BioMarin Pharmaceutical Australia Clinical Trials

Company details

Status: Public
HQ: 770 Lindaro Street, San Rafael, CA 94901, US
Founded: 1997
Employees: 3,171
Programs: 91
Drugs: 36
Patents: 175

Official website ↗ All projects Drug pipeline Patents News & analysis

Recent coverage

BioMarin Rare Disease Therapy Fails Phase 3: Key Takeaways for Pharma Teams Jun 4, 2026
This Week’s Biopharma News: New Approvals, Appeals, and AI Jun 2, 2026

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Recent trial activity

8 updates

Latest clinical program signals indexed for BioMarin Pharmaceutical Australia.

Trial registry

125 studies

2023-506737-30-00
Exon 51 specific phosphorothioate oligonucleotide

Phase 2 active small_molecule

Duchenne Muscular Dystrophy

CTIS ↗
2023-507749-27-00
ROCTAVIAN 2 × 1013 vector genomes/mL solution for infusion

Approved active small_molecule

Hemophilia A

CTIS ↗
2023-508754-26-00
111-205

Phase 2 active small_molecule

Achondroplasia

CTIS ↗
2023-508864-31-00
111-302

Phase 3 active small_molecule

Achondroplasia

CTIS ↗
2024-510875-39-00
165-306

Phase 3 active small_molecule

Phenylketonuria (PKU)

CTIS ↗
2024-511656-41-00
Exon 51 specific phosphorothioate oligonucleotide

Phase 2 active small_molecule

Duchenne Muscular Dystrophy

CTIS ↗
2024-512261-14-00
111-212

Phase 2 active small_molecule

Hypochondroplasia

CTIS ↗
2024-513129-22-00
111-303

Phase 3 active small_molecule

Hypochondroplasia

CTIS ↗
2024-515861-33-00
111-211

Phase 2 active small_molecule

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

CTIS ↗
2024-517238-16-00
111-308

Phase 3 active small_molecule

Hypochondroplasia

CTIS ↗
2024-520137-74-00
111-210

Phase 2 active small_molecule

Idiopathic Short Stature (ISS)

CTIS ↗
2025-523811-12-00
333-301

Phase 3 active small_molecule

Achondroplasia

CTIS ↗
NCT00048620
N-acetylgalactosamine 4-sulfatase

Phase 3 completed small_molecule

Mucopolysaccharidosis VI

CT.gov ↗
NCT00048711
N-acetylgalactosamine 4-sulfatase

Phase 3 completed small_molecule

Mucopolysaccharidosis VI

CT.gov ↗
NCT00067470
Placebo

Phase 3 completed small_molecule

Mucopolysaccharidosis VI

CT.gov ↗
NCT00104234
N-acetylgalactosamine 4-sulfatase

Phase 3 completed small_molecule

Mucopolysaccharidosis VI

CT.gov ↗
NCT00104247
PKU-003

Phase 3 completed small_molecule

Phenylketonurias

CT.gov ↗
NCT00104260
Sapropterin Dihydrochloride

Phase 3 completed small_molecule

Phenylketonurias

CT.gov ↗
NCT00144768
laronidase

Approved completed small_molecule

Mucopolysaccharidosis I

CT.gov ↗
NCT00144781
Aldurazyme (Recombinant Human Alpha-L-Iduronidase)

Approved completed mab

Mucopolysaccharidosis I

CT.gov ↗
NCT00146757
Aldurazyme (Recombinant Human Alpha-L-Iduronidase)

Approved completed mab

Mucopolysaccharidosis I

CT.gov ↗
NCT00146770
Aldurazyme

Phase 3 completed mab

Mucopolysaccharidosis I

CT.gov ↗
NCT00225615
Sapropterin Dihydrochloride

Phase 3 completed small_molecule

Phenylketonurias

CT.gov ↗
NCT00258011
Aldurazyme (Recombinant Human Alpha-L-Iduronidase)

Approved completed mab

Mucopolysaccharidosis I

CT.gov ↗
NCT00272792
Sapropterin Dihydrochloride

Phase 3 completed small_molecule

Phenylketonurias

CT.gov ↗
NCT00299000
Naglazyme

Approved completed small_molecule

Mucopolysaccharidosis VI

CT.gov ↗
NCT00325962
6R-BH4 (sapropterin dihydrochloride)

