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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. Phenoptin

    BioMarin Pharmaceutical Australia Pty Ltd

    Tetrahydrobiopterin Deficiencies

    phase 2 small molecule completed

    1 trial

  2. Regimen Selection Phase Group 2

    BioMarin Pharmaceutical Australia Pty Ltd

    Duchenne Muscular Dystrophy

    phase 2 small molecule terminated

    1 trial

  3. Sapropterin

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonuria

    phase 2 small molecule terminated

    1 trial

  4. Sapropterin

    BioMarin Pharmaceutical Australia Pty Ltd

    GTP Cyclohydrolase Deficiency

    phase 2 small molecule completed

    1 trial

  5. Sapropterin Dihydrochloride

    BioMarin Pharmaceutical Australia Pty Ltd

    Endothelial Dysfunction

    phase 2 small molecule completed

    1 trial

  6. Sapropterin Dihydrochloride

    BioMarin Pharmaceutical Australia Pty Ltd

    Sickle Cell Disease

    phase 2 small molecule completed

    1 trial

  7. Sapropterin Dihydrochloride

    BioMarin Pharmaceutical Australia Pty Ltd

    Intermittent Claudication

    phase 2 small molecule completed

    1 trial

  8. Talazoparib Tosylate

    BioMarin Pharmaceutical Australia Pty Ltd

    Advanced Breast Cancer

    phase 2 small molecule completed

    1 trial

  9. Valoctocogene roxaparvovec

    BioMarin Pharmaceutical Australia Pty Ltd

    Hemophilia A With Inhibitor

    phase 2 mab active

    1 trial

  10. Vosoritide Injection

    BioMarin Pharmaceutical Australia Pty Ltd

    Idiopathic Short Stature

    phase 2 small molecule active

    1 trial

  11. Vosoritide Injection

    BioMarin Pharmaceutical Australia Pty Ltd

    Noonan Syndrome

    phase 2 small molecule active

    1 trial

  12. 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

    Achondroplasia

    phase 2 small molecule active

    1 trial

  13. Voxzogo 0.56 mg powder and solvent for solution for injection, modified recombinant human C-type natriuretic peptide (rhCNP), Lyophilized powder for reconstitution, Voxzogo 1.2 mg powder and solvent for solution for injection

    BioMarin Pharmaceutical Australia Pty Ltd

    Idiopathic Short Stature (ISS)

    phase 2 small molecule active

    1 trial

  14. Voxzogo 0.56 mg powder and solvent for solution for injection, powder and solvent for solution for injection

    BioMarin Pharmaceutical Australia Pty Ltd

    Hypochondroplasia

    phase 2 small molecule active

    1 trial

  15. Voxzogo 1.2 mg powder and solvent for solution for injection, Voxzogo 0.56 mg powder and solvent for solution for injection, modified recombinant human C-type natriuretic peptide (rhCNP)

    BioMarin Pharmaceutical Australia Pty Ltd

    Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome

    phase 2 small molecule active

    1 trial

  16. rAvPAL-PEG

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonuria

    phase 2 small molecule completed

    3 trials

  17. rAvPAL-PEG 0.001 mg/kg

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonuria

    phase 2 small molecule completed

    1 trial

  18. sapropterin

    BioMarin Pharmaceutical Australia Pty Ltd

    Autistic Disorder

    phase 2 small molecule completed

    1 trial

  19. valoctocogene roxaparvovec

    BioMarin Pharmaceutical Australia Pty Ltd

    Severe Haemophilia A

    phase 2 mab completed

    1 trial

  20. vosoritide

    BioMarin Pharmaceutical Australia Pty Ltd

    Achondroplasia

    phase 2 mab active

    1 trial

  21. BMN 111 administration via Injector Pen

    BioMarin Pharmaceutical Australia Pty Ltd

    Achondroplasia

    phase 1 other terminated

    1 trial

  22. BMN 255

    BioMarin Pharmaceutical Australia Pty Ltd

    Hyperoxaluria

    phase 1 small molecule terminated

    1 trial

  23. BMN 349

    BioMarin Pharmaceutical Australia Pty Ltd

    Alpha 1-Antitrypsin Deficiency

    phase 1 small molecule active

    1 trial

  24. Cisplatin

    BioMarin Pharmaceutical Australia Pty Ltd

    Solid Tumors

    phase 1 small molecule terminated

    1 trial

  25. PARP inhibitor BMN-673

    BioMarin Pharmaceutical Australia Pty Ltd

    Metastatic Cancer

    phase 1 small molecule completed

    1 trial

  26. Talazoparib

    BioMarin Pharmaceutical Australia Pty Ltd

    Childhood Solid Tumors

    phase 1 small molecule completed

    1 trial

  27. Tetrahydrobiopterin 10 mg/kg

    BioMarin Pharmaceutical Australia Pty Ltd

    Claudication, Intermittent

    phase 1 small molecule active

    1 trial

  28. sapropterin dihydrochloride (6R-BH4)

    BioMarin Pharmaceutical Australia Pty Ltd

    Pulmonary Arterial Hypertension

    phase 1 small molecule completed

    1 trial

  29. Aldurazyme (Recombinant Human Alpha-L-Iduronidase)

    BioMarin Pharmaceutical Australia Pty Ltd

    Mucopolysaccharidosis I

    approved mab completed

    3 trials

  30. BH4

    BioMarin Pharmaceutical Australia Pty Ltd

    Pku Phenylketonuria

    approved small molecule active

    1 trial

  31. Kuvan

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonuria (PKU)

    approved small molecule completed

    2 trials

  32. Kuvan®

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonuria

    approved small molecule completed

    2 trials

  33. Naglazyme

    BioMarin Pharmaceutical Australia Pty Ltd

    Mucopolysaccharidosis VI

    approved small molecule completed

    1 trial

  34. Pegvaliase

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonuria

    approved mab active

    1 trial

  35. RDD to Palynziq

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonuria

    approved small molecule active

    1 trial

  36. ROCTAVIAN 2 × 1013 vector genomes/mL solution for infusion

    BioMarin Pharmaceutical Australia Pty Ltd

    Hemophilia A

    approved small molecule active

    1 trial

  37. laronidase

    BioMarin Pharmaceutical Australia Pty Ltd

    Mucopolysaccharidosis I

    approved small molecule completed

    3 trials

  38. saproterin dihydrochloride

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonuria

    approved small molecule completed

    1 trial

  39. Kuvan

    BioMarin Pharmaceutical Australia Pty Ltd

    Heart Failure

    small molecule completed

    1 trial

  40. Naglazyme®

    BioMarin Pharmaceutical Australia Pty Ltd

    Maroteaux-Lamy Syndrome

    small molecule terminated

    1 trial

  41. Sapropterin

    BioMarin Pharmaceutical Australia Pty Ltd

    Hyperphenylalaninemia

    small molecule terminated

    1 trial

  42. laronidase

    BioMarin Pharmaceutical Australia Pty Ltd

    Cognitive Decline

    small molecule completed

    1 trial

  43. sapropterin dihydrochloride

    BioMarin Pharmaceutical Australia Pty Ltd

    Autism Spectrum Disorder

    small molecule completed

    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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