Wednesday, June 10, 2026

pharma Β· Phenylketonuria Β· Achondroplasia

BioMarin Pharmaceutical Australia

BioMarin Pharmaceutical Australia drug portfolio β€” 36 indexed compounds with therapeutic class and molecular targets from public FDA and regional approval metadata.

770 Lindaro Street, San Rafael, CA 94901, US HQ
1997 Founded
3,171 Employees
TGA registrant Type
Company details
Intelligence Β· Drug Pipeline

BioMarin Pharmaceutical Australia pipeline

Portfolio lens on 36 drugs and development assets β€” regulatory status, targets, and deep links to NovaPharmaNews drug profiles.

BioMarin Pharmaceutical Australia drug portfolio β€” 36 indexed compounds with therapeutic class and molecular targets from public FDA and regional approval metadata.

Drug portfolio

36 compounds
  1. Development asset linked to BioMarin Pharmaceutical Australia in public FDA and regional records.

  2. Development asset linked to BioMarin Pharmaceutical Australia in public FDA and regional records.

  3. US regulatory status: approved. Molecular target: Aromatic-L-amino-acid decarboxylase.

    Target
    Aromatic-L-amino-acid decarboxylase
    Mechanism
    DOPA decarboxylase inhibitor
  4. cerliponase alfa

    Alimentary tract and metabolism (A16)

    US regulatory status: approved. Molecular target: Polypeptides.

    Target
    Polypeptides
    Mechanism
    Polypeptides proteolytic enzyme
  5. Development asset linked to BioMarin Pharmaceutical Australia in public FDA and regional records.

  6. US regulatory status: approved. Molecular target: DNA.

    Target
    DNA
    Mechanism
    DNA inhibitor
  7. Development asset linked to BioMarin Pharmaceutical Australia in public FDA and regional records.

  8. elosulfase alfa

    Alimentary tract and metabolism (A16)

    US regulatory status: approved. Molecular target: Chondroitin-6-sulfate.

    Target
    Chondroitin-6-sulfate
    Mechanism
    Chondroitin-6-sulfate hydrolytic enzyme
  9. US regulatory status: approved. Molecular target: Bacterial 70S ribosome.

    Target
    Bacterial 70S ribosome
    Mechanism
    Bacterial 70S ribosome inhibitor
  10. US regulatory status: approved. Molecular target: Estrogen receptor.

    Target
    Estrogen receptor
    Mechanism
    Estrogen receptor alpha agonist
  11. galsulfase

    Alimentary tract and metabolism (A16)

    US regulatory status: approved. Molecular target: Dermatan sulfate.

    Target
    Dermatan sulfate
    Mechanism
    Dermatan sulfate hydrolytic enzyme
  12. Development asset linked to BioMarin Pharmaceutical Australia in public FDA and regional records.

  13. Development asset linked to BioMarin Pharmaceutical Australia in public FDA and regional records.

  14. Development asset linked to BioMarin Pharmaceutical Australia in public FDA and regional records.

  15. Development asset linked to BioMarin Pharmaceutical Australia in public FDA and regional records.

  16. laronidase

    Alimentary tract and metabolism (A16)

    US regulatory status: approved. Molecular target: Dermatan sulfate.

    Target
    Dermatan sulfate
    Mechanism
    Dermatan sulfate hydrolytic enzyme
  17. melatonin

    Nervous system (N05)

    Nervous system (N05) compound in BioMarin Pharmaceutical Australia's portfolio. Molecular target: Melatonin receptor.

    Target
    Melatonin receptor
    Mechanism
    Melatonin receptor agonist
  18. Development asset linked to BioMarin Pharmaceutical Australia in public FDA and regional records.

  19. pegvaliase

    Alimentary tract and metabolism (A16)

    Alimentary tract and metabolism (A16) compound in BioMarin Pharmaceutical Australia's portfolio. Molecular target: Phenylalanine.

    Target
    Phenylalanine
    Mechanism
    Phenylalanine hydrolytic enzyme
  20. US regulatory status: approved. Molecular target: Phenylalanine.

    Target
    Phenylalanine
    Mechanism
    Phenylalanine hydrolytic enzyme
  21. US regulatory status: approved. Molecular target: Peroxisome proliferator-activated receptor gamma.

    Target
    Peroxisome proliferator-activated receptor gamma
    Mechanism
    Peroxisome proliferator-activated receptor gamma agonist
  22. Development asset linked to BioMarin Pharmaceutical Australia in public FDA and regional records.

  23. Development asset linked to BioMarin Pharmaceutical Australia in public FDA and regional records.

  24. Development asset linked to BioMarin Pharmaceutical Australia in public FDA and regional records.

  25. Development asset linked to BioMarin Pharmaceutical Australia in public FDA and regional records.

  26. sapropterin

    Alimentary tract and metabolism (A16)

    Alimentary tract and metabolism (A16) compound in BioMarin Pharmaceutical Australia's portfolio. Mechanism: Phenylalanine-4-hydroxylase activator.

    Mechanism
    Phenylalanine-4-hydroxylase activator
  27. US regulatory status: approved. Molecular target: Phenylalanine-4-hydroxylase.

    Target
    Phenylalanine-4-hydroxylase
    Mechanism
    Phenylalanine-4-hydroxylase activator
  28. Development asset linked to BioMarin Pharmaceutical Australia in public FDA and regional records.

  29. Development asset linked to BioMarin Pharmaceutical Australia in public FDA and regional records.

  30. Development asset linked to BioMarin Pharmaceutical Australia in public FDA and regional records.

  31. Development asset linked to BioMarin Pharmaceutical Australia in public FDA and regional records.

  32. talazoparib

    Antineoplastic and immunomodulating agents (L01)

    Antineoplastic and immunomodulating agents (L01) compound in BioMarin Pharmaceutical Australia's portfolio. Molecular target: Poly [ADP-ribose] polymerase 2.

    Target
    Poly [ADP-ribose] polymerase 2
    Mechanism
    Poly [ADP-ribose] polymerase 2 inhibitor
  33. US regulatory status: approved. Molecular target: Poly [ADP-ribose] polymerase 2.

    Target
    Poly [ADP-ribose] polymerase 2
    Mechanism
    Poly [ADP-ribose] polymerase-1 inhibitor
  34. valoctocogene roxaparvovec

    Blood and blood forming organs (B02)

    Blood and blood forming organs (B02) compound in BioMarin Pharmaceutical Australia's portfolio. Molecular target: Coagulation factor IX.

    Target
    Coagulation factor IX
    Mechanism
    Coagulation factor IX exogenous protein
  35. vosoritide

    Musculo-skeletal system (M05)

    US regulatory status: approved. Molecular target: Atrial natriuretic peptide receptor 2.

    Target
    Atrial natriuretic peptide receptor 2
    Mechanism
    Atrial natriuretic peptide receptor B binding agent