Profile: Jazz Pharmaceuticals PLC (JAZZ.O)
Jazz Pharmaceuticals Public Limited Company, incorporated on March 15 2005, is a specialty biopharmaceutical company focused on developing and commercializing differentiated therapeutic products. The Company's marketed products include Xyrem (sodium oxybate oral solution) for the treatment of both cataplexy and excessive daytime sleepiness in patients with narcolepsy; Erwinaze for the treatment of acute lymphoblastic leukemia (ALL); Defitelio (defibrotide) indicated for the treatment of severe hepatic veno-occlusive disease (VOD), and Prialt (ziconotide) for the treatment of severe chronic pain. Its other product candidates include JZP-110 for treatment of excessive daytime sleepiness (EDS) in narcolepsy and EDS in patients with obstructive sleep apnea (OSA); JZP-386 for the treatment of EDS in narcolepsy, JZP-416, to treat ALL and Leukotac for the treatment of steroid-refractory acute Graft-versus-host disease (GvHD).
Xyrem is a treatment approved by the United States Food and Drug Administration (FDA) for both EDS and cataplexy in patients with narcolepsy. Sodium oxybate, the active pharmaceutical ingredient in Xyrem, is a formulation of the sodium salt of gamma-hydroxybutyrate, an endogenous neurotransmitter and metabolite of gamma-aminobutyric acid. Xyrem was approved in the United States for the treatment of cataplexy in patients with narcolepsy and was approved for EDS in patients with narcolepsy. The American Academy of Sleep Medicine recommended Xyrem as a standard of care for the treatment of both EDS and cataplexy associated with narcolepsy. The Company is conducting a Phase III clinical trial of Xyrem for the treatment of Cataplexy in narcolepsy in children and adolescents.
Erwinaze, a biologic product, is used in conjunction with chemotherapy to treat patients with ALL who have developed hypersensitivity to E. coli-derived asparaginase. Erwinaze is an asparaginase, a type of enzyme that can deprive leukemic cells of an amino acid essential for their growth. It is derived from a rare bacterium (Erwinia chrysanthemi) and is immunologically distinct from E. coli-derived asparaginase and suitable for patients with hypersensitivity to E. coli-derived treatments. Erwinaze was approved by the United States Food and Drug Administration (FDA) under a biologics license application, or BLA, for administration via intramuscular injection in conjunction with chemotherapy. The FDA has also approved a supplemental BLA for administration of Erwinaze via intravenous infusion in conjunction with chemotherapy. Erwinaze is available in the United States. Outside of the United States, Erwinaze is sold under the name Erwinase in multiple countries in Europe and elsewhere. The Company is conducting a Phase II pharmacokinetic study of Erwinaze.
Defibrotide, the active pharmaceutical ingredient in Defitelio, is the sodium salt of a complex mixture of single-stranded oligodeoxyribonucleotides derived from porcine DNA. In in-vitro studies, defibrotide has shown a number of pharmacological effects that suggest it has a role in both protection of the endothelial cells that form the inner lining of blood vessels and the restoration of the balance between clot formation and breakdown in the blood. Defibrotide has been developed for the treatment and prevention of VOD. The European Commission, or EC, granted marketing authorization for Defitelio for the treatment of severe VOD in adults and children undergoing hematopoietic stem cell transplantation (HSCT) therapy. Defitelio has been well-tolerated; the most frequent adverse reactions observed during pre-marketing use of the product are hemorrhage, hypotension and coagulopathy.
Prialt is an intrathecally administered infusion of ziconotide. Prialt is approved by the United States Food and Drug Administration (FDA) for the management of severe chronic pain in patients for whom intrathecal therapy is warranted, and who are intolerant of or refractory to other treatment, such as systemic analgesics, adjunctive therapies or intrathecal morphine. For most patients who achieve good pain relief and tolerability with Prialt, pain relief can be maintained over time without cumulative toxicity.
The Company is engaged in the development of JZP-110 is a late-stage investigational compound. JZP-110 is being developed for treatment of EDS in patients with narcolepsy and EDS in patients with OSA.
JZP-386 is a deuterium-modified analog of sodium oxybate, the active pharmaceutical ingredient in Xyrem. The Company has conducted preclinical research and development work on JZP-386 for the use in patients with narcolepsy. It received an approval of an investigational medicinal product dossier (IMPD). The Company conducted and completed the first study of JZP-386 in humans to evaluate the safety, pharmacokinetics and pharmacodynamics of the compound. It initiated and is conducting a second Phase I study.
The Company completed a Phase I clinical trial in Europe of JZP-416 (pegcrisantaspase). JZP-416 is a PEGylated recombinant Erwinia chrysanthemi L-asparaginase, being developed for the treatment of patients with ALL who are hypersensitive to E. coli-derived asparaginase. The FDA granted Fast Track designation to the investigation of JZP-416 for the treatment of ALL.
The Company is conducting a Phase III clinical trial in Europe of Leukotac (inolimomab). Leukotac is an anti-CD25 monoclonal antibody for the treatment of steroid-refractory acute GvHD. The Company completed enrollment for this Phase III study.
Jazz Pharmaceuticals PLC
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