Phase 2 completed small_molecule

Hypertension

CT.gov ↗
NCT00332189
Sapropterin dihydrochloride

Phase 3 completed small_molecule

Phenylketonuria

CT.gov ↗
NCT00355264
Phenoptin

Phase 2 completed small_molecule

Tetrahydrobiopterin Deficiencies

CT.gov ↗
NCT00403494
Sapropterin Dihydrochloride

Phase 2 completed small_molecule

Intermittent Claudication

CT.gov ↗
NCT00418821
laronidase

Approved completed small_molecule

Mucopolysaccharidosis I

CT.gov ↗
NCT00423280
6R-BH4

Phase 2 active small_molecule

Coronary Artery Disease

CT.gov ↗
NCT00435331
sapropterin dihydrochloride (6R-BH4)

Phase 1 completed small_molecule

Pulmonary Arterial Hypertension

CT.gov ↗
NCT00445978
Sapropterin Dihydrochloride

Phase 2 completed small_molecule

Sickle Cell Disease

CT.gov ↗
NCT00532844
Sapropterin Dihydrochloride

Phase 2 completed small_molecule

Endothelial Dysfunction

CT.gov ↗
NCT00625820
6R BH4

Phase 2 completed small_molecule

Kidney Disease

CT.gov ↗
NCT00634660
rAvPAL-PEG

Phase 2 completed small_molecule

Phenylketonuria

CT.gov ↗
NCT00741338
laronidase

Approved completed small_molecule

Mucopolysaccharidosis I

CT.gov ↗
NCT00789568
Sapropterin dihydrochloride

Phase 3 completed small_molecule

Phenylketonuria

CT.gov ↗
NCT00802893
6R-BH4

Phase 2 terminated small_molecule

Isolated Systolic Hypertension

CT.gov ↗
NCT00838435
Sapropterin dihydrochloride

Phase 3 completed small_molecule

Phenylketonuria

CT.gov ↗
NCT00850070
sapropterin

Phase 2 completed small_molecule

Autistic Disorder

CT.gov ↗
NCT00852358
laronidase

Unknown completed small_molecule

Cognitive Decline

CT.gov ↗
NCT00884949
BMN 110

Phase 2 terminated small_molecule

MPS IV A

CT.gov ↗
NCT00912925
rhIDU (recombinant human-Alpha-L-Iduronidase)

Phase 3 completed mab

Mucopolysaccharidosis I

CT.gov ↗
NCT00924703
rAvPAL-PEG

Phase 2 completed small_molecule

Phenylketonuria

CT.gov ↗
NCT00925054
rAvPAL-PEG 0.001 mg/kg

Phase 2 completed small_molecule

Phenylketonuria

CT.gov ↗
NCT00935753
sapropterin

Phase 3 terminated small_molecule

Behavioral Manifestations of Lesch-Nyhan Disease

CT.gov ↗
NCT00943579
Kuvan®

Phase 3 completed small_molecule

Autistic Disorder

CT.gov ↗
NCT01037309
PRO044 SC

Phase 2 completed small_molecule

Duchenne Muscular Dystrophy

CT.gov ↗
NCT01114737
Sapropterin dihydrochloride

Phase 3 completed small_molecule

Phenylketonuria

CT.gov ↗
NCT01141595
sapropterin dihydrochloride

Unknown completed small_molecule

Autism Spectrum Disorder

CT.gov ↗
NCT01212744
rAvPAL-PEG

Phase 2 completed small_molecule

Phenylketonuria

CT.gov ↗
NCT01230801
BMN 701

Phase 2 completed mab

Pompe Disease

CT.gov ↗
NCT01242111
BMN 110

Phase 2 terminated small_molecule

MPS IV A

CT.gov ↗
NCT01275066
BMN 110 Weekly

Phase 3 completed small_molecule

MPS IV A

CT.gov ↗
NCT01376908
Kuvan®

Approved completed small_molecule

Phenylketonuria

CT.gov ↗
NCT01415427
BMN 110 - Weekly

Phase 3 completed small_molecule

Mucopolysaccharidosis IV A

CT.gov ↗
NCT01425528
Sapropterin

Phase 2 completed small_molecule

GTP Cyclohydrolase Deficiency

CT.gov ↗
NCT01435772
BMN 701

Phase 2 completed mab

Pompe Disease

CT.gov ↗
NCT01515956
BMN 110

Phase 2 terminated small_molecule

Mucopolysaccharidosis IVA

CT.gov ↗
NCT01541397
Sapropterin

Unknown terminated small_molecule

Hyperphenylalaninemia

CT.gov ↗
NCT01560286
BMN 165 (rAvPAL-PEG)

