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Biomarin Pharmaceuticals(BMRN-Q)

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BioMarin Pharmaceutical Inc. focuses on the development and commercialization of treatments for serious life-threatening medical conditions, mostly for children. The company's portfolio comprises seven marketed products namely, Aldurazyme, Naglazyme, Kuvan a rare genetic enzyme deficiency disorder), Vimizim , Brineura, Palynziq and Voxzogo. BioMarin has a collaboration agreement with Sanofi's subsidiary Genzyme for Aldurazyme. Genzyme is BioMarin's sole customer for Aldurazyme and is responsible for marketing and selling Aldurazyme to third parties.'The newest drug in BioMarin's portfolio, Voxzogo for treating achondroplasia, the most common form of dwarfism. BioMarin's biologics license application (BLA) for Roctavian/valoctocogene roxaparvovec (valrox), a gene therapy for severe hemophilia A, was given a complete response letter (CRL) by the FDA.