| Symbol | BMRN |
|---|---|
| Name | BIOMARIN PHARMACEUTICAL INC |
| Sector | HEALTH CARE |
| Region | North America |
| Industry | Pharmaceutical Preparations |
| Address | 105 DIGITAL DRIVE, NOVATO, CA 94949 |
| Telephone | 4155066700 |
| Fax | — |
| — | |
| Website | https://www.biomarin.com |
| Incorporation | UNDEFINED |
| Incorporated On | — |
| Employees | — |
| Fiscal Year | — |
| Public Since | — |
| Exchanges | NASDAQ |
| Auditor | — |
| Audit Status | NOT PROVIDED |
| Reporting Status | — |
| CIK | 0001048477 |
| Description | BioMarins focus is on rare-disease therapies. Genzyme (now part of Sanofi) markets Aldurazyme through its joint venture with BioMarin, and BioMarin markets Naglazyme, Vimizim, and Brineura independently. BioMarin also markets Kuvan and Palynziq to treat the rare metabolic disorder PKU (in addition to long-standing U.S. rights, BioMarin has reacquired international rights for Kuvan and Palynziq from Merck KGaA). Voxzogo (vosoritide) was approved in achondroplasia in 2021. BioMarins Roctavian (hemophilia A gene therapy) is poised to potentially launch in the 2022-23 timeframe. Additional info from NASDAQ: |
2026-06-04 22:10
Director Countouriotis Athena 🟢 acquired 7.5K shares of BIOMARIN PHARMACEUTICAL INC (BMRN) Transaction Date: Jun 02, 2026 | Filing ID: 040922
Read more
2026-06-04 22:09
Director Walbert Timothy P 🟢 acquired 7.5K shares of BIOMARIN PHARMACEUTICAL INC (BMRN) Transaction Date: Jun 02, 2026 | Filing ID: 040920
Read more
2026-06-04 22:07
Director Ho Maykin 🟢 acquired 7.5K shares of BIOMARIN PHARMACEUTICAL INC (BMRN) Transaction Date: Jun 02, 2026 | Filing ID: 040918
Read more
2026-06-04 22:06
Director HOMBACH ROBERT J. 🟢 acquired 7.5K shares of BIOMARIN PHARMACEUTICAL INC (BMRN) Transaction Date: Jun 02, 2026 | Filing ID: 040916
Read more
2026-06-04 22:05
Director Enyedy Mark J 🟢 acquired 7.5K shares of BIOMARIN PHARMACEUTICAL INC (BMRN) Transaction Date: Jun 02, 2026 | Filing ID: 040914
Read more
2026-06-04 22:04
Director Dere Willard H 🟢 acquired 7.5K shares of BIOMARIN PHARMACEUTICAL INC (BMRN) Transaction Date: Jun 02, 2026 | Filing ID: 040911
Read more
2026-06-04 22:03
Director CLARK IAN T 🟢 acquired 7.5K shares of BIOMARIN PHARMACEUTICAL INC (BMRN) Transaction Date: Jun 02, 2026 | Filing ID: 040909
Read more
2026-06-04 22:02
Director Bodem Barbara W. 🟢 acquired 7.5K shares of BIOMARIN PHARMACEUTICAL INC (BMRN) Transaction Date: Jun 02, 2026 | Filing ID: 040907
Read more
2026-06-04 22:01
Director ANDERSON ELIZABETH M 🟢 acquired 7.5K shares of BIOMARIN PHARMACEUTICAL INC (BMRN) Transaction Date: Jun 02, 2026 | Filing ID: 040903
Read more
2026-06-04 20:24
(99% Neutral) BIOMARIN PHARMACEUTICAL INC (BMRN) Announces Executive Changes
Read more| Trial ID | Title | Phase | Indication | Status | Start Date | Completion Date | Source |
|---|---|---|---|---|---|---|---|
| NCT02963350 | A Multicenter, Multi-national Open-label Program to Provide BMN 190 to Patients… | — | CLN2 Disease | Approved_For_Marketing | — | — | ClinicalTrials.gov |
| NCT02636686 | Extension Study of Drisapersen in DMD Subjects | — | Duchenne Muscular Dystrophy | No_Longer_Available | — | — | ClinicalTrials.gov |
| NCT00484991 | Sapropterin Expanded Access Program | — | Phenylketonuria | Approved_For_Marketing | — | — | ClinicalTrials.gov |
| NCT01858103 | BMN 110 US Expanded Access Program | — | Mucopolysaccharidosis IVA | Approved_For_Marketing | — | — | ClinicalTrials.gov |
| NCT07477691 | Immune Modulation During Palynziq® Treatment in Adults (IMPALA) | Phase4 | Phenylketonuria | Recruiting | 2026-05-29 | 2027-09-30 | ClinicalTrials.gov |
| NCT07573631 | An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of … | Phase2 | Duchenne Muscular Dystrophy (DMD) | Enrolling_By_Invitation | 2026-04-20 | 2031-12-01 | ClinicalTrials.gov |
| NCT07441876 | Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Child… | Phase2 | Achondroplasia | Recruiting | 2026-04-20 | 2029-09-01 | ClinicalTrials.gov |
| NCT07126262 | A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 … | Phase2 | Hypochondroplasia | Recruiting | 2025-07-30 | 2028-06-30 | ClinicalTrials.gov |
| NCT07073014 | Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia | Phase3 | Hypochondroplasia | Enrolling_By_Invitation | 2025-06-20 | 2040-12-01 | ClinicalTrials.gov |
