BioMarin Pharmaceutical ( BMRN )

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$20M Part D

Peak

Flagship revenue driver at 35% of company revenue; only pegvaliase PAL enzyme approved for PKU, representing novel mechanism competing with traditional cofactor therapy.

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Data Sources

Drug label: DailyMed / FDA
Clinical trials: ClinicalTrials.gov
Patent data: FDA Orange Book
Spending data: CMS Medicare

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.

Pipeline & Clinical Trials

ATHN 11: Liver Transplantation Outcomes Study

Hemophilia A and B

N/A

Clinical Trials (1)

NCT04055051ATHN 11: Liver Transplantation Outcomes Study

N/A

A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin

Hemophilia A

N/A

Clinical Trials (1)

NCT05768386A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)

N/A

Observational study

Duchenne Muscular Dystrophy

N/A

Clinical Trials (1)

NCT01753804A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.

N/A

Blood sample collection

Hemophilia A

N/A

Clinical Trials (1)

NCT04560933A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A

N/A

Pegvaliase

N/A

Clinical Trials (1)

NCT06305234A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)

N/A

A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia

Duchenne Muscular Dystrophy

N/A

Clinical Trials (1)

NCT01603095A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia

N/A

Drisapersen

N/A

Clinical Trials (1)

NCT02636686Extension Study of Drisapersen in DMD Subjects

N/A

Biospecimen Collection

Hemophilia A

N/A

Clinical Trials (1)

NCT05580692A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV)

N/A

Sapropterin

N/A

Clinical Trials (1)

NCT00730080Sapropterin in Individuals With Phenylketonuria

N/A

Neurocognitive Outcomes in Mild Hyperphenylalaninemia (MHP)MHP Study

N/A

Clinical Trials (1)

NCT01924026Neurocognitive Outcomes in Mild Hyperphenylalaninemia (MHP)MHP Study

N/A

Sapropterin

Mucopolysaccharidosis Type IVA

N/A

Clinical Trials (1)

NCT00964236The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)

N/A

Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis

N/A

Clinical Trials (1)

NCT01733615Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA

N/A

Skin Tape

N/A

Clinical Trials (1)

NCT04368624PKU Skin Stripping

N/A

Gait Analysis in MPS IVA

MPS IVA

N/A

Clinical Trials (1)

NCT01920828Gait Analysis in MPS IVA

N/A

Lifetime Impact Study for Achondroplasia

Classical Phenylketonuria(PKU)

N/A

Clinical Trials (1)

NCT03872531Lifetime Impact Study for Achondroplasia

N/A

The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With

N/A

Clinical Trials (1)

NCT01650909The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With Classical PKU

N/A

Administration of CANTAB and Subject Global Assessment

N/A

Clinical Trials (1)

NCT02468570A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302

N/A

A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults

N/A

Clinical Trials (1)

NCT03505125A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults

N/A

CLN2 Treatment

Batten Disease

N/A

Clinical Trials (1)

NCT03862274Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease

N/A

Pegvaliase

N/A

Clinical Trials (1)

NCT05813678A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)

N/A

VIrtual STudy in Achondroplasia for the US (VISTA)

Phenylketonuria, Maternal

N/A

Clinical Trials (1)

NCT06168201VIrtual STudy in Achondroplasia for the US (VISTA)

N/A

Pegvaliase

N/A

Clinical Trials (1)

NCT05579548A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding

N/A

Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therap

Morquio A Syndrome

N/A

Clinical Trials (1)

NCT02208661Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study)

N/A

Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia

N/A

Clinical Trials (1)

NCT01869972Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia

N/A

Bone Mineral Density in Adults With Hyperphenylalaninemia

Mucopolysaccharidosis IVA

N/A

Clinical Trials (1)

NCT01209819Bone Mineral Density in Adults With Hyperphenylalaninemia

N/A

Psychological Concomitants of Morquio Syndrome (The MAP Study)

Morquio Disease

N/A

Clinical Trials (1)

