-3516
R/R AML (COG)

CPX-3514,6
Newly diagnosed older adults with HR-AML

CPX-351 + venetoclax4,6
HR-AML

JZP-458 (Recombinant Crisantaspase)4
ALL (Phase 2/3)

CX-8998
Essential Tremor

PHASE 3
JZP-258
Cataplexy & EDS in narcolepsy

JZP-258
Idiopathic hypersomnia

Defibrotide
Prevention of VOD

CPX-3516
AML or HR-MDS (AML19)

CPX-3516
AML or HR-MDS (AML18)

CPX-3516
Newly diagnosed adults with standard- and HR-AML

CPX-3514,6
Newly diagnosed pediatric patients

REGULATORY
Solriamfetol EU
EDS in OSA and Narcolepsy

Sleep / Neuroscience
Hematology / Oncology
1Opt-in opportunity
2Partnered collaboration
3Jazz & MD Anderson Cancer Center collaboration study
4Planned
5Including AML and BPDCN
6Cooperative group study

Through Jazz’s patented CombiPlex® platform, Jazz is evaluating oncology combinations to support identification of clinical product candidate(s) and is leading a collaborative approach with partners to research and develop new therapies.