| NCT07473973 | ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP… | Phase3 | Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency | Recruiting | 2025-03-26 | 2028-11-07 | ClinicalTrials.gov |
| NCT06738017 | Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants | Phase1 | Alpha 1-Antitrypsin Deficiency | Active_Not_Recruiting | 2025-02-21 | 2026-08-19 | ClinicalTrials.gov |
| NCT06780332 | Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reacti… | Phase4 | Phenylketonuria | Active_Not_Recruiting | 2025-02-07 | 2026-08-01 | ClinicalTrials.gov |
| NCT06668805 | A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth D… | Phase2 | Noonan Syndrome | Recruiting | 2024-11-22 | 2041-09-01 | ClinicalTrials.gov |
| NCT06382155 | A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature | Phase2 | Idiopathic Short Stature | Recruiting | 2024-10-21 | 2036-12-01 | ClinicalTrials.gov |
| NCT06309979 | A Study to Assess Growth in Children With Idiopathic Short Stature | — | Idiopathic Short Stature | Recruiting | 2024-08-08 | 2040-12-31 | ClinicalTrials.gov |
| NCT06455059 | Interventional Study of Vosoritide for the Treatment of Children With Hypochond… | Phase3 | Hypochondroplasia | Active_Not_Recruiting | 2024-06-17 | 2026-08-01 | ClinicalTrials.gov |
| NCT06305234 | A Long Term, Post-marketing Study of Immune Response in Patients Receiving Paly… | — | Phenylketonuria (PKU) | Recruiting | 2024-01-20 | 2033-11-18 | ClinicalTrials.gov |
| NCT06280209 | A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pha… | Phase1 | Duchenne Muscular Dystrophy | Active_Not_Recruiting | 2024-01-03 | 2027-04-14 | ClinicalTrials.gov |
| NCT06224907 | Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With S… | Phase3 | Hemophilia A | Active_Not_Recruiting | 2023-12-25 | 2029-03-01 | ClinicalTrials.gov |
| NCT06212947 | A Multicenter Multinational Observational Study of Children With Hypochondropla… | — | Hypochondroplasia | Recruiting | 2023-11-27 | 2043-12-31 | ClinicalTrials.gov |
| NCT06138327 | A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And H… | Phase1 | Hyperoxaluria | Withdrawn | 2023-09-26 | 2024-03-25 | ClinicalTrials.gov |
| NCT05734196 | The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants W… | Phase1 | Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency | Recruiting | 2023-06-25 | 2027-11-11 | ClinicalTrials.gov |
| NCT05813314 | Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Pa… | Phase1 | Achondroplasia | Terminated | 2023-03-15 | 2023-06-14 | ClinicalTrials.gov |
| NCT05580692 | A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies … | Na | Hemophilia A | Terminated | 2023-03-14 | 2024-07-31 | ClinicalTrials.gov |
| NCT06168201 | VIrtual STudy in Achondroplasia for the US (VISTA) | — | Achondroplasia | Recruiting | 2023-02-21 | 2028-02-01 | ClinicalTrials.gov |
| NCT05768386 | A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 27… | — | Hemophilia A | Enrolling_By_Invitation | 2023-01-01 | 2040-01-01 | ClinicalTrials.gov |
| NCT05314751 | Learning to Live With Non-severe Haemophilia | — | Hemophilia | Completed | 2022-12-21 | 2023-09-29 | ClinicalTrials.gov |
| NCT05579548 | A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Ex… | — | Phenylketonuria, Maternal | Recruiting | 2022-11-22 | 2032-10-04 | ClinicalTrials.gov |
| NCT05270837 | Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12… | Phase3 | Phenylketonuria (PKU) | Active_Not_Recruiting | 2022-06-17 | 2027-10-01 | ClinicalTrials.gov |
| NCT05813678 | A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALa… | — | Phenylketonuria (PKU) | Recruiting | 2022-06-06 | 2033-11-01 | ClinicalTrials.gov |
| NCT05356377 | Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine… | — | Phenylketonurias | Completed | 2022-05-25 | 2025-07-01 | ClinicalTrials.gov |
| NCT05121376 | A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema | Phase1 | Hereditary Angioedema | Active_Not_Recruiting | 2022-02-15 | 2028-11-01 | ClinicalTrials.gov |
| NCT04800692 | The Effects of ATLAS Therapy on Nitric Oxide Bioavailability in Patients With I… | Phase1 | Claudication, Intermittent | Active_Not_Recruiting | 2021-06-15 | 2027-12-01 | ClinicalTrials.gov |
| NCT05368038 | ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program | — | Acid Sphingomyelinase Deficiency | Enrolling_By_Invitation | 2021-05-10 | 2029-08-31 | ClinicalTrials.gov |
