Wednesday, June 10, 2026

pharma · Phenylketonuria · Achondroplasia

BioMarin Pharmaceutical Australia

BioMarin Pharmaceutical Australia clinical programs — 91 development programs from ClinicalTrials.gov with phase, modality, indication, and trial identifiers.

770 Lindaro Street, San Rafael, CA 94901, US HQ
1997 Founded
3,171 Employees
TGA registrant Type
Company details
Status
Public
HQ
770 Lindaro Street, San Rafael, CA 94901, US
Founded
1997
Employees
3,171
Programs
91
Drugs
36
Patents
175
Intelligence · Clinical Programs

BioMarin Pharmaceutical Australia projects

91 ClinicalTrials.gov programs sponsored by BioMarin Pharmaceutical Australia.

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

Clinical programs

Page 2 of 2
  1. Exon 51 specific phosphorothioate oligonucleotide

    BioMarin Pharmaceutical Australia Pty Ltd

    Duchenne Muscular Dystrophy

    phase 2 small molecule active

    1 trial
  2. PRO044 SC

    BioMarin Pharmaceutical Australia Pty Ltd

    Duchenne Muscular Dystrophy

    phase 2 small molecule completed

    1 trial
  3. PRO044 SC 6 mg/kg

    BioMarin Pharmaceutical Australia Pty Ltd

    Duchenne Muscular Dystrophy

    phase 2 small molecule terminated

    1 trial
  4. PRO045, 0.15 mg/kg/week

    BioMarin Pharmaceutical Australia Pty Ltd

    Duchenne Muscular Dystrophy

    phase 2 small molecule terminated

    1 trial
  5. Phenoptin

    BioMarin Pharmaceutical Australia Pty Ltd

    Tetrahydrobiopterin Deficiencies

    phase 2 small molecule completed

    1 trial
  6. Regimen Selection Phase Group 2

    BioMarin Pharmaceutical Australia Pty Ltd

    Duchenne Muscular Dystrophy

    phase 2 small molecule terminated

    1 trial
  7. Sapropterin

    BioMarin Pharmaceutical Australia Pty Ltd

    GTP Cyclohydrolase Deficiency

    phase 2 small molecule completed

    1 trial
  8. Sapropterin

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonuria

    phase 2 small molecule terminated

    1 trial
  9. Sapropterin Dihydrochloride

    BioMarin Pharmaceutical Australia Pty Ltd

    Sickle Cell Disease

    phase 2 small molecule completed

    1 trial
  10. Sapropterin Dihydrochloride

    BioMarin Pharmaceutical Australia Pty Ltd

    Endothelial Dysfunction

    phase 2 small molecule completed

    1 trial
  11. Sapropterin Dihydrochloride

    BioMarin Pharmaceutical Australia Pty Ltd

    Intermittent Claudication

    phase 2 small molecule completed

    1 trial
  12. Talazoparib Tosylate

    BioMarin Pharmaceutical Australia Pty Ltd

    Advanced Breast Cancer

    phase 2 small molecule completed

    1 trial
  13. Valoctocogene roxaparvovec

    BioMarin Pharmaceutical Australia Pty Ltd

    Hemophilia A With Inhibitor

    phase 2 mab active

    1 trial
  14. Vosoritide Injection

    BioMarin Pharmaceutical Australia Pty Ltd

    Idiopathic Short Stature

    phase 2 small molecule active

    1 trial
  15. Vosoritide Injection

    BioMarin Pharmaceutical Australia Pty Ltd

    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 clinical projects 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 directory lists published programs for the sponsor in our database. Partnered or out-licensed assets may appear under multiple companies.