BioMarin Pharmaceutical Inc. (BMRN)

業務介紹

BioMarin Pharmaceutical Inc.（BioMarin製藥公司）開發和商業化治療嚴重和危及生命的罕見疾病和醫療狀況的療法。其商業產品包括Vimizim，一種用於治療黏多醣代謝異常（MPS）IV型A的酶替代療法，一種溶酶體儲存疾病；Naglazyme，一種重組N-乙醯半乳糖胺4-硫酸酶，用於治療MPS VI患者；以及Kuvan，一種專有的合成口服6R-BH4，用於治療苯丙酮尿症（PKU），一種遺傳代謝疾病。該公司的商業產品還包括Palynziq，一種PEG化重組苯丙氨酸氨基酸酶，通過皮下注射降低血液苯丙氨酸濃度；Brineura，一種重組人三肽酶1，用於治療類脂質脂褐質素沉積症2型（巴頓病的一種形式）患者；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症；以及Aldurazyme，一種純化蛋白質，設計成與人體酶α-L-去甲基葡萄糖苷酸酶的天然形式完全相同。此外，該公司還開發了valoctocogene roxaparvovec，一種腺病毒載體，正在進行III期臨床試驗，用於治療嚴重血友病A患者；BMN 307，一種AAV5介導的基因治療，正在進行1/2期臨床試驗，以使PKU患者的血液苯丙氨酸濃度正常化；以及BMN 255，正在進行1/2期臨床試驗，用於治療原發性高草酸尿症。該公司為美國、歐洲、拉丁美洲和國際上的專科藥房、醫院和非美國政府機構提供服務，以及美國、歐洲、拉丁美洲和國際上的分銷商和藥品批發商。BioMarin Pharmaceutical Inc.與Sarepta Therapeutics、Ares Trading S.A.、Catalyst Pharmaceutical Partners, Inc.和Asubio Pharma Co., Ltd.簽訂了許可和合作協議。該公司成立於1996年，總部位於加利福尼亞州聖拉斐爾。

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