| NCT04684940 | Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemop… | Phase1 | Hemophilia A With Inhibitor | Completed | 2020-12-10 | 2026-04-20 | ClinicalTrials.gov |
| NCT04323098 | Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With P… | Phase3 | Hemophilia A | Completed | 2020-12-08 | 2025-05-08 | ClinicalTrials.gov |
| NCT04554940 | A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achon… | Phase2 | Achondroplasia | Active_Not_Recruiting | 2020-10-10 | 2027-12-01 | ClinicalTrials.gov |
| NCT04480567 | AAV Gene Therapy Study for Subjects with PKU | Phase1 | Phenylketonuria (PKU) | Active_Not_Recruiting | 2020-09-24 | 2027-12-01 | ClinicalTrials.gov |
| NCT04560933 | A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-As… | — | Hemophilia A | Completed | 2020-08-25 | 2023-10-20 | ClinicalTrials.gov |
| NCT04476862 | Cerliponase Alfa Observational Study in the US | — | Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 | Active_Not_Recruiting | 2020-08-19 | 2030-08-31 | ClinicalTrials.gov |
| NCT04055051 | ATHN 11: Liver Transplantation Outcomes Study | — | Hemophilia A and B | Completed | 2020-06-18 | 2022-12-01 | ClinicalTrials.gov |
| NCT04227080 | BH4 Responsiveness in PAH Deficiency PKU Patients | Phase4 | Pku Phenylketonuria | Unknown | 2020-06-01 | 2022-06-01 | ClinicalTrials.gov |
| NCT04452513 | A Prospective Clinical Study of Phenylketonuria (PKU) | — | Phenylketonurias | Completed | 2019-10-11 | 2022-06-02 | ClinicalTrials.gov |
| NCT04404530 | Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU) | — | Phenylketonurias | Recruiting | 2019-10-08 | 2028-12-01 | ClinicalTrials.gov |
| NCT03989947 | An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With … | Phase2 | Achondroplasia | Active_Not_Recruiting | 2019-06-12 | 2038-05-01 | ClinicalTrials.gov |
| NCT03872531 | Lifetime Impact Study for Achondroplasia | — | Achondroplasia | Completed | 2019-03-31 | 2021-02-06 | ClinicalTrials.gov |
| NCT03792451 | Nutrition Status of Adults Treated With Pegvaliase | — | Phenylketonurias | Completed | 2019-01-17 | 2020-12-31 | ClinicalTrials.gov |
| NCT03820804 | Nutritional Status in Phenylketonuria | — | Phenylketonurias | Completed | 2019-01-07 | 2019-09-10 | ClinicalTrials.gov |
| NCT03862274 | Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease | — | Batten Disease | Enrolling_By_Invitation | 2018-12-01 | 2025-12-01 | ClinicalTrials.gov |
| NCT03694353 | Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Do… | Phase3 | Phenylketonuria (PKU) | Completed | 2018-09-13 | 2021-01-13 | ClinicalTrials.gov |
| NCT03583697 | A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and … | Phase2 | Achondroplasia | Completed | 2018-06-13 | 2022-01-26 | ClinicalTrials.gov |
| NCT03520712 | Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5 | Phase1 | Hemophilia A | Terminated | 2018-04-24 | 2024-08-07 | ClinicalTrials.gov |
| NCT03505125 | A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults | — | Phenylketonuria (PKU) | Completed | 2018-03-31 | 2019-07-31 | ClinicalTrials.gov |
| NCT03392974 | Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvo… | Phase3 | Hemophilia A | Completed | 2018-03-14 | 2023-06-05 | ClinicalTrials.gov |
| NCT03204370 | Natural History of Atypical Morquio A Disease | — | Mucopolysaccharidosis IV A | Completed | 2018-02-01 | 2025-09-01 | ClinicalTrials.gov |
| NCT02677870 | The Effectiveness of Kuvan in Amish PKU Patients | Phase4 | Phenylketonuria | Completed | 2018-01-22 | 2019-01-01 | ClinicalTrials.gov |
| NCT03017677 | A Cross-specialty Collaboration Platform for Mucopolysaccharidosis Confirmative… | — | Mucopolysaccharidoses | Unknown | 2018-01-01 | 2020-12-01 | ClinicalTrials.gov |
| NCT03370913 | Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvo… | Phase3 | Hemophilia A | Completed | 2017-12-19 | 2024-11-20 | ClinicalTrials.gov |
| NCT03449368 | Lifetime Impact of Achondroplasia Study in Europe-LIAISE | — | Achondroplasia | Completed | 2017-12-17 | 2020-04-29 | ClinicalTrials.gov |
| NCT03424018 | An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children W… | Phase3 | Achondroplasia | Active_Not_Recruiting | 2017-12-12 | 2031-06-01 | ClinicalTrials.gov |
| NCT03864029 | Retrospective Observational Safety Effectiveness With Kuvan in hpA | — | Tetrahydrobiopterin Deficiency | Completed | 2017-10-10 | 2018-07-25 | ClinicalTrials.gov |
| NCT03150069 | Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT C… | — | Morquio Disease | Completed | 2017-05-10 | 2018-08-17 | ClinicalTrials.gov |
