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  • News bot Oct. 1, 2025, 10:53 p.m.

    📋 BIOMARIN PHARMACEUTICAL INC (BMRN) - Regulatory Update

    Filing Date: 2022-05-31

    Accepted: 2022-05-31 15:01:00

    Event Type: Regulatory Update

    Event Details:

    Biomarin Pharmaceutical Inc (BMRN) Announces Regulatory Update Biomarin Pharmaceutical Inc (BMRN) provided an update on regulatory developments for its product portfolio. Regulatory Update Details: None Clinical Development:

    🔬 Clinical Development Pipeline (BIOMARIN PHARMACEUTICAL INC):

    Product Type Development Stage Therapeutic Area Source
    Diet Only OTHER Phase PHASE3 Phenylketonuria (PKU) ClinicalTrials.gov
    Pegvaliase DRUG Phase PHASE3 Phenylketonuria (PKU) ClinicalTrials.gov
    sapropterin dihydrochloride DRUG Phase PHASE2 Phenylketonurias ClinicalTrials.gov
    N-acetylgalactosamine 4-sulfatase DRUG Phase PHASE1 Mucopolysaccharidosis VI ClinicalTrials.gov
    6R-BH4 DRUG Phase PHASE2 Coronary Artery Disease ClinicalTrials.gov
    6R-BH4 (sapropterin dihydrochloride) DRUG Phase PHASE2 Hypertension ClinicalTrials.gov
    Placebo/rhASB DRUG Phase PHASE3 Mucopolysaccharidosis VI ClinicalTrials.gov
    Placebo DRUG Phase PHASE3 Mucopolysaccharidosis VI ClinicalTrials.gov
    Kuvan DRUG Preclinical Phenylketonuria ClinicalTrials.gov
    sapropterin dihydrochloride (6R-BH4) DRUG Phase PHASE1 Pulmonary Arterial Hypertension ClinicalTrials.gov
    Naglazyme DRUG Phase PHASE4 Mucopolysaccharidosis VI ClinicalTrials.gov
    Normal Saline DRUG Phase PHASE1 Achondroplasia ClinicalTrials.gov
    BMN 111 DRUG Phase PHASE1 Achondroplasia ClinicalTrials.gov
    BMN 110 DRUG Phase PHASE2 Mucopolysaccharidosis IVA ClinicalTrials.gov
    BMN 110 Every Other Week DRUG Phase PHASE3 MPS IV A ClinicalTrials.gov
    BMN 110 Weekly DRUG Phase PHASE3 MPS IV A ClinicalTrials.gov
    Enzyme testing OTHER Preclinical Mucopolysaccharidosis IV A ClinicalTrials.gov
    sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin DRUG Phase PHASE3 Phenylketonurias ClinicalTrials.gov
    Drisapersen DRUG Phase PHASE2 Muscular Dystrophies ClinicalTrials.gov
    6R BH4 DRUG Phase PHASE2 Kidney Disease ClinicalTrials.gov
    KuvanTM Therapy DRUG Preclinical Phenylketonuria ClinicalTrials.gov
    laronidase DRUG Approved Cognitive Decline ClinicalTrials.gov
    Talazoparib DRUG Phase PHASE1 Solid Tumors ClinicalTrials.gov
    Gemcitabine DRUG Phase PHASE1 Solid Tumors ClinicalTrials.gov
    Cisplatin DRUG Phase PHASE1 Solid Tumors ClinicalTrials.gov
    Sapropterin DRUG Approved Hyperphenylalaninemia ClinicalTrials.gov
    PRO045, selected dose DRUG Phase PHASE1 Duchenne Muscular Dystrophy ClinicalTrials.gov
    PRO045, 9.0 mg/kg/week DRUG Phase PHASE1 Duchenne Muscular Dystrophy ClinicalTrials.gov
    PRO045, 6.0 mg/kg/week DRUG Phase PHASE1 Duchenne Muscular Dystrophy ClinicalTrials.gov
    PRO045, 3.0 mg/kg/week DRUG Phase PHASE1 Duchenne Muscular Dystrophy ClinicalTrials.gov
    PRO045, 1.0 mg/kg/week DRUG Phase PHASE1 Duchenne Muscular Dystrophy ClinicalTrials.gov
    PRO045, 0.15 mg/kg/week DRUG Phase PHASE1 Duchenne Muscular Dystrophy ClinicalTrials.gov
