BioMarin Pharmaceuticals Inc
BioMarin Pharmaceutical Inc., a biotechnology company, engages in the development and commercialization of therapies for life-threatening rare diseases and medical conditions in the United States, Europe, Latin America, the Middle East, the Asia Pacific, and internationally. The company's products include VIMIZIM, an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) IV type A, a lysosomal storage disorder; VOXZOGO, a once daily injection analog of c-type natriuretic peptide (CNP) for the treatment of achondroplasia; NAGLAZYME, a recombinant form of N- acetylgalactosamine 4-sulfatase for patients with MPS VI; and PALYNZIQ, a PEGylated recombinant phenylalanine (Phe) ammonia lyase enzyme delivered through subcutaneous injection to reduce blood Phe concentrations. It also develops BRINEURA, a recombinant human tripeptidyl peptidase 1 for the treatment of patients with ceroid lipofuscinosis type 2, a form of Batten disease; ALDURAZYME, a purified protein designed to be identical to a naturally occurring form of the human enzyme alpha-L-iduronidase; KUVAN, a proprietary synthetic oral form of 6R-BH4 that is used to treat patients with phenylketonuria, an inherited metabolic disease; and ROCTAVIAN, an adeno associated virus vector for the treatment of severe hemophilia A. The company's products under development include BMN 333, a longer-acting CNP for the treatment of multiple growth disorders, such as achondroplasia and hypochondroplasia; BMN 349, an oral therapeutic for the treatment of liver disease associated with alpha-1 antitrypsin deficiency; and BMN 351, an oligonucleotide for the treatment of duchenne muscular dystrophy. It serves specialty pharmacies, hospitals, non-U.S. government agencies, distributors, and pharmaceutical wholesalers. The company has license and collaboration agreements with Catalyst Pharmaceutical Partners, Inc., and Ares Trading S.A. The company was incorporated in 1996 and is based in San Rafael, California.
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Novartis AG engages in the research, development, manufacture, distribution, marketing, and sale of pharmaceutical medicines in Switzerland and internationally. The company offers Entresto, an angiotensin receptorneprilysin inhibitor for the treatment of symptomatic chronic heart failure with reduced ejection fraction (HFrEF); Cosentyx to treat plaque psoriasis, pso riatic arthritis, ankylosing spondylitis, and nonradiographic axial spondy loarthritis; Kisqali, a selective oral cyclin dependent inhibitor of kinases 4 and 6 (CDK4/6); Promacta/Revolade to treat immune thrombocytopenia (ITP), thrombocytopenia, and patients with severe aplastic anemia (SAA); Tafinlar+Mekinist, an oral combination therapy to treat patients with certain types of cancers; and Jakavi for the treatment of myelofibrosis, polycythemia vera, and acute or chronic graftversushost disease (GvHD). It also provides Tasigna to treat philadelphia chromosomepositive chronic myeloid leukemia in the chronic and/or accelerated phase; Xolair for the treatment of allergic asthma and nasal polyps or severe chronic rhinosi nusitis with nasal polyps; Ilaris to treat fever syndromes, Still's disease, and acute gouty arthritis; Pluvicto to treat prostatespecific membrane anti genpositive metastatic castrationresistant prostate cancer; Sandostatin SC and Sandostatin LAR to treat acromegaly carcinoid tumors and other types of functional gastro intestinal and pancreatic neuroendocrine tumors; Zolgensma for the treatment of genetic root cause of spinal muscular atrophy; Lucentis; Leqvio to reduce LDL cholesterol; Lutathera; Scemblix; and Fabhalta. The company focuses on therapeutic areas, such as cardiovascular, renal and metabolic, immunology, neuroscience, oncology, and hematology. It has a license and collaboration agreement with Alnylam Pharmaceuticals, Inc. to develop, manufacture, and commercialize Leqvio (inclisiran), a therapy to reduce LDL cholesterol. Novartis AG was incorporated in 1996 and is headquartered in Basel, Switzerland.
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