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  • News bot Aug. 11, 2026, 12:35 p.m.

    📋 ULTRAGENYX PHARMACEUTICAL INC. (RARE) - Regulatory Update

    Filing Date: 2026-08-11

    Accepted: 2026-08-11 08:30:17

    Event Type: Regulatory Update

    Event Details:

    ULTRAGENYX PHARMACEUTICAL INC. (RARE) Announces Regulatory Update ULTRAGENYX PHARMACEUTICAL INC. (RARE) provided an update on regulatory developments for its product portfolio. Regulatory Update Details: None Clinical Development:

    🔬 Clinical Development Pipeline (ULTRAGENYX PHARMACEUTICAL INC.):

    Product Type Development Stage Therapeutic Area Source
    GTX-102 Other Phase PHASE2 Angelman Syndrome ClinicalTrials.gov
    No intervention Other Phase PHASE2 Angelman Syndrome ClinicalTrials.gov
    Sham-LP Other Phase PHASE3 Angelman Syndrome ClinicalTrials.gov
    Triheptanoin Other Preclinical Long Chain Fatty Acid Oxidation Disorders ClinicalTrials.gov
    Mepsevii Other Preclinical MPS VII ClinicalTrials.gov
    Burosumab Other Phase EARLY_PHASE1 Cutaneous Skeletal Hypophosphatemia Syndrome (CSHS) ClinicalTrials.gov
    MCT Oil Other Phase PHASE3 Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) ClinicalTrials.gov
    Adjuvant Immunomodulatory (IM) Therapy Drug Phase PHASE3 Mucopolysaccharidosis IIIA ClinicalTrials.gov
    No Investigational Product Other Phase PHASE3 Mucopolysaccharidosis IIIA ClinicalTrials.gov
    Adjuvant IM Therapy Drug Phase PHASE2 MPS IIIA ClinicalTrials.gov
    Optimized Prophylactic IM Therapy Drug Phase PHASE2 MPS IIIA ClinicalTrials.gov
    UX111 Other Phase PHASE2 MPS IIIA ClinicalTrials.gov
    Prophylactic Immunomodulatory (IM) Therapy Drug Phase PHASE2 MPS IIIA ClinicalTrials.gov
    setrusumab Other Phase PHASE3 Osteogenesis Imperfecta ClinicalTrials.gov
    Standard of Care (SOC) Other Phase PHASE1 Wilson Disease ClinicalTrials.gov
    UX701 Other Phase PHASE1 Wilson Disease ClinicalTrials.gov
    Placebo Other Phase PHASE2 Huntington Disease ClinicalTrials.gov
    Triheptanoin oil Other Phase PHASE2 Huntington Disease ClinicalTrials.gov
    Sodium Acetate Other Phase PHASE3 OTC Deficiency ClinicalTrials.gov
    Placebo for oral corticosteroids Other Phase PHASE3 OTC Deficiency ClinicalTrials.gov
    Oral Corticosteroids Other Phase PHASE3 OTC Deficiency ClinicalTrials.gov
    DTX301 Other Phase PHASE3 OTC Deficiency ClinicalTrials.gov
    SA-IR 500 mg Other Phase PHASE2 GNE Myopathy ClinicalTrials.gov
    SA-ER 500 mg Other Phase PHASE2 GNE Myopathy ClinicalTrials.gov
    Sialic Acid Extended Release (SA-ER) Tablets Other Phase PHASE1 Hereditary Inclusion Body Myopathy (HIBM) ClinicalTrials.gov
    Sialic Acid Extended Release (SA-ER) Tables Other Phase PHASE1 Hereditary Inclusion Body Myopathy (HIBM) ClinicalTrials.gov
    Sialic Acid Extended Release (SA-ER) Other Phase PHASE2 GNE Myopathy ClinicalTrials.gov
    UX003 Other Preclinical Mucopolysaccharidosis Type 7 ClinicalTrials.gov
    Tridecanoic Acid Other Phase PHASE2 Rett Syndrome ClinicalTrials.gov
    DTX101 Other Phase PHASE1 Hemophilia B ClinicalTrials.gov
    Aceneuramic Acid Extended-Release Other Phase PHASE2 Hereditary Inclusion Body Myopathy ClinicalTrials.gov
