Jazz Pharmaceuticals, Inc.
Jazz Pharmaceuticals plc identifies, develops, and commercializes pharmaceutical products in the United States, Europe, and internationally. The company offers Xywav to treat cataplexy or excessive daytime sleepiness (EDS) with narcolepsy and idiopathic hypersomnia (IH); Xyrem for the treatment of cataplexy or EDS with narcolepsy; Epidiolex for seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC); Rylaze for the treatment of acute lymphoblastic leukemia or lymphoblastic lymphoma; Enrylaze to treat acute lymphoblastic leukemia and lymphoblastic lymphoma; Zepzelca for the treatment of metastatic small cell lung cancer with disease progression on or after platinum-based chemotherapy; Ziihera to treat HER2-positive biliary tract cancers; Defitelio to treat severe veno-occlusive disease; and Vyxeos for the treatment of newly-diagnosed therapy-related acute myeloid leukemia. It also develops Zanidatamab in Phase 3 trial to treat HER2-expressing gastroesophageal adenocarcinoma (GEA), and patients with HER2-expressing metastatic GEA; Epidiolex in Phase 3 trial to treat LGS, DS, and TSC; Suvecaltamide in Phase 2 trial to treat Parkinson's disease tremor; JZP815, a pan-RAF kinase inhibitor in Phase 1 trial that targets components of the mitogen-activated protein kinase; JZP898, a conditionally activated interferon alpha molecule in Phase 1 trial; JZP441 in Phase 1 trial to treat narcolepsy, IH, and other sleep disorders; JZP324, an extended-release low sodium oxybate formulation in Phase 1 trial for patients with sleep disorders; and JZP150 to treat post-traumatic stress disorder. The company has licensing and collaboration agreements with Redx Pharma plc, Autifony Therapeutics Limited, Zymeworks Inc., Sumitomo Pharma Co., Ltd., and Werewolf Therapeutics, Inc. Jazz Pharmaceuticals plc was founded in 2003 and is headquartered in Dublin, Ireland.
Open JAZZ dashboard ↗Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for the treatment of long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidates that are in Phase 3 clinical trials include UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; and GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome. It also develops UX701 that is in Phase 2 clinical trial for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; GeneTx; Mereo; University of Pennsylvania; Solid Biosciences Inc.; Regeneron; Abeona; and Arcturus Therapeutics Holdings Inc. The company was incorporated in 2010 and is headquartered in Novato, California.
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