| NCT03197766 | A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondr… | Phase3 | Achondroplasia | Completed | 2016-12-12 | 2019-10-30 | ClinicalTrials.gov |
| NCT02153255 | Dynamic Gait Analysis in Children With Mucopolysaccharidosis Type IVa | — | Mucopolysaccharidosis IV | Withdrawn | 2016-07-01 | 2016-07-01 | ClinicalTrials.gov |
| NCT02958202 | Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD) | Phase2 | Duchenne Muscular Dystrophy | Terminated | 2016-04-01 | 2016-09-01 | ClinicalTrials.gov |
| NCT02597881 | Achondroplasia Natural History Multicenter Clinical Study | — | Achondroplasia | Recruiting | 2016-04-01 | 2030-12-31 | ClinicalTrials.gov |
| NCT02724228 | A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Ch… | Phase2 | Achondroplasia | Active_Not_Recruiting | 2016-01-26 | 2028-02-01 | ClinicalTrials.gov |
| NCT02156674 | Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome | Na | Maroteaux-Lamy Syndrome | Terminated | 2016-01-26 | 2019-12-01 | ClinicalTrials.gov |
| NCT02678689 | A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 1… | Phase2 | Jansky-Bielschowsky Disease | Completed | 2016-01-22 | 2022-04-20 | ClinicalTrials.gov |
| NCT02537561 | Talazoparib in Combination With Gemcitabine and Cisplatin in Patients With Adva… | Phase1 | Solid Tumors | Withdrawn | 2015-12-01 | 2018-12-01 | ClinicalTrials.gov |
| NCT02583152 | New Imaging Technology to Assess Effect of Enzyme Replacment Therapy on Eye Dis… | — | Mucopolysaccharidoses | Unknown | 2015-11-01 | 2020-11-01 | ClinicalTrials.gov |
| NCT04368624 | PKU Skin Stripping | — | Phenylketonurias | Completed | 2015-10-12 | 2021-04-01 | ClinicalTrials.gov |
| NCT02576795 | Gene Therapy Study in Severe Haemophilia A Patients (270-201) | Phase1 | Severe Haemophilia A | Completed | 2015-09-28 | 2024-02-14 | ClinicalTrials.gov |
| NCT02468570 | A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Pa… | — | Phenylketonuria | Completed | 2015-08-05 | 2017-02-01 | ClinicalTrials.gov |
| NCT02401347 | Phase II Trial of Talazoparib in BRCA1/2 Wild-type HER2-negative Breast Cancer … | Phase2 | Advanced Breast Cancer | Completed | 2015-08-01 | 2022-12-01 | ClinicalTrials.gov |
| NCT02354664 | Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease | — | Pompe Disease | Completed | 2015-04-01 | 2016-10-19 | ClinicalTrials.gov |
| NCT02392793 | Talazoparib Plus Irinotecan With or Without Temozolomide in Children With Refra… | Phase1 | Childhood Solid Tumors | Completed | 2015-03-25 | 2019-08-30 | ClinicalTrials.gov |
| NCT02353312 | Rhode Island Diastolic Dysfunction - Heart Failure | Na | Heart Failure | Completed | 2015-03-01 | 2018-12-01 | ClinicalTrials.gov |
| NCT02485899 | An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in … | Phase1 | Jansky-Bielschowsky Disease | Completed | 2015-02-01 | 2020-12-10 | ClinicalTrials.gov |
| NCT02329769 | Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD) | Phase2 | Duchenne Muscular Dystrophy | Terminated | 2014-12-01 | 2016-08-31 | ClinicalTrials.gov |
| NCT02221362 | A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disea… | — | Late-onset Pompe Patients Untreated or Treated With rhGAA | Terminated | 2014-12-01 | 2016-09-01 | ClinicalTrials.gov |
| NCT02191917 | A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Diseas… | — | Late-onset Pompe Disease | Terminated | 2014-10-21 | 2016-06-22 | ClinicalTrials.gov |
| NCT02294877 | A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) | — | Mucopolysaccharidosis IV Type A | Completed | 2014-09-01 | 2024-02-21 | ClinicalTrials.gov |
| NCT02049593 | PARP Inhibitor BMN-673 and Temozolomide or Irinotecan Hydrochloride in Treating… | Phase1 | Metastatic Cancer | Completed | 2014-06-12 | 2019-10-07 | ClinicalTrials.gov |
| NCT01924845 | BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIR… | Phase3 | Late-onset Pompe Disease | Terminated | 2014-04-01 | 2016-09-12 | ClinicalTrials.gov |
| NCT02208661 | Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzy… | — | Morquio A Syndrome | Completed | 2014-03-01 | 2018-03-01 | ClinicalTrials.gov |
| NCT01977820 | Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria | Phase2 | Phenylketonuria | Terminated | 2014-02-01 | 2014-11-01 | ClinicalTrials.gov |
| NCT02055157 | A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Ch… | Phase2 | Achondroplasia | Completed | 2014-01-13 | 2017-10-02 | ClinicalTrials.gov |
