Biomarin Pharmaceutical Inc. drugs

8 results
  • brineura - cerliponase alfa

    (Cerliponase Alfa)
    Biomarin Pharmaceutical Inc.
    Brineura slows the loss of walking ability in children with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), a rare inherited disorder caused by TPP1 enzyme deficiency.
  • kuvan - sapropterin dihydrochloride tablet

    (Sapropterin Dihydrochloride)
    Biomarin Pharmaceutical Inc.
    Kuvan reduces blood phenylalanine levels in adults and pediatric patients one month and older with hyperphenylalaninemia (HPA) due to BH4-responsive Phenylketonuria (PKU). Used alongside a phenylalanine-restricted diet.
  • naglazyme - galsulfase solution

    (Galsulfase)
    Biomarin Pharmaceutical Inc.
    Naglazyme is indicated for patients with Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome), improving walking and stair-climbing capacity.
  • vimizim - elosulfase alfa injection, solution, concentrate

    (Elosulfase Alfa)
    Biomarin Pharmaceutical Inc.
    Vimizim (elosulfase alfa) is indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).
  • voxzogo 0.4mg - vosoritide

    (Vosoritide)
    Biomarin Pharmaceutical Inc.
    Voxzogo 0.4mg is indicated to increase linear growth in pediatric patients with achondroplasia who have open epiphyses. Approved under accelerated approval based on improved annualized growth velocity; continued approval may depend on confirmatory trial results.