Pipeline — registered trials by phase
A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
— · Achondroplasia · Completed · est. 2021-02
Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency
— · Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency · Completed · est. 2021-05-31
Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02
— · MPS VI · Completed · est. 2012-10
VIrtual STudy in Achondroplasia for the US (VISTA)
— · Achondroplasia · Recruiting · est. 2028-02
A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
— · Phenylketonuria (PKU) · Recruiting · est. 2033-11-18
PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
— · Phenylketonuria, Hyperphenylalaninaemia · Completed · est. 2021-01-29
Kuvan®'s Effect on the Cognition of Children With Phenylketonuria
— · Phenylketonuria · Completed · est. 2023-01-04
Lifetime Impact of Achondroplasia Study in Europe-LIAISE
— · Achondroplasia · Completed · est. 2020-04-29
A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
— · Phenylketonuria, Maternal · Recruiting · est. 2032-10-04
A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302
— · Phenylketonuria · Completed · est. 2017-02-01
Immune Modulation During Palynziq® Treatment in Adults (IMPALA)
— · Phenylketonuria · Recruiting · est. 2027-09-30
Lifetime Impact Study for Achondroplasia
— · Achondroplasia · Completed · est. 2021-02-06
Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq
— · Phenylketonuria, PKU · Completed · est. 2026-08-17
A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)
— · Hemophilia A · Enrolling By Invitation · est. 2040-01
Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
— · Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome) · Completed · est. 2020-05-01
A Prospective Clinical Study of Phenylketonuria (PKU)
— · Phenylketonurias · Completed · est. 2022-06-02
Retrospective Observational Safety Effectiveness With Kuvan in hpA
— · Tetrahydrobiopterin Deficiency · Completed · est. 2018-03-09
Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI
— · Mucopolysaccharidosis VI · Completed
A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers
— · Achondroplasia · Completed · est. 2012-06
A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects
— · Phenylketonuria · Completed · est. 2009-01
Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants
— · Alpha 1-Antitrypsin Deficiency · Active Not Recruiting · est. 2026-08-19
Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria
— · Phenylketonuria · Completed · est. 2009-04
Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria
— · Phenylketonurias · Completed
Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)
— · Phenylketonuria · Completed · est. 2015-04
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
— · Hypochondroplasia · Recruiting · est. 2028-06-30
A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
— · Achondroplasia · Active Not Recruiting · est. 2027-12
An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
— · Achondroplasia · Active Not Recruiting · est. 2038-05
Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
— · Duchenne Muscular Dystrophy · Completed · est. 2013-05
Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI
— · Mucopolysaccharidosis VI · Completed
A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease
— · Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 · Completed · est. 2015-11
Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU
— · Phenylketonuria · Completed · est. 2015-06
A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia
— · Achondroplasia · Completed · est. 2017-10-02
A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease
— · Sickle Cell Disease · Completed · est. 2008-08
Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease
— · Pompe Disease · Completed · est. 2013-03-06
Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
— · Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA · Completed · est. 2016-02
Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
— · Hemophilia A With Inhibitor, Hemophilia A With Anti Factor VIII · Completed · est. 2026-04-20
A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
— · Noonan Syndrome · Active Not Recruiting · est. 2027-03
A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction
— · Endothelial Dysfunction · Completed · est. 2008-11
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
— · Duchenne Muscular Dystrophy · Active Not Recruiting · est. 2027-04-14
A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
— · Idiopathic Short Stature · Recruiting · est. 2026-06
A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA
— · MPS IV A · Completed · est. 2011-02
A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia
— · Achondroplasia · Completed · est. 2022-01-26
A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension
— · Hypertension · Completed · est. 2006-12
A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
— · Achondroplasia · Active Not Recruiting · est. 2027-12
Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A
— · Hemophilia A · Completed · est. 2023-01-27
Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels
— · Phenylketonurias · Completed
A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
— · MPS IV A · Completed · est. 2012-08
Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU
— · Phenylketonuria (PKU) · Completed · est. 2016-01-13
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
— · Achondroplasia · Recruiting · est. 2029-06
A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
— · Achondroplasia · Completed · est. 2019-10-30
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
— · Hypochondroplasia · Active Not Recruiting · est. 2026-08-01
Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU
— · Phenylketonuria · Completed · est. 2018-09-14
BMN 110 Phase 3B in Australian Patients
— · Mucopolysaccharidosis IVA (Morquio A Syndrome) · Completed · est. 2016-07
Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients
— · Phenylketonuria · Completed · est. 2013-03
Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI
— · Mucopolysaccharidosis VI · Completed
Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU
— · Phenylketonuria (PKU) · Completed · est. 2021-01-13
Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet
— · Phenylketonurias · Completed
Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
— · Phenylketonuria (PKU) · Active Not Recruiting · est. 2025-01-14
Kuvan® in Phenylketonuria Patients Less Than 4 Years Old
— · Phenylketonuria · Completed · est. 2014-07
Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A
— · Hemophilia A · Active Not Recruiting · est. 2025-04-16
Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006
— · Phenylketonuria · Completed · est. 2009-08
Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia
— · Hypochondroplasia · Enrolling By Invitation · est. 2044-10
Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
— · Mucopolysaccharidosis IV A, Morquio A Syndrome, MPS IVA · Completed · est. 2016-06-16
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)
— · Hemophilia A · Completed · est. 2020-11-16
AldurazymeLaronidase
NaglazymeGalsulfase
Source: ClinicalTrials.gov (trials where BioMarin is lead sponsor; 64 active or completed) and Drugs@FDA (2 approved products). Registry data can include older or partnered programs; the company's own pipeline page is the official list.
SEC filings — insider trading forms excluded
- 8-K2026-10-06Current report (material event)
- 8-K2026-09-30Current report (material event)
- 8-K2026-09-15Current report (material event)
- 8-K2026-09-14Current report (material event)
- 8-K2026-09-01Current report (material event)
- 8-K2026-08-31Current report (material event)
- 8-K2026-08-18Current report (material event)
- S-82026-08-06Employee stock plan
- 10-Q2026-08-06Quarterly report
- 8-K2026-08-06Current report (material event)
- 8-K/A2026-07-13Current report (amended)
- 8-K2026-06-04Current report (material event)
- 10-Q2026-05-05Quarterly report
- 8-K2026-05-04Current report (material event)
- SCHEDULE 13G2026-04-29SCHEDULE 13G
- 8-K2026-04-27Current report (material event)
- 8-K2026-04-23Current report (material event)
- ARS2026-04-21Annual report to holders
- DEFA14A2026-04-21DEFA14A
- DEF 14A2026-04-21Proxy statement
- SCHEDULE 13G/A2026-03-26SCHEDULE 13G/A
- 8-K2026-03-16Current report (material event)
- 10-K2026-02-26Annual report
- 8-K2026-02-23Current report (material event)
- 8-K2026-02-12Current report (material event)
- 8-K2026-01-29Current report (material event)
- 8-K2026-01-26Current report (material event)
- SCHEDULE 13G/A2026-01-21SCHEDULE 13G/A
- 8-K2026-01-12Current report (material event)
- 8-K2025-12-22Current report (material event)
- 8-K2025-12-19Current report (material event)
- 8-K2025-12-17Current report (material event)
- SCHEDULE 13G/A2025-11-14SCHEDULE 13G/A
- 10-Q2025-10-28Quarterly report
- 8-K2025-10-27Current report (material event)
- 8-K2025-10-07Current report (material event)
- SCHEDULE 13G/A2025-08-14SCHEDULE 13G/A
- SCHEDULE 13G/A2025-08-14SCHEDULE 13G/A
- 8-K2025-08-11Current report (material event)
- S-82025-08-05Employee stock plan
Source: SEC EDGAR. Forms 3/4/5 and 144 (insider transactions) are intentionally left out.