Wednesday, June 10, 2026

pharma · Phenylketonuria · Achondroplasia

BioMarin Pharmaceutical Australia

BioMarin Pharmaceutical Australia clinical programs — 27 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

27 ClinicalTrials.gov programs sponsored by BioMarin Pharmaceutical Australia.

BioMarin Pharmaceutical Australia has 27 clinical development programs indexed from ClinicalTrials.gov, including phase, modality, indication text, and linked NCT identifiers.

Clinical programs

  1. 111-302

    BioMarin Pharmaceutical Australia Pty Ltd

    Achondroplasia

    phase 3 small molecule active

    1 trial
  2. 111-303

    BioMarin Pharmaceutical Australia Pty Ltd

    Hypochondroplasia

    phase 3 small molecule active

    1 trial
  3. 111-308

    BioMarin Pharmaceutical Australia Pty Ltd

    Hypochondroplasia

    phase 3 small molecule active

    1 trial
  4. 165-306

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonuria (PKU)

    phase 3 small molecule active

    1 trial
  5. 333-301

    BioMarin Pharmaceutical Australia Pty Ltd

    Achondroplasia

    phase 3 small molecule active

    1 trial
  6. Aldurazyme

    BioMarin Pharmaceutical Australia Pty Ltd

    Mucopolysaccharidosis I

    phase 3 mab completed

    1 trial
  7. BMN 110

    BioMarin Pharmaceutical Australia Pty Ltd

    Mucopolysaccharidosis IVA (Morquio A Syndrome)

    phase 3 small molecule completed

    1 trial
  8. BMN 110 - Weekly

    BioMarin Pharmaceutical Australia Pty Ltd

    Mucopolysaccharidosis IV A

    phase 3 small molecule completed

    1 trial
  9. BMN 110 Weekly

    BioMarin Pharmaceutical Australia Pty Ltd

    MPS IV A

    phase 3 small molecule completed

    1 trial
  10. BMN 111

    BioMarin Pharmaceutical Australia Pty Ltd

    Achondroplasia

    phase 3 small molecule active

    6 trials
  11. BMN 165

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonuria

    phase 3 small molecule completed

    1 trial
  12. BMN 333

    BioMarin Pharmaceutical Australia Pty Ltd

    Achondroplasia

    phase 3 small molecule active

    1 trial
  13. BMN 701

    BioMarin Pharmaceutical Australia Pty Ltd

    Late-onset Pompe Disease

    phase 3 small molecule terminated

    1 trial
  14. BMN165 20mg/day

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonuria (PKU)

    phase 3 small molecule completed

    1 trial
  15. Drisapersen

    BioMarin Pharmaceutical Australia Pty Ltd

    Muscular Dystrophies

    phase 3 small molecule terminated

    2 trials
  16. INZ-701

    BioMarin Pharmaceutical Australia Pty Ltd

    Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency

    phase 3 small molecule active

    2 trials
  17. Kuvan®

    BioMarin Pharmaceutical Australia Pty Ltd

    Autistic Disorder

    phase 3 small molecule completed

    1 trial
  18. N-acetylgalactosamine 4-sulfatase

    BioMarin Pharmaceutical Australia Pty Ltd

    Mucopolysaccharidosis VI

    phase 3 small molecule completed

    3 trials
  19. PKU-003

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonurias

    phase 3 small molecule completed

    1 trial
  20. Pegvaliase

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonuria (PKU)

    phase 3 small molecule active

    2 trials
  21. Placebo

    BioMarin Pharmaceutical Australia Pty Ltd

    Mucopolysaccharidosis VI

    phase 3 small molecule completed

    1 trial
  22. Sapropterin Dihydrochloride

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonurias

    phase 3 small molecule completed

    3 trials
  23. Sapropterin dihydrochloride

    BioMarin Pharmaceutical Australia Pty Ltd

    Phenylketonuria

    phase 3 small molecule completed

    4 trials
  24. Vosoritide

    BioMarin Pharmaceutical Australia Pty Ltd

    Hypochondroplasia

    phase 3 small molecule active

    3 trials
  25. rhIDU (recombinant human-Alpha-L-Iduronidase)

    BioMarin Pharmaceutical Australia Pty Ltd

    Mucopolysaccharidosis I

    phase 3 mab completed

    1 trial
  26. sapropterin

    BioMarin Pharmaceutical Australia Pty Ltd

    Behavioral Manifestations of Lesch-Nyhan Disease

    phase 3 small molecule terminated

    1 trial
  27. valoctocogene roxaparvovec

    BioMarin Pharmaceutical Australia Pty Ltd

    Hemophilia A

    phase 3 mab active

    5 trials

Frequently asked questions

How many clinical projects does BioMarin Pharmaceutical Australia have?

27 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.