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Intelligence · Clinical Pipeline

BioMarin Pharmaceutical Australia Pipeline

All ClinicalTrials.gov programs at BioMarin Pharmaceutical Australia — phase, modality, and trial IDs.

Programs
91

Quick answer

BioMarin Pharmaceutical Australia has 91 clinical development programs indexed from ClinicalTrials.gov, including phase, modality, indication text, and linked NCT identifiers.

  1. Aldurazyme

    BioMarin Pharmaceutical Australia Pty Ltd

    Mucopolysaccharidosis I

    phase 3 mab completed

    1 trial

  2. BMN 110

    BioMarin Pharmaceutical Australia Pty Ltd

    Mucopolysaccharidosis IVA (Morquio A Syndrome)

    phase 3 small molecule completed

    1 trial

  3. BMN 110 - Weekly

    BioMarin Pharmaceutical Australia Pty Ltd

    Mucopolysaccharidosis IV A

    phase 3 small molecule completed

    1 trial

  4. BMN 110 Weekly

    BioMarin Pharmaceutical Australia Pty Ltd

    MPS IV A

    phase 3 small molecule completed

    1 trial

  5. BMN 111

    BioMarin Pharmaceutical Australia Pty Ltd

    Achondroplasia

    phase 3 small molecule active

    6 trials

  6. BMN 165

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonuria

    phase 3 small molecule completed

    1 trial

  7. BMN 333

    BioMarin Pharmaceutical Australia Pty Ltd

    Achondroplasia

    phase 3 small molecule active

    1 trial

  8. BMN 333, Voxzogo 0.56 mg powder and solvent for solution for injection, Voxzogo 1.2 mg powder and solvent for solution for injection, BMN 333

    BioMarin Pharmaceutical Australia Pty Ltd

    Achondroplasia

    phase 3 small molecule active

    1 trial

  9. BMN 701

    BioMarin Pharmaceutical Australia Pty Ltd

    Late-onset Pompe Disease

    phase 3 small molecule terminated

    1 trial

  10. BMN165 20mg/day

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonuria (PKU)

    phase 3 small molecule completed

    1 trial

  11. Drisapersen

    BioMarin Pharmaceutical Australia Pty Ltd

    Muscular Dystrophies

    phase 3 small molecule terminated

    2 trials

  12. INZ-701

    BioMarin Pharmaceutical Australia Pty Ltd

    Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency

    phase 3 small molecule active

    2 trials

  13. Kuvan®

    BioMarin Pharmaceutical Australia Pty Ltd

    Autistic Disorder

    phase 3 small molecule completed

    1 trial

  14. N-acetylgalactosamine 4-sulfatase

    BioMarin Pharmaceutical Australia Pty Ltd

    Mucopolysaccharidosis VI

    phase 3 small molecule completed

    3 trials

  15. Palynziq 2.5 mg solution for injection in pre-filled syringe, Palynziq 20 mg solution for injection in pre-filled syringe, Palynziq 10 mg solution for injection in pre-filled syringe

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonuria (PKU)

    phase 3 small molecule active

    1 trial

  16. Pegvaliase

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonuria (PKU)

    phase 3 small molecule active

    2 trials

  17. Placebo

    BioMarin Pharmaceutical Australia Pty Ltd

    Mucopolysaccharidosis VI

    phase 3 small molecule completed

    1 trial

  18. Sapropterin Dihydrochloride

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonurias

    phase 3 small molecule completed

    3 trials

  19. Sapropterin dihydrochloride

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonuria

    phase 3 small molecule completed

    4 trials

  20. Vosoritide

    BioMarin Pharmaceutical Australia Pty Ltd

    Hypochondroplasia

    phase 3 small molecule active

    3 trials

  21. Voxzogo 0.4 mg powder and solvent for solution for injection, Voxzogo 0.56 mg powder and solvent for solution for injection, Voxzogo 1.2 mg powder and solvent for solution for injection

    BioMarin Pharmaceutical Australia Pty Ltd

    Hypochondroplasia

    phase 3 small molecule active

    1 trial

  22. Voxzogo 1.2 mg powder and solvent for solution for injection, Voxzogo 0.4 mg powder and solvent for solution for injection, Voxzogo 0.56 mg powder and solvent for solution for injection

    BioMarin Pharmaceutical Australia Pty Ltd

    Achondroplasia

    phase 3 small molecule active

    1 trial

  23. Voxzogo 1.2 mg powder and solvent for solution for injection, powder and solvent for solution for injection, Voxzogo 0.56 mg powder and solvent for solution for injection

    BioMarin Pharmaceutical Australia Pty Ltd

    Hypochondroplasia

    phase 3 small molecule active

    1 trial

  24. rhIDU (recombinant human-Alpha-L-Iduronidase)