| NCT01965912 | Kuvan®'s Effect on the Cognition of Children With Phenylketonuria | Phase4 | Phenylketonuria | Completed | 2013-10-01 | 2023-01-04 | ClinicalTrials.gov |
| NCT01961518 | Screening an Orthopedic Population for Mildly-affected Individuals With Morquio… | — | Morquio Syndrome A | Completed | 2013-10-01 | 2015-05-01 | ClinicalTrials.gov |
| NCT01907087 | A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, … | Phase1 | Jansky-Bielschowsky Disease | Completed | 2013-09-01 | 2015-11-01 | ClinicalTrials.gov |
| NCT01924026 | Neurocognitive Outcomes in Mild Hyperphenylalaninemia (MHP)MHP Study | — | Phenylketonuria | Completed | 2013-09-01 | 2016-02-01 | ClinicalTrials.gov |
| NCT01920828 | Gait Analysis in MPS IVA | — | MPS IVA | Completed | 2013-08-01 | 2017-06-01 | ClinicalTrials.gov |
| NCT01889862 | Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections… | Phase3 | Phenylketonuria (PKU) | Completed | 2013-07-29 | 2019-02-05 | ClinicalTrials.gov |
| NCT01966029 | BMN 110 Phase 3B in Australian Patients | Phase3 | Mucopolysaccharidosis IVA (Morquio A Syndrome) | Completed | 2013-07-01 | 2016-12-01 | ClinicalTrials.gov |
| NCT02365376 | Collection and Storage of Human Biospecimens for Research Into Rare Diseases an… | — | Rare Genetic Disorders | Unknown | 2013-06-01 | — | ClinicalTrials.gov |
| NCT01957059 | A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD) | Phase1 | Duchenne Muscular Dystrophy | Terminated | 2013-06-01 | 2016-08-03 | ClinicalTrials.gov |
| NCT01803412 | A Study of the Safety, Tolerability & Efficacy of Long-term Administration of D… | Phase3 | Muscular Dystrophies | Terminated | 2013-05-01 | 2016-10-01 | ClinicalTrials.gov |
| NCT01869972 | Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia | — | Phenylketonuria | Completed | 2013-05-01 | 2015-12-01 | ClinicalTrials.gov |
| NCT01819727 | An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treat… | Phase3 | Phenylketonuria | Completed | 2013-05-01 | 2015-11-25 | ClinicalTrials.gov |
| NCT01806051 | A Pilot Study on Diurnal Variation | Na | Phenylketonuria (PKU) | Terminated | 2013-03-01 | 2014-05-01 | ClinicalTrials.gov |
| NCT01826474 | Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy | Phase1 | Duchenne Muscular Dystrophy | Terminated | 2013-01-01 | 2016-08-31 | ClinicalTrials.gov |
| NCT01707433 | Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilate… | — | Mucopolysaccharidosis IV A | Completed | 2012-10-01 | 2015-06-01 | ClinicalTrials.gov |
| NCT01753804 | A Prospective Natural History Study of Progression of Subjects With Duchenne Mu… | — | Duchenne Muscular Dystrophy | Terminated | 2012-09-01 | 2016-10-01 | ClinicalTrials.gov |
| NCT01697319 | Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have L… | Phase2 | Mucopolysaccharidosis IVA | Terminated | 2012-08-01 | 2014-10-01 | ClinicalTrials.gov |
| NCT01650909 | The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Sta… | — | Classical Phenylketonuria(PKU) | Withdrawn | 2012-07-01 | — | ClinicalTrials.gov |
| NCT01752296 | Psychological Concomitants of Morquio Syndrome (The MAP Study) | — | Morquio Disease | Completed | 2012-07-01 | 2013-10-01 | ClinicalTrials.gov |
| NCT01733615 | Discovering New Biomarkers For Monitoring Disease Progression in Patients With … | — | Mucopolysaccharidosis Type IVA | Terminated | 2012-06-01 | 2013-02-01 | ClinicalTrials.gov |
| NCT01560286 | A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phe… | Phase2 | Phenylketonuria | Completed | 2012-05-01 | 2015-07-01 | ClinicalTrials.gov |
| NCT01617070 | Effects of Kuvan on Melatonin Secretion | Phase4 | Phenylketonuria (PKU) | Completed | 2012-05-01 | 2014-12-01 | ClinicalTrials.gov |
| NCT01603095 | A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients W… | — | Achondroplasia | Completed | 2012-04-01 | 2021-02-01 | ClinicalTrials.gov |
| NCT01609062 | Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome | Phase2 | Mucopolysaccharidosis IVA | Terminated | 2012-04-01 | 2014-11-01 | ClinicalTrials.gov |
| NCT01590446 | A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy … | Phase1 | Achondroplasia | Completed | 2012-02-01 | 2012-06-01 | ClinicalTrials.gov |
| NCT01515956 | Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharido… | Phase2 | Mucopolysaccharidosis IVA | Completed | 2011-10-01 | 2016-02-01 | ClinicalTrials.gov |