    PRO044 IV 9 mg/kg DRUG Phase PHASE2 Duchenne Muscular Dystrophy ClinicalTrials.gov
    PRO044 IV 6 mg/kg DRUG Phase PHASE2 Duchenne Muscular Dystrophy ClinicalTrials.gov
    PRO044 SC 6 mg/kg DRUG Phase PHASE2 Duchenne Muscular Dystrophy ClinicalTrials.gov
    Dosing Extension DRUG Phase PHASE1 Duchenne Muscular Dystrophy ClinicalTrials.gov
    Regimen Selection Phase Group 1 (COMPLETED) DRUG Phase PHASE1 Duchenne Muscular Dystrophy ClinicalTrials.gov
    Treatment Phase Group 4 DRUG Phase PHASE1 Duchenne Muscular Dystrophy ClinicalTrials.gov
    Regimen Selection Phase Group 3 DRUG Phase PHASE1 Duchenne Muscular Dystrophy ClinicalTrials.gov
    Regimen Selection Phase Group 2 DRUG Phase PHASE1 Duchenne Muscular Dystrophy ClinicalTrials.gov
    BMN 701 BIOLOGICAL Phase PHASE2 Pompe Disease ClinicalTrials.gov
    Kuvan® DRUG Phase PHASE2 Autistic Disorder ClinicalTrials.gov
    Alpha 1-Antitrypsin (AAT, Aralast NP) DRUG Phase PHASE1 Diabetes ClinicalTrials.gov
    Sapropterin (Kuvan) DRUG Preclinical Phenylketonuria ClinicalTrials.gov
    BMN190, recombinant human tripeptidyl peptidase-1 (rhTPP1) DRUG Preclinical CLN2 Disease ClinicalTrials.gov
    Phenylalanine (Phe)-restricted diet OTHER Phase PHASE3 Phenylketonuria ClinicalTrials.gov
    Observational study OTHER Preclinical Duchenne Muscular Dystrophy ClinicalTrials.gov
    rAvPAL-PEG DRUG Phase PHASE1 Phenylketonuria ClinicalTrials.gov
    BMN 044 SC 6 mg/kg DRUG Phase PHASE2 Duchenne Muscular Dystrophy ClinicalTrials.gov
    BMN 044 IV 9 mg/kg DRUG Phase PHASE2 Duchenne Muscular Dystrophy ClinicalTrials.gov
    BMN 044 IV 6 mg/kg DRUG Phase PHASE2 Duchenne Muscular Dystrophy ClinicalTrials.gov
    PRO044 IV DRUG Phase PHASE1 Duchenne Muscular Dystrophy ClinicalTrials.gov
    PRO044 SC DRUG Phase PHASE1 Duchenne Muscular Dystrophy ClinicalTrials.gov
    URC102 DRUG Phase PHASE1 Healthy ClinicalTrials.gov
    Phenoptin DRUG Phase PHASE2 Tetrahydrobiopterin Deficiencies ClinicalTrials.gov
    BMN 190 BIOLOGICAL Phase PHASE1 Jansky-Bielschowsky Disease ClinicalTrials.gov
    Ferinject (Ferric Carboxymaltose) DRUG Phase PHASE2 Pancreatic Cancer ClinicalTrials.gov
    SOC Tissue biopsy PROCEDURE Preclinical Healthy Volunteers ClinicalTrials.gov
    Phlebotomy - Blood sampling for GP88 at MM OTHER Preclinical Healthy Volunteers ClinicalTrials.gov
    Study drug DRUG Phase PHASE1 Healthy ClinicalTrials.gov
    BMN 165 DRUG Phase PHASE3 Phenylketonuria ClinicalTrials.gov
    BH4 DRUG Phase PHASE4 Pku Phenylketonuria ClinicalTrials.gov
    BMN 165 (rAvPAL-PEG) BIOLOGICAL Phase PHASE2 Phenylketonuria ClinicalTrials.gov
    Administration of CANTAB and Subject Global Assessment OTHER Preclinical Phenylketonuria ClinicalTrials.gov
    Phase 1b: CWP232291, Lenalidomide, Dexamethasone DRUG Phase PHASE1 Multiple Myeloma ClinicalTrials.gov
    Phase 1a: CWP232291 DRUG Phase PHASE1 Multiple Myeloma ClinicalTrials.gov
    Diet OTHER Preclinical Phenylketonurias ClinicalTrials.gov
    rAvPAL-PEG 0.1 mg/kg DRUG Phase PHASE2 Phenylketonuria ClinicalTrials.gov
    rAvPAL-PEG 0.03 mg/kg DRUG Phase PHASE2 Phenylketonuria ClinicalTrials.gov
    rAvPAL-PEG 0.01 mg/kg DRUG Phase PHASE2 Phenylketonuria ClinicalTrials.gov
