BioMarin Pharmaceutical Inc. (BMRN)

業務介紹

碧瑪瑞製藥公司開發和商業化治療罕見和危及生命的嚴重疾病和醫療狀況的療法。其商業產品包括:

Vimizim,一種酶替代療法,用於治療黏多醣症IV型A,一種溶酶體儲積症;

Naglazyme,一種重組的N-乙酰半乳糖胺4-硫酸酶,用於治療黏多醣症VI型患者;

Kuvan,一種專有的合成口服6R-BH4,用於治療苯丙酮尿症(PKU)這種遺傳性代謝疾病的患者。

公司的商業產品還包括:

Palynziq,一種PEGylated重組苯丙氨酸氨基酶,通過皮下注射來降低血液中的苯丙氨酸濃度;

Brineura,一種重組人三肽肽酶1,用於治療2型神經鈣質沉著症(一種巴登病);

Voxzogo,一種每日一次注射的c型利尿肽類似物,用於治療軟骨發育不全;

Aldurazyme,一種純化蛋白質,設計為與人體天然存在的α-L-iduronidase酶相同。

此外,公司還在開發以下產品:

valoctocogene roxaparvovec,一種腺相關病毒載體,正處於III期臨床試驗,用於治療嚴重血友病A;

BMN 307,一種AAV5介導的基因療法,正處於I/II期臨床試驗,用於使PKU患者的血液中苯丙氨酸濃度正常化;

BMN 255,正處於I/II期臨床試驗,用於治療原發性高草酸尿症。

該公司在美國、歐洲、拉丁美洲和國際上為專科藥房、醫院和非美國政府機構以及分銷商和藥品批發商提供服務。碧瑪瑞製藥公司成立於1996年,總部位於加州聖拉斐爾。



BioMarin Pharmaceutical Inc. develops and commercializes therapies for people with serious and life-threatening rare diseases and medical conditions. Its commercial products include Vimizim, an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) IV type A, a lysosomal storage disorder; Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for patients with MPS VI; and Kuvan, a proprietary synthetic oral form of 6R-BH4 that is used to treat patients with phenylketonuria (PKU), an inherited metabolic disease. The company's commercial products also comprise Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, which is delivered through subcutaneous injection to reduce blood Phe concentrations; Brineura, a recombinant human tripeptidyl peptidase 1 for the treatment of patients with ceroid lipofuscinosis type 2, a form of Batten disease; Voxzogo, a once daily injection analog of c-type natriuretic peptide for the treatment of achondroplasia; and Aldurazyme, a purified protein designed to be identical to a naturally occurring form of the human enzyme alpha-L-iduronidase. In addition, it develops valoctocogene roxaparvovec, an adeno associated virus vector, which is in Phase III clinical trial for the treatment of patients with severe hemophilia A; BMN 307, an AAV5 mediated gene therapy, which is in Phase 1/2 clinical trial to normalize blood Phe concentration levels in patients with PKU; and BMN 255 that is in Phase 1/2 clinical trial for treating primary hyperoxaluria. The company serves specialty pharmacies, hospitals, and non-U.S. government agencies, as well as distributors and pharmaceutical wholesalers in the United States, Europe, Latin America, and internationally. BioMarin Pharmaceutical Inc. has license and collaboration agreements with Sarepta Therapeutics, Ares Trading S.A., Catalyst Pharmaceutical Partners, Inc., and Asubio Pharma Co., Ltd. The company was incorporated in 1996 and is headquartered in San Rafael, California.

公司網址

https://www.biomarin.com