NCT01752296Psychological Concomitants of Morquio Syndrome (The MAP Study)

N/A

BMN 110

N/A

Clinical Trials (1)

NCT01858103BMN 110 US Expanded Access Program

N/A

Questionnaire and interview

Hemophilia

N/A

Clinical Trials (1)

NCT05314751Learning to Live With Non-severe Haemophilia

N/A

Diet

N/A

Clinical Trials (1)

NCT03820804Nutritional Status in Phenylketonuria

N/A

Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase

Acid Sphingomyelinase Deficiency

N/A

Clinical Trials (1)

NCT03856203Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase

N/A

Dynamic Gait Analysis in Children With Mucopolysaccharidosis Type IVa

Mucopolysaccharidosis IV

N/A

Clinical Trials (1)

NCT02153255Dynamic Gait Analysis in Children With Mucopolysaccharidosis Type IVa

N/A

Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in A

MPS VI

N/A

Clinical Trials (1)

NCT01387854Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02

N/A

Confirmatory Testing

N/A

Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)

Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)

N/A

Clinical Trials (1)

NCT00214773Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)

N/A

Enzyme testing

Mucopolysaccharidosis IV A

N/A

Clinical Trials (1)

NCT01707433Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease

N/A

Pegvaliase

N/A

Clinical Trials (2)

NCT07477691Immune Modulation During Palynziq® Treatment in Adults (IMPALA)

Phase 4

NCT03792451Nutrition Status of Adults Treated With Pegvaliase

N/A

A Cross-specialty Collaboration Platform for Mucopolysaccharidosis Confirmative Diagnosis

Mucopolysaccharidoses

N/A

Clinical Trials (1)

NCT03017677A Cross-specialty Collaboration Platform for Mucopolysaccharidosis Confirmative Diagnosis

N/A

Achondroplasia Natural History Multicenter Clinical Study

N/A

Clinical Trials (1)

NCT02597881Achondroplasia Natural History Multicenter Clinical Study

N/A

BMN190, recombinant human tripeptidyl peptidase-1

CLN2 Disease

N/A

Clinical Trials (1)

NCT02963350A Multicenter, Multi-national Open-label Program to Provide BMN 190 to Patients Diagnosed With CLN2 Disease

N/A

A Prospective Clinical Study of Phenylketonuria (PKU)

N/A

Clinical Trials (1)

NCT04452513A Prospective Clinical Study of Phenylketonuria (PKU)

N/A

Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Marot

Morquio Syndrome A

N/A

Clinical Trials (1)

NCT01961518Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome

N/A

Lifetime Impact of Achondroplasia Study in Europe-LIAISE

Late-onset Pompe Patients Untreated or Treated With rhGAA

N/A

Clinical Trials (1)

NCT03449368Lifetime Impact of Achondroplasia Study in Europe-LIAISE

N/A

A Multicenter Multinational Observational Study of Children With Hypochondroplasia

Hypochondroplasia

N/A

Clinical Trials (1)

NCT06212947A Multicenter Multinational Observational Study of Children With Hypochondroplasia

N/A

A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease

N/A

Clinical Trials (1)

NCT02221362A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease

N/A

Interview

Morquio Disease

N/A

Clinical Trials (1)

NCT03150069Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them?

N/A

KuvanTM Therapy

N/A

Clinical Trials (1)

NCT00688844Nutritional and Neurotransmitter Changes in PKU Subjects on BH4

N/A

New Imaging Technology to Assess Effect of Enzyme Replacment Therapy on Eye Disease Progession in Mu

Mucopolysaccharidoses

N/A

Clinical Trials (1)

NCT02583152New Imaging Technology to Assess Effect of Enzyme Replacment Therapy on Eye Disease Progession in Mucopolysacchardiosis

N/A

Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical Conditions

Rare Genetic Disorders

N/A

Clinical Trials (1)

NCT02365376Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical Conditions

N/A

A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

MPS IV A

N/A

Clinical Trials (1)

NCT00787995A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

N/A

Sapropterin dihydrochloride