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BioMarin Pharmaceutical Inc.

Pipeline5 assets

  • BMN 333BMN 333 / C-type natriuretic peptide pathway / Long-acting CNP peptide / Skeletal Conditions
    AchondroplasiaPhase 2/3Registration-enabling study enrolling
  • BMN 351BMN 351 / Dystrophin / Oligonucleotide / Innovation
    Duchenne muscular dystrophyPhase 1/2Clinical data update expected year-end 2026
  • BMN 401BMN 401 / ENPP1 / Subcutaneous enzyme replacement therapy / Enzyme Therapies
    ENPP1 deficiencyPhase 3ENERGY 3 met 1 of 2 co-primary endpoints; next steps under evaluation
  • vosoritideVOXZOGO / CNP receptor/NPR-B pathway / Peptide / Skeletal Conditions
    HypochondroplasiaPhase 3Positive pivotal data; sNDA planned Q3 2026USEUAchondroplasiasNDAFull approval sNDA submitted April 2026USHypochondroplasia under 3 years oldPhase 2Enrollment progressing
  • pegvaliase-pqpzPALYNZIQ / Phenylalanine ammonia lyase / Enzyme therapy / Enzyme Therapies
    Phenylketonuria adolescents 12+RegulatoryFDA approved February 2026; EU approval expected 2026USEU

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