Phase 2 completed mab

Phenylketonuria

CT.gov ↗
NCT01590446
BMN 111

Phase 3 active small_molecule

Achondroplasia

CT.gov ↗
NCT01609062
BMN 110

Phase 2 terminated small_molecule

Mucopolysaccharidosis IVA

CT.gov ↗
NCT01617070
Kuvan

Approved completed small_molecule

Phenylketonuria (PKU)

CT.gov ↗
NCT01697319
BMN 110

Phase 2 terminated small_molecule

Mucopolysaccharidosis IVA

CT.gov ↗
NCT01803412
Drisapersen

Phase 3 terminated small_molecule

Muscular Dystrophies

CT.gov ↗
NCT01806051
Kuvan

Approved completed small_molecule

Phenylketonuria (PKU)

CT.gov ↗
NCT01819727
BMN 165

Phase 3 completed small_molecule

Phenylketonuria

CT.gov ↗
NCT01826474
PRO045, 0.15 mg/kg/week

Phase 2 terminated small_molecule

Duchenne Muscular Dystrophy

CT.gov ↗
NCT01889862
BMN165 20mg/day

Phase 3 completed small_molecule

Phenylketonuria (PKU)

CT.gov ↗
NCT01907087
BMN 190

Phase 2 completed mab

Jansky-Bielschowsky Disease

CT.gov ↗
NCT01910649
Drisapersen

Phase 3 terminated small_molecule

Muscular Dystrophies

CT.gov ↗
NCT01924845
BMN 701

Phase 3 terminated small_molecule

Late-onset Pompe Disease

CT.gov ↗
NCT01957059
Regimen Selection Phase Group 2

Phase 2 terminated small_molecule

Duchenne Muscular Dystrophy

CT.gov ↗
NCT01965912
Kuvan®

Approved completed small_molecule

Phenylketonuria

CT.gov ↗
NCT01966029
BMN 110

Phase 3 completed small_molecule

Mucopolysaccharidosis IVA (Morquio A Syndrome)

CT.gov ↗
NCT01977820
Sapropterin

Phase 2 terminated small_molecule

Phenylketonuria

CT.gov ↗
NCT02049593
PARP inhibitor BMN-673

Phase 1 completed small_molecule

Metastatic Cancer

CT.gov ↗
NCT02055157
BMN 111

Phase 3 active small_molecule

Achondroplasia

CT.gov ↗
NCT02156674
Naglazyme®

Unknown terminated small_molecule

Maroteaux-Lamy Syndrome

CT.gov ↗
NCT02329769
PRO044 SC 6 mg/kg

Phase 2 terminated small_molecule

Duchenne Muscular Dystrophy

CT.gov ↗
NCT02353312
Kuvan

Unknown completed small_molecule

Heart Failure

CT.gov ↗
NCT02392793
Talazoparib

Phase 1 completed small_molecule

Childhood Solid Tumors

CT.gov ↗
NCT02401347
Talazoparib Tosylate

Phase 2 completed small_molecule

Advanced Breast Cancer

CT.gov ↗
NCT02485899
BMN 190

Phase 2 completed mab

Jansky-Bielschowsky Disease

CT.gov ↗
NCT02537561
Cisplatin

Phase 1 terminated small_molecule

Solid Tumors

CT.gov ↗
NCT02576795
valoctocogene roxaparvovec

Phase 2 completed mab

Severe Haemophilia A

CT.gov ↗
NCT02677870
saproterin dihydrochloride

Approved completed small_molecule

Phenylketonuria

CT.gov ↗
NCT02678689
BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1)