| NCT01435772 | Extension Study for Patients Who Have Participated in a BMN 701 Study | Phase2 | Pompe Disease | Terminated | 2011-08-15 | 2016-09-09 | ClinicalTrials.gov |
| NCT01425528 | Study of Kuvan Treatment in Adults With GTPCH Deficiency | Phase1 | GTP Cyclohydrolase Deficiency | Completed | 2011-08-01 | 2013-08-01 | ClinicalTrials.gov |
| NCT01415427 | Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucop… | Phase3 | Mucopolysaccharidosis IV A | Completed | 2011-07-01 | 2016-06-16 | ClinicalTrials.gov |
| NCT01541397 | Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy | Na | Hyperphenylalaninemia | Terminated | 2011-06-01 | 2012-08-01 | ClinicalTrials.gov |
| NCT01376908 | Kuvan® in Phenylketonuria Patients Less Than 4 Years Old | Phase3 | Phenylketonuria | Completed | 2011-06-01 | 2017-02-17 | ClinicalTrials.gov |
| NCT01387854 | Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previou… | — | MPS VI | Completed | 2011-06-01 | 2013-02-01 | ClinicalTrials.gov |
| NCT01212744 | Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Su… | Phase2 | Phenylketonuria | Completed | 2011-03-01 | 2015-04-01 | ClinicalTrials.gov |
| NCT01275066 | A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients… | Phase3 | MPS IV A | Completed | 2011-02-01 | 2012-08-01 | ClinicalTrials.gov |
| NCT01230801 | Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 … | Phase1 | Pompe Disease | Completed | 2011-01-17 | 2013-03-06 | ClinicalTrials.gov |
| NCT01274026 | Evaluation of Behavior, Executive Function, Neurotransmitter Function and Genom… | — | Phenylketonuria | Completed | 2011-01-01 | 2013-12-01 | ClinicalTrials.gov |
| NCT01242111 | A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients Wi… | Phase1 | MPS IV A | Terminated | 2010-11-01 | 2014-07-01 | ClinicalTrials.gov |
| NCT01209819 | Bone Mineral Density in Adults With Hyperphenylalaninemia | — | Phenylketonurias | Completed | 2010-09-01 | 2012-10-01 | ClinicalTrials.gov |
| NCT01114737 | Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatr… | Phase3 | Phenylketonuria | Completed | 2010-08-01 | 2013-03-01 | ClinicalTrials.gov |
| NCT01141595 | Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Development… | Na | Autism Spectrum Disorder | Completed | 2010-07-01 | 2011-09-01 | ClinicalTrials.gov |
| NCT00924703 | Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-… | Phase2 | Phenylketonuria | Completed | 2010-01-13 | 2019-01-31 | ClinicalTrials.gov |
| NCT01037309 | Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD) | Phase1 | Duchenne Muscular Dystrophy | Completed | 2009-12-01 | 2013-10-01 | ClinicalTrials.gov |
| NCT01016392 | Observational Study on the Long Term Safety of Kuvan® Treatment in Patients Wit… | — | Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency | Completed | 2009-12-01 | 2021-05-31 | ClinicalTrials.gov |
| NCT00925054 | Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple… | Phase2 | Phenylketonuria | Completed | 2009-09-01 | 2015-06-01 | ClinicalTrials.gov |
| NCT00964236 | The Effects of Kuvan on Functional Brain Connectivity in Individuals With Pheny… | — | Phenylketonuria | Completed | 2009-08-01 | 2011-08-01 | ClinicalTrials.gov |
| NCT00943579 | Open-Label Extension Study of Kuvan for Autism | Phase2 | Autistic Disorder | Completed | 2009-08-01 | 2012-03-01 | ClinicalTrials.gov |
| NCT00852358 | A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I | Na | Cognitive Decline | Completed | 2009-06-01 | 2015-04-01 | ClinicalTrials.gov |
| NCT00935753 | Trial of Kuvan in Lesch-Nyhan Disease | Phase2 | Behavioral Manifestations of Lesch-Nyhan Disease | Withdrawn | 2009-04-01 | 2010-02-01 | ClinicalTrials.gov |
| NCT00884949 | A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subject… | Phase1 | MPS IV A | Completed | 2009-04-01 | 2011-03-01 | ClinicalTrials.gov |
| NCT00850070 | Sapropterin as a Treatment for Autistic Disorder | Phase2 | Autistic Disorder | Completed | 2009-03-01 | 2011-10-01 | ClinicalTrials.gov |
| NCT00838435 | Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, … | Phase3 | Phenylketonuria | Completed | 2009-02-01 | 2018-09-14 | ClinicalTrials.gov |
| NCT00827762 | Behavioral Effects of Kuvan in Children With Mild Phenylketonuria | — | Phenylketonuria | Terminated | 2009-01-01 | 2010-01-01 | ClinicalTrials.gov |
