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KMDA vs RARE

Kamada Ltd and Ultragenyx Pharmaceutical Inc.

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Kamada Ltd

Kamada Ltd. manufactures and sells plasma-derived protein therapeutics. Its products include KAMRAB/KEDRAB indicated as prophylaxis of rabies; CYTOGAM for prophylaxis of Cytomegalovirus disease in kidney, lung, liver, pancreas, heart, and heart/lung transplants; VARIZIG for post exposure prophylaxis of varicella; WINRHO SDF for immune thrombocytopenic purpura and suppression of rhesus isoimmunization; HEPAGAM B for prevention of hepatitis B recurrence liver transplants and post-exposure prophylaxis; GLASSIA for intravenous AATD; KAMRHO (D) IM for prophylaxis of hemolytic disease of newborns; and Echis coloratus and Vipera palaestinae Antiserum to treat snake bite. The company also distributes biopharmaceutical products in Israel, including BRAMITOB to manage chronic pulmonary infection; FOSTER to treat asthma; TRIMBOW for chronic obstructive pulmonary disease; PROVOCHOLINE for the diagnosis of bronchial airway hyperactivity; AEROBIKA, an OPEP device; RUPAFIN and RUPAFIN ORAL SOLUTION for allergic rhinitis and Urticaria; SINTREDIUS for rheumatoid arthritis, systemic lupus erythematosus, and mild-moderate juvenile dermatomyositis; IVIG for various immunodeficiency-related conditions; VARITECT for chicken pox and zoster herpes; ZUTECTRA and HEPATECT CP for prevention of hepatitis B; MEGALOTECT CP for CMV virus; RUCONEST for angioedema attack; HEPARIN SODIUM INJECTION for thrombo-embolic disorders and prophylaxis of deep vein thrombosis and thromboembolic events; ALBUMIN and ALBUMIN for blood plasma; Factor VIII for hemophilia type A; and Factor IX for hemophilia type B. In addition, it distributes COAGADEX to treat hereditary factor X deficiency; IXIARO for Japanese encephalitis; VIVOTIF for salmonella typhi; PROCYSBI for nephropathic cystinosis; LAMZEDE for alpha-mannosidosis; RYPLAZIM for hypoplasminogenemia; ELIGARD for prostate cancer; and BEVACIZUMAB KAMADA for various cancers. The company was incorporated in 1990 and is headquartered in Rehovot, Israel.

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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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