BioMarin Pharmaceutical

BioMarin Pharmaceutical products

Aldurazyme®

• Generic: laronidase

• Indication: enzyme replacement therapy for Mucopolysaccharidosis I (Hurler and Hurler–Scheie forms)

• Dose / Usage: intravenous infusion regularly as per prescribing guidelines

Brineura®

• Generic: cerliponase alfa

• Indication: slowing motor function loss in children with neuronal ceroid lipofuscinosis type 2 (CLN2 disease / Batten disease), now approved for all ages

• Dose / Usage: administered via intraventricular infusion per treatment protocol

Kuvan®

• Generic: sapropterin dihydrochloride

• Indication: treatment of phenylketonuria (PKU) to help lower phenylalanine levels

• Dose / Usage: oral dosing individualized based on response

Naglazyme®

• Generic: galsulfase

• Indication: enzyme replacement therapy for Mucopolysaccharidosis VI (MPS VI, Maroteaux–Lamy syndrome)

• Dose / Usage: intravenous infusion as scheduled

Palynziq®

• Generic: pegvaliase-pqpz

• Indication: treatment of PKU in adults—alternative mechanism to Kuvan

• Dose / Usage: subcutaneous injection following induction and maintenance protocols

Roctavian™

• Generic: valoctocogene roxaparvovec-rvox

• Indication: one-time gene therapy for adults with severe hemophilia A (congenital factor VIII deficiency, with FVIII activity < 1 IU/dL), without AAV5 antibodies

• Dose / Usage: single intravenous infusion

Vimizim®

• Generic: elosulfase alfa

• Indication: enzyme replacement therapy for Morquio A syndrome (MPS IVA)

• Dose / Usage: regular intravenous infusion as directed

Voxzogo®

• Generic: vosoritide

• Indication: increases linear growth in children with achondroplasia and open growth plates, now approved in younger age groups (including under 5 years)

• Dose / Usage: daily subcutaneous injection