| NCT00802893 | Oral 6R-BH4 for the Treatment of Isolated Systolic Hypertension and Endothelial… | Phase2 | Isolated Systolic Hypertension | Terminated | 2008-12-01 | 2009-05-01 | ClinicalTrials.gov |
| NCT00688844 | Nutritional and Neurotransmitter Changes in PKU Subjects on BH4 | — | Phenylketonuria | Completed | 2008-10-01 | 2010-10-01 | ClinicalTrials.gov |
| NCT00789568 | A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Inter… | Phase1 | Phenylketonuria | Completed | 2008-10-01 | 2009-10-01 | ClinicalTrials.gov |
| NCT00787995 | A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio… | — | MPS IV A | Terminated | 2008-10-01 | 2014-07-01 | ClinicalTrials.gov |
| NCT00778206 | PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry | — | Phenylketonuria | Completed | 2008-09-01 | 2021-01-29 | ClinicalTrials.gov |
| NCT00728676 | Study to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU… | — | Phenylketonuria | Completed | 2008-08-01 | 2010-05-01 | ClinicalTrials.gov |
| NCT00730080 | Sapropterin in Individuals With Phenylketonuria | — | Phenylketonuria | Completed | 2008-07-01 | 2018-05-01 | ClinicalTrials.gov |
| NCT00634660 | Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria | Phase1 | Phenylketonuria | Completed | 2008-05-01 | 2009-10-01 | ClinicalTrials.gov |
| NCT00625820 | Tetrahydrobiopterin in Patients With Chronic Kidney Disease (CKD) and Albuminur… | Phase2 | Kidney Disease | Completed | 2008-05-01 | 2008-09-01 | ClinicalTrials.gov |
| NCT00435331 | 6R-BH4 Pulmonary Arterial Hypertension Study | Phase1 | Pulmonary Arterial Hypertension | Completed | 2008-03-01 | 2008-10-01 | ClinicalTrials.gov |
| NCT01910649 | A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety… | Phase2 | Muscular Dystrophies | Terminated | 2008-03-01 | 2016-09-01 | ClinicalTrials.gov |
| NCT00532844 | A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C … | Phase2 | Endothelial Dysfunction | Completed | 2007-09-01 | 2009-03-01 | ClinicalTrials.gov |
| NCT00445978 | A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease | Phase2 | Sickle Cell Disease | Completed | 2007-05-01 | 2009-06-01 | ClinicalTrials.gov |
| NCT00403494 | A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Pe… | Phase2 | Intermittent Claudication | Completed | 2006-12-01 | 2009-01-01 | ClinicalTrials.gov |
| NCT00423280 | Effect of 6R-BH4 Treatment in Coronary Artery Disease (OXBIO Study) | Phase2 | Coronary Artery Disease | Unknown | 2006-11-01 | 2009-02-01 | ClinicalTrials.gov |
| NCT00355264 | Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia D… | Phase2 | Tetrahydrobiopterin Deficiencies | Completed | 2006-08-01 | 2009-06-01 | ClinicalTrials.gov |
| NCT00332189 | Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protoco… | Phase3 | Phenylketonuria | Completed | 2006-07-01 | 2009-08-01 | ClinicalTrials.gov |
| NCT00299000 | A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MP… | Phase4 | Mucopolysaccharidosis VI | Completed | 2006-05-01 | 2009-04-01 | ClinicalTrials.gov |
| NCT00325962 | A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Cont… | Phase2 | Hypertension | Completed | 2006-05-01 | 2008-12-01 | ClinicalTrials.gov |
| NCT00272792 | Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Child… | Phase3 | Phenylketonurias | Completed | 2006-02-01 | 2006-11-01 | ClinicalTrials.gov |
| NCT00225615 | A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects Wit… | Phase3 | Phenylketonurias | Completed | 2005-11-01 | 2006-06-01 | ClinicalTrials.gov |
| NCT00214773 | Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP) | — | Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome) | Completed | 2005-07-01 | 2020-05-01 | ClinicalTrials.gov |
| NCT00104247 | Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenyl… | Phase3 | Phenylketonurias | Completed | 2005-03-01 | 2006-02-01 | ClinicalTrials.gov |
| NCT00104260 | Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ T… | Phase2 | Phenylketonurias | Completed | 2004-12-01 | 2005-11-01 | ClinicalTrials.gov |
| NCT00104234 | Study of rhASB in Patients With Mucopolysaccharidosis VI | Phase3 | Mucopolysaccharidosis VI | Completed | 2004-02-01 | 2006-10-01 | ClinicalTrials.gov |
| NCT00067470 | Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patient… | Phase3 | Mucopolysaccharidosis VI | Completed | 2003-09-01 | 2004-03-01 | ClinicalTrials.gov |