    rAvPAL-PEG 0.003 mg/kg DRUG Phase PHASE2 Phenylketonuria ClinicalTrials.gov
    rAvPAL-PEG 0.001 mg/kg DRUG Phase PHASE2 Phenylketonuria ClinicalTrials.gov
    Atorvastatin DRUG Phase PHASE4 Hypercholesterolemia With Type2DM ClinicalTrials.gov
    Pitavastatin DRUG Phase PHASE4 Hypercholesterolemia With Type2DM ClinicalTrials.gov
    Skin Tape OTHER Preclinical Phenylketonurias ClinicalTrials.gov
    Moxifloxacin placebo DRUG Phase PHASE1 Phenylketonuria ClinicalTrials.gov
    Moxifloxacin DRUG Phase PHASE1 Phenylketonuria ClinicalTrials.gov
    Naglazyme® DRUG Approved Maroteaux-Lamy Syndrome ClinicalTrials.gov
    Peg-filgrastim DRUG Phase PHASE1 Childhood Solid Tumors ClinicalTrials.gov
    Filgrastim DRUG Phase PHASE1 Childhood Solid Tumors ClinicalTrials.gov
    Temozolomide DRUG Phase PHASE1 Childhood Solid Tumors ClinicalTrials.gov
    Irinotecan DRUG Phase PHASE1 Childhood Solid Tumors ClinicalTrials.gov
    BMN 110 - Every Other Week DRUG Phase PHASE3 Mucopolysaccharidosis IV A ClinicalTrials.gov
    BMN 110 - Weekly DRUG Phase PHASE3 Mucopolysaccharidosis IV A ClinicalTrials.gov
    Interview OTHER Preclinical Morquio Disease ClinicalTrials.gov
    BMN165 40mg/day DRUG Phase PHASE3 Phenylketonuria (PKU) ClinicalTrials.gov
    BMN165 20mg/day DRUG Phase PHASE3 Phenylketonuria (PKU) ClinicalTrials.gov
    Sapropterin Dihydrochloride and Vitamin C DRUG Phase PHASE2 Endothelial Dysfunction ClinicalTrials.gov
    Intracerebroventricular (ICV) access device DEVICE Phase PHASE1 Jansky-Bielschowsky Disease ClinicalTrials.gov
    laboratory biomarker analysis OTHER Phase PHASE1 Metastatic Cancer ClinicalTrials.gov
    pharmacological study OTHER Phase PHASE1 Metastatic Cancer ClinicalTrials.gov
    irinotecan hydrochloride DRUG Phase PHASE1 Metastatic Cancer ClinicalTrials.gov
    PARP inhibitor BMN-673 DRUG Phase PHASE1 Metastatic Cancer ClinicalTrials.gov
    Diet treatment OTHER Phase PHASE4 Phenylketonuria ClinicalTrials.gov
    saproterin dihydrochloride DRUG Phase PHASE4 Phenylketonuria ClinicalTrials.gov
    Islet isolation using BMX-010 DRUG Phase PHASE1 Type 1 Diabetes ClinicalTrials.gov
    Talazoparib Tosylate DRUG Phase PHASE2 Advanced Breast Cancer ClinicalTrials.gov
    CLN2 Treatment OTHER Preclinical Batten Disease ClinicalTrials.gov
    Respiratory muscle strength measurements by different techniques. PROCEDURE Preclinical Late-onset Pompe Disease ClinicalTrials.gov
    BMN 255 DRUG Phase PHASE1 Hyperoxaluria ClinicalTrials.gov
    Respiratory Muscle Endurance Test OTHER Preclinical Pompe Disease ClinicalTrials.gov
    Resting Breathing Pattern OTHER Preclinical Pompe Disease ClinicalTrials.gov
    Maximal Inspiratory Pressure OTHER Preclinical Pompe Disease ClinicalTrials.gov
    Inspiratory Load Compensation OTHER Preclinical Pompe Disease ClinicalTrials.gov
    Spirometry OTHER Preclinical Pompe Disease ClinicalTrials.gov
    Thoracic MRI OTHER Preclinical Pompe Disease ClinicalTrials.gov
    Pegvaliase-Pqpz DRUG Preclinical Phenylketonurias ClinicalTrials.gov
    Valoctocogene Roxaparvovec BIOLOGICAL Phase PHASE3 Hemophilia A ClinicalTrials.gov
    Elosulfase Alfa 1 MG/ML Intravenous Solution [VIMIZIM] DRUG Preclinical Mucopolysaccharidosis IV A ClinicalTrials.gov
