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BioMarin Pharmaceutical Australia Pipeline

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

Programs
41

Quick answer

BioMarin Pharmaceutical Australia has 41 clinical development programs indexed from ClinicalTrials.gov, including phase, modality, indication text, and linked NCT identifiers.

  1. 6R BH4

    BioMarin Pharmaceutical Australia Pty Ltd

    Kidney Disease

    phase 2 small molecule completed

    1 trial

  2. 6R-BH4

    BioMarin Pharmaceutical Australia Pty Ltd

    Coronary Artery Disease

    phase 2 small molecule active

    1 trial

  3. 6R-BH4

    BioMarin Pharmaceutical Australia Pty Ltd

    Isolated Systolic Hypertension

    phase 2 small molecule terminated

    1 trial

  4. 6R-BH4 (sapropterin dihydrochloride)

    BioMarin Pharmaceutical Australia Pty Ltd

    Hypertension

    phase 2 small molecule completed

    1 trial

  5. 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

  6. BMN 044 IV 6 mg/kg

    BioMarin Pharmaceutical Australia Pty Ltd

    Duchenne Muscular Dystrophy

    phase 2 small molecule terminated

    1 trial

  7. BMN 110

    BioMarin Pharmaceutical Australia Pty Ltd

    MPS IV A

    phase 2 small molecule terminated

    2 trials

  8. BMN 110

    BioMarin Pharmaceutical Australia Pty Ltd

    Mucopolysaccharidosis IVA

    phase 2 small molecule terminated

    3 trials

  9. BMN 165 (rAvPAL-PEG)

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonuria

    phase 2 mab completed

    1 trial

  10. BMN 190

    BioMarin Pharmaceutical Australia Pty Ltd

    Jansky-Bielschowsky Disease

    phase 2 mab completed

    2 trials

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

    BioMarin Pharmaceutical Australia Pty Ltd

    Jansky-Bielschowsky Disease

    phase 2 mab completed

    1 trial

  12. BMN 307

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonuria (PKU)

    phase 2 small molecule active

    1 trial

  13. BMN 351

    BioMarin Pharmaceutical Australia Pty Ltd

    Duchenne Muscular Dystrophy

    phase 2 small molecule active

    1 trial

  14. BMN 351

    BioMarin Pharmaceutical Australia Pty Ltd

    Duchenne Muscular Dystrophy (DMD)

    phase 2 small molecule active

    1 trial

  15. BMN 701

    BioMarin Pharmaceutical Australia Pty Ltd

    Pompe Disease

    phase 2 mab completed

    2 trials

  16. Dose 1 of BMN 331

    BioMarin Pharmaceutical Australia Pty Ltd

    Hereditary Angioedema

    phase 2 gene therapy active

    1 trial

  17. Exon 51 specific phosphorothioate oligonucleotide

    BioMarin Pharmaceutical Australia Pty Ltd

    Duchenne Muscular Dystrophy

    phase 2 small molecule active

    1 trial

  18. Exon 51 specific phosphorothioate oligonucleotide

    BioMarin Pharmaceutical Australia Pty Ltd

    Duchenne Muscular Dystrophy

    phase 2 small molecule active

    1 trial

  19. PRO044 SC

    BioMarin Pharmaceutical Australia Pty Ltd

    Duchenne Muscular Dystrophy

    phase 2 small molecule completed

    1 trial

  20. PRO044 SC 6 mg/kg

    BioMarin Pharmaceutical Australia Pty Ltd

    Duchenne Muscular Dystrophy

    phase 2 small molecule terminated

    1 trial

  21. PRO045, 0.15 mg/kg/week

    BioMarin Pharmaceutical Australia Pty Ltd

    Duchenne Muscular Dystrophy

    phase 2 small molecule terminated

    1 trial

  22. Phenoptin

    BioMarin Pharmaceutical Australia Pty Ltd

    Tetrahydrobiopterin Deficiencies

    phase 2 small molecule completed

    1 trial

  23. Regimen Selection Phase Group 2

    BioMarin Pharmaceutical Australia Pty Ltd

    Duchenne Muscular Dystrophy

    phase 2 small molecule terminated

    1 trial

  24. Sapropterin

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonuria

    phase 2 small molecule terminated

    1 trial

  25. Sapropterin

    BioMarin Pharmaceutical Australia Pty Ltd

    GTP Cyclohydrolase Deficiency

    phase 2 small molecule completed

    1 trial

  26. Sapropterin Dihydrochloride

    BioMarin Pharmaceutical Australia Pty Ltd

    Intermittent Claudication

    phase 2 small molecule completed

    1 trial

  27. Sapropterin Dihydrochloride

    BioMarin Pharmaceutical Australia Pty Ltd

    Endothelial Dysfunction

    phase 2 small molecule completed

    1 trial

  28. Sapropterin Dihydrochloride

    BioMarin Pharmaceutical Australia Pty Ltd

    Sickle Cell Disease

    phase 2 small molecule completed

    1 trial

  29. Talazoparib Tosylate

    BioMarin Pharmaceutical Australia Pty Ltd

    Advanced Breast Cancer

    phase 2 small molecule completed

    1 trial

  30. Valoctocogene roxaparvovec

    BioMarin Pharmaceutical Australia Pty Ltd

    Hemophilia A With Inhibitor

    phase 2 mab active

    1 trial

  31. Vosoritide Injection

    BioMarin Pharmaceutical Australia Pty Ltd

    Idiopathic Short Stature

    phase 2 small molecule active

    1 trial

  32. Vosoritide Injection

    BioMarin Pharmaceutical Australia Pty Ltd

    Noonan Syndrome

    phase 2 small molecule active

    1 trial

  33. 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

  34. 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

  35. 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

  36. 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

  37. rAvPAL-PEG

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonuria

    phase 2 small molecule completed

    3 trials

  38. rAvPAL-PEG 0.001 mg/kg

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonuria

    phase 2 small molecule completed

    1 trial

  39. sapropterin

    BioMarin Pharmaceutical Australia Pty Ltd

    Autistic Disorder

    phase 2 small molecule completed

    1 trial

  40. valoctocogene roxaparvovec

    BioMarin Pharmaceutical Australia Pty Ltd

    Severe Haemophilia A

    phase 2 mab completed

    1 trial

  41. vosoritide

    BioMarin Pharmaceutical Australia Pty Ltd

    Achondroplasia

    phase 2 mab active

    1 trial

Frequently asked questions

How many pipeline programs does BioMarin Pharmaceutical Australia have?

41 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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