| NCT00048711 | Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosam… | Phase2 | Mucopolysaccharidosis VI | Completed | 2002-03-01 | 2006-06-01 | ClinicalTrials.gov |
| NCT00048620 | Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With M… | Phase1 | Mucopolysaccharidosis VI | Completed | 2000-09-01 | 2005-11-01 | ClinicalTrials.gov |
Total clinical trials: 168
| Product Name | Type | Development Stage | Therapeutic Area | Study Status | Trial ID |
|---|---|---|---|---|---|
| Confirmatory Testing | Other | Preclinical | Acid Sphingomyelinase Deficiency | ENROLLING_BY_INVITATION | NCT05368038 |
| Vosoritide Injection [Voxzogo] | Other | Phase PHASE2 | Achondroplasia | RECRUITING | NCT07441876 |
| BMN 333 | Other | Phase PHASE2 | Achondroplasia | RECRUITING | NCT07441876 |
| BMN 351 | Other | Phase PHASE2 | Duchenne Muscular Dystrophy (DMD) | ENROLLING_BY_INVITATION | NCT07573631 |
| Diet Only | Other | Phase PHASE3 | Phenylketonuria (PKU) | ACTIVE_NOT_RECRUITING | NCT05270837 |
| Pegvaliase | Other | Phase PHASE3 | Phenylketonuria (PKU) | ACTIVE_NOT_RECRUITING | NCT05270837 |
| Valoctocogene roxaparvovec | Other | Phase PHASE1 | Hemophilia A With Inhibitor | COMPLETED | NCT04684940 |
| Vosoritide Injection [Voxzogo] | Other | Phase PHASE2 | Achondroplasia | RECRUITING | NCT07441876 |
| BMN 333 | Other | Phase PHASE2 | Achondroplasia | RECRUITING | NCT07441876 |
| Administration Kit | Other | Preclinical | Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 | ACTIVE_NOT_RECRUITING | NCT04476862 |
| Cerliponase Alfa | Other | Preclinical | Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 | ACTIVE_NOT_RECRUITING | NCT04476862 |
| BMN 351 | Other | Phase PHASE1 | Duchenne Muscular Dystrophy | ACTIVE_NOT_RECRUITING | NCT06280209 |
| Methotrexate | Other | Phase PHASE4 | Phenylketonuria | RECRUITING | NCT07477691 |
| Pegvaliase | Other | Phase PHASE4 | Phenylketonuria | RECRUITING | NCT07477691 |
| Vosoritide Injection [Voxzogo] | Other | Phase PHASE2 | Achondroplasia | RECRUITING | NCT07441876 |
| BMN 333 | Other | Phase PHASE2 | Achondroplasia | RECRUITING | NCT07441876 |
| Placebo | Other | Phase PHASE2 | Idiopathic Short Stature | RECRUITING | NCT06382155 |
| Human Growth Hormone | Other | Phase PHASE2 | Idiopathic Short Stature | RECRUITING | NCT06382155 |
| Vosoritide Injection | Other | Phase PHASE2 | Idiopathic Short Stature | RECRUITING | NCT06382155 |
| Placebo | Other | Phase PHASE2 | Idiopathic Short Stature | RECRUITING | NCT06382155 |
| Human Growth Hormone | Other | Phase PHASE2 | Idiopathic Short Stature | RECRUITING | NCT06382155 |
| Vosoritide Injection | Other | Phase PHASE2 | Idiopathic Short Stature | RECRUITING | NCT06382155 |
| Placebo | Other | Phase PHASE2 | Idiopathic Short Stature | RECRUITING | NCT06382155 |
| Human Growth Hormone | Other | Phase PHASE2 | Idiopathic Short Stature | RECRUITING | NCT06382155 |
| Vosoritide Injection | Other | Phase PHASE2 | Idiopathic Short Stature | RECRUITING | NCT06382155 |
| Placebo | Other | Phase PHASE2 | Idiopathic Short Stature | RECRUITING | NCT06382155 |
| Human Growth Hormone | Other | Phase PHASE2 | Idiopathic Short Stature | RECRUITING | NCT06382155 |
| Vosoritide Injection | Other | Phase PHASE2 | Idiopathic Short Stature | RECRUITING | NCT06382155 |
| Placebo | Other | Phase PHASE2 | Idiopathic Short Stature | RECRUITING | NCT06382155 |
| Human Growth Hormone | Other | Phase PHASE2 | Idiopathic Short Stature | RECRUITING | NCT06382155 |
| Vosoritide Injection | Other | Phase PHASE2 | Idiopathic Short Stature | RECRUITING | NCT06382155 |
| BMN 351 | Other | Phase PHASE2 | Duchenne Muscular Dystrophy (DMD) | ENROLLING_BY_INVITATION | NCT07573631 |
| BMN 351 | Other | Phase PHASE2 | Duchenne Muscular Dystrophy (DMD) | ENROLLING_BY_INVITATION | NCT07573631 |
| BMN 351 | Other | Phase PHASE2 | Duchenne Muscular Dystrophy (DMD) | ENROLLING_BY_INVITATION | NCT07573631 |
| BMN 351 | Other | Phase PHASE2 | Duchenne Muscular Dystrophy (DMD) | ENROLLING_BY_INVITATION | NCT07573631 |
| BMN 351 | Other | Phase PHASE2 | Duchenne Muscular Dystrophy (DMD) | ENROLLING_BY_INVITATION | NCT07573631 |
| BMN 351 | Other | Phase PHASE2 | Duchenne Muscular Dystrophy (DMD) | ENROLLING_BY_INVITATION | NCT07573631 |
| BMN 351 | Other | Phase PHASE2 | Duchenne Muscular Dystrophy (DMD) | ENROLLING_BY_INVITATION | NCT07573631 |
| BMN 351 | Other | Phase PHASE2 | Duchenne Muscular Dystrophy (DMD) | ENROLLING_BY_INVITATION | NCT07573631 |
Total products: 39