    Vimizim® (elosulfase alfa) DRUG Preclinical Mucopolysaccharidosis IV Type A ClinicalTrials.gov
    Blood sample collection PROCEDURE Preclinical Hemophilia A ClinicalTrials.gov
    BMN 111 administration via vial and syringe DRUG Phase PHASE1 Achondroplasia ClinicalTrials.gov
    Questionnaire and interview BEHAVIORAL Preclinical Hemophilia ClinicalTrials.gov
    Dose 7 of BMN 331 GENETIC Phase PHASE1 Hereditary Angioedema ClinicalTrials.gov
    Dose 6 of BMN 331 GENETIC Phase PHASE1 Hereditary Angioedema ClinicalTrials.gov
    Dose 5 of BMN 331 GENETIC Phase PHASE1 Hereditary Angioedema ClinicalTrials.gov
    Dose 4 of BMN 331 GENETIC Phase PHASE1 Hereditary Angioedema ClinicalTrials.gov
    Dose 3 of BMN 331 GENETIC Phase PHASE1 Hereditary Angioedema ClinicalTrials.gov
    Dose 2 of BMN 331 GENETIC Phase PHASE1 Hereditary Angioedema ClinicalTrials.gov
    Dose 1 of BMN 331 GENETIC Phase PHASE1 Hereditary Angioedema ClinicalTrials.gov
    Biospecimen Collection PROCEDURE Approved Hemophilia A ClinicalTrials.gov
    Administration Kit DEVICE Preclinical Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 ClinicalTrials.gov
    Cerliponase Alfa DRUG Preclinical Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 ClinicalTrials.gov
    L-Arginine DRUG Phase PHASE1 Claudication, Intermittent ClinicalTrials.gov
    L-Ascorbate DRUG Phase PHASE1 Claudication, Intermittent ClinicalTrials.gov
    Tetrahydrobiopterin 20 mg/kg DRUG Phase PHASE1 Claudication, Intermittent ClinicalTrials.gov
    Tetrahydrobiopterin 10 mg/kg DRUG Phase PHASE1 Claudication, Intermittent ClinicalTrials.gov
    Human Growth Hormone DRUG Phase PHASE2 Idiopathic Short Stature ClinicalTrials.gov
    Vosoritide Injection DRUG Phase PHASE2 Idiopathic Short Stature ClinicalTrials.gov
    Vosoritide DRUG Phase PHASE3 Hypochondroplasia ClinicalTrials.gov
    Palynziq DRUG Preclinical Phenylketonurias ClinicalTrials.gov
    BMN 349 DRUG Phase PHASE1 Alpha 1-Antitrypsin Deficiency ClinicalTrials.gov
    BMN 351 DRUG Phase PHASE1 Duchenne Muscular Dystrophy ClinicalTrials.gov
    Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily. DRUG Phase PHASE2 Achondroplasia ClinicalTrials.gov
    BMN 307 DRUG Phase PHASE1 Phenylketonuria (PKU) ClinicalTrials.gov
    RDD to Palynziq DRUG Phase PHASE4 Phenylketonuria ClinicalTrials.gov
    Intracerebroventricular access device DEVICE Phase PHASE2 Jansky-Bielschowsky Disease ClinicalTrials.gov
    BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1) BIOLOGICAL Phase PHASE2 Jansky-Bielschowsky Disease ClinicalTrials.gov
    JW0108 + C2407 placebo DRUG Phase PHASE3 Dyslipidemia ClinicalTrials.gov
    JW0108 + C2407 DRUG Phase PHASE3 Dyslipidemia ClinicalTrials.gov

    💼 Business Developments:

    • Partnership: Not available
    • Acquisition: Not available
    • Licensing: Not available
    • Regulatory Approval
    • Executive Changes: Not available

    Structured Data:

    • Company Name: Biomarin Pharmaceutical Inc
    • CIK: 0001048477
    • Ticker Symbol: BMRN
    • Period End Date: 2022-05-24
    • Document Type: 8-K

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