Phase 2 completed mab

Jansky-Bielschowsky Disease

CT.gov ↗
NCT02724228
BMN 111

Phase 3 active small_molecule

Achondroplasia

CT.gov ↗
NCT02958202
BMN 044 IV 6 mg/kg

Phase 2 terminated small_molecule

Duchenne Muscular Dystrophy

CT.gov ↗
NCT03197766
BMN 111

Phase 3 active small_molecule

Achondroplasia

CT.gov ↗
NCT03370913
valoctocogene roxaparvovec

Phase 3 active mab

Hemophilia A

CT.gov ↗
NCT03392974
valoctocogene roxaparvovec

Phase 3 active mab

Hemophilia A

CT.gov ↗
NCT03424018
BMN 111

Phase 3 active small_molecule

Achondroplasia

CT.gov ↗
NCT03520712
valoctocogene roxaparvovec

Phase 3 active mab

Hemophilia A

CT.gov ↗
NCT03583697
BMN 111

Phase 3 active small_molecule

Achondroplasia

CT.gov ↗
NCT03694353
Pegvaliase

Phase 3 active small_molecule

Phenylketonuria (PKU)

CT.gov ↗
NCT03989947
111-208

Phase 2 active small_molecule

Achondroplasia

CT.gov ↗
NCT04227080
BH4

Approved active small_molecule

Pku Phenylketonuria

CT.gov ↗
NCT04323098
valoctocogene roxaparvovec

Phase 3 active mab

Hemophilia A

CT.gov ↗
NCT04480567
BMN 307

Phase 2 active small_molecule

Phenylketonuria (PKU)

CT.gov ↗
NCT04554940
vosoritide

Phase 2 active mab

Achondroplasia

CT.gov ↗
NCT04684940
Valoctocogene roxaparvovec

Phase 2 active mab

Hemophilia A With Inhibitor

CT.gov ↗
NCT04800692
Tetrahydrobiopterin 10 mg/kg

Phase 1 active small_molecule

Claudication, Intermittent

CT.gov ↗
NCT05121376
Dose 1 of BMN 331

Phase 2 active gene_therapy

Hereditary Angioedema

CT.gov ↗
NCT05270837
Pegvaliase

Phase 3 active small_molecule

Phenylketonuria (PKU)

CT.gov ↗
NCT05734196
INZ-701

Phase 3 active small_molecule

Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency

CT.gov ↗
NCT05813314
BMN 111 administration via Injector Pen

Phase 1 terminated other

Achondroplasia

CT.gov ↗
NCT06138327
BMN 255

Phase 1 terminated small_molecule

Hyperoxaluria

CT.gov ↗
NCT06224907
valoctocogene roxaparvovec

Phase 3 active mab

Hemophilia A

CT.gov ↗
NCT06280209
BMN 351

Phase 2 active small_molecule

Duchenne Muscular Dystrophy

CT.gov ↗
NCT06382155
Vosoritide Injection

Phase 2 active small_molecule

Idiopathic Short Stature

CT.gov ↗
NCT06455059
Vosoritide

Phase 3 active small_molecule

Hypochondroplasia

CT.gov ↗
NCT06668805
Vosoritide Injection

Phase 2 active small_molecule

Noonan Syndrome

CT.gov ↗
NCT06738017
BMN 349

Phase 1 active small_molecule

Alpha 1-Antitrypsin Deficiency

CT.gov ↗
NCT06780332
RDD to Palynziq

Approved active small_molecule

Phenylketonuria

CT.gov ↗
NCT07073014
Vosoritide

Phase 3 active small_molecule

Hypochondroplasia

CT.gov ↗
NCT07126262
Vosoritide

Phase 3 active small_molecule

Hypochondroplasia

CT.gov ↗
NCT07441876
BMN 333

Phase 3 active small_molecule

Achondroplasia

CT.gov ↗
NCT07473973
INZ-701

Phase 3 active small_molecule

Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency

CT.gov ↗
NCT07477691
Pegvaliase

Approved active mab

Phenylketonuria

CT.gov ↗
NCT07573631
BMN 351

Phase 2 active small_molecule

Duchenne Muscular Dystrophy (DMD)

CT.gov ↗