    BioMarin Pharmaceutical Australia Pty Ltd

    Mucopolysaccharidosis I

    phase 3 mab completed

    1 trial

  25. sapropterin

    BioMarin Pharmaceutical Australia Pty Ltd

    Behavioral Manifestations of Lesch-Nyhan Disease

    phase 3 small molecule terminated

    1 trial

  26. sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonurias

    phase 3 small molecule completed

    1 trial

  27. valoctocogene roxaparvovec

    BioMarin Pharmaceutical Australia Pty Ltd

    Hemophilia A

    phase 3 mab active

    5 trials

  28. 6R BH4

    BioMarin Pharmaceutical Australia Pty Ltd

    Kidney Disease

    phase 2 small molecule completed

    1 trial

  29. 6R-BH4

    BioMarin Pharmaceutical Australia Pty Ltd

    Coronary Artery Disease

    phase 2 small molecule active

    1 trial

  30. 6R-BH4

    BioMarin Pharmaceutical Australia Pty Ltd

    Isolated Systolic Hypertension

    phase 2 small molecule terminated

    1 trial

  31. 6R-BH4 (sapropterin dihydrochloride)

    BioMarin Pharmaceutical Australia Pty Ltd

    Hypertension

    phase 2 small molecule completed

    1 trial

  32. Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.

    BioMarin Pharmaceutical Australia Pty Ltd

    Achondroplasia

    phase 2 small molecule active

    1 trial

  33. BMN 044 IV 6 mg/kg

    BioMarin Pharmaceutical Australia Pty Ltd

    Duchenne Muscular Dystrophy

    phase 2 small molecule terminated

    1 trial

  34. BMN 110

    BioMarin Pharmaceutical Australia Pty Ltd

    Mucopolysaccharidosis IVA

    phase 2 small molecule terminated

    3 trials

  35. BMN 110

    BioMarin Pharmaceutical Australia Pty Ltd

    MPS IV A

    phase 2 small molecule terminated

    2 trials

  36. BMN 165 (rAvPAL-PEG)

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonuria

    phase 2 mab completed

    1 trial

  37. BMN 190

    BioMarin Pharmaceutical Australia Pty Ltd

    Jansky-Bielschowsky Disease

    phase 2 mab completed

    2 trials

  38. BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1)

    BioMarin Pharmaceutical Australia Pty Ltd

    Jansky-Bielschowsky Disease

    phase 2 mab completed

    1 trial

  39. BMN 307

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonuria (PKU)

    phase 2 small molecule active

    1 trial

  40. BMN 351

    BioMarin Pharmaceutical Australia Pty Ltd

    Duchenne Muscular Dystrophy (DMD)

    phase 2 small molecule active

    1 trial

  41. BMN 351

    BioMarin Pharmaceutical Australia Pty Ltd

    Duchenne Muscular Dystrophy

    phase 2 small molecule active

    1 trial

  42. BMN 701

    BioMarin Pharmaceutical Australia Pty Ltd

    Pompe Disease

    phase 2 mab completed

    2 trials

  43. Dose 1 of BMN 331

    BioMarin Pharmaceutical Australia Pty Ltd

    Hereditary Angioedema

    phase 2 gene therapy active

    1 trial

  44. Exon 51 specific phosphorothioate oligonucleotide

    BioMarin Pharmaceutical Australia Pty Ltd

    Duchenne Muscular Dystrophy

    phase 2 small molecule active

    1 trial

  45. Exon 51 specific phosphorothioate oligonucleotide

    BioMarin Pharmaceutical Australia Pty Ltd

    Duchenne Muscular Dystrophy

    phase 2 small molecule active

    1 trial

  46. PRO044 SC

    BioMarin Pharmaceutical Australia Pty Ltd

    Duchenne Muscular Dystrophy

    phase 2 small molecule completed

    1 trial

  47. PRO044 SC 6 mg/kg

    BioMarin Pharmaceutical Australia Pty Ltd

    Duchenne Muscular Dystrophy

    phase 2 small molecule terminated

    1 trial

  48. PRO045, 0.15 mg/kg/week

    BioMarin Pharmaceutical Australia Pty Ltd

    Duchenne Muscular Dystrophy

    phase 2 small molecule terminated

    1 trial

Frequently asked questions

How many pipeline programs does BioMarin Pharmaceutical Australia have?

91 published programs are linked to this sponsor in our ClinicalTrials.gov index.

Is this the complete company pipeline?

This hub lists all published programs for the sponsor in our database. Partnered or out-licensed assets may appear under multiple companies.

Data attribution: Data sources · All pipeline programs

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