    aceneuramic acid extended-release (Ace-ER) Other Phase PHASE3 Hereditary Inclusion Body Myopathy ClinicalTrials.gov
    Placebo oil Other Phase PHASE2 Glycogen Storage Disease Type V ClinicalTrials.gov
    UX007 Other Phase PHASE2 Glucose Transporter Type 1 Deficiency Syndrome ClinicalTrials.gov
    POST Triheptanoin Other Phase PHASE1 Frontal Lobe Hypometabolism ClinicalTrials.gov
    BPS804 Other Phase PHASE2 Osteogenesis Imperfecta ClinicalTrials.gov
    [6,6-2H2]glucose Other Phase PHASE1 Glycogen Storage Disease Type IA ClinicalTrials.gov
    Placebo to 20mg/Kg BPS804 Other Phase PHASE2 Osteopenia ClinicalTrials.gov
    BPS804 20mg/Kg Other Phase PHASE2 Osteopenia ClinicalTrials.gov
    Crysvita (burosumab-twza) Treatment Drug Phase PHASE4 Epidermal Nevus Syndrome ClinicalTrials.gov
    steroid regimen Other Phase PHASE1 GSD1 ClinicalTrials.gov
    DTX401 Other Phase PHASE1 GSD1 ClinicalTrials.gov
    Prophylactic Corticosteroid Taper Regimen Other Phase PHASE1 Ornithine Transcarbamylase (OTC) Deficiency ClinicalTrials.gov
    Reactive Corticosteroid Taper Regimen Other Phase PHASE1 Ornithine Transcarbamylase (OTC) Deficiency ClinicalTrials.gov
    scAAV8OTC Other Phase PHASE1 Ornithine Transcarbamylase (OTC) Deficiency ClinicalTrials.gov
    Aceneuramic Acid Extended-Release Tablets Other Phase PHASE3 Hereditary Inclusion Body Myopathy ClinicalTrials.gov
    zoledronic acid (optional) Other Phase PHASE2 Osteogenesis Imperfecta, Type I ClinicalTrials.gov
    Vitamin D Other Phase PHASE2 Osteogenesis Imperfecta, Type I ClinicalTrials.gov
    Calcium Other Phase PHASE2 Osteogenesis Imperfecta, Type I ClinicalTrials.gov
    ABO-102 Other Phase PHASE1 MPS IIIA ClinicalTrials.gov
    Newborn Screening Assay Other Preclinical Angelman Syndrome ClinicalTrials.gov
    H1 Blocker Other Phase PHASE1 Glycogen Storage Disease Type III ClinicalTrials.gov
    H2 Blocker Other Phase PHASE1 Glycogen Storage Disease Type III ClinicalTrials.gov
    Antipyretic Other Phase PHASE1 Glycogen Storage Disease Type III ClinicalTrials.gov
    UX053 Other Phase PHASE1 Glycogen Storage Disease Type III ClinicalTrials.gov
    Evinacumab Other Phase PHASE3 Homozygous Familial Hypercholesterolemia ClinicalTrials.gov
    Venipuncture Other Preclinical Mucopolysaccharidoses ClinicalTrials.gov
    Echocardiography, transthoracic Other Preclinical Mucopolysaccharidoses ClinicalTrials.gov
    Carotid ultrasonography Other Preclinical Mucopolysaccharidoses ClinicalTrials.gov
    GLUT1 DS Other Phase PHASE2 Glut1 Deficiency Syndrome ClinicalTrials.gov
    Bisphosphonate Other Phase PHASE3 Osteogenesis Imperfecta ClinicalTrials.gov
    Placebo for oral prednisolone Other Phase PHASE3 Glycogen Storage Disease Type IA ClinicalTrials.gov
    Oral prednisolone Other Phase PHASE3 Glycogen Storage Disease Type IA ClinicalTrials.gov
    UX016 DRUG Phase PHASE1 GNE Myopathy ClinicalTrials.gov
    Vegetable Oil OTHER Phase PHASE2 Adult Polyglucosan Body Disease ClinicalTrials.gov

    💼 Business Developments:

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

    Structured Data:

    • Company Name: ULTRAGENYX PHARMACEUTICAL INC.
    • Ticker Symbol: RARE

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