BioMarin Pharmaceutical ( BMRN )

Share Interview Share Compensation

$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.

Help build intelligence for BioMarin Pharmaceutical

No one has shared interview or compensation data for this company yet. You'll earn 7 days of Pro access.

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.

Portfolio of 7 marketed biologics across rare diseases with meaningful patient populations generates stable revenue ($2.4B FY2025, +14% YoY) and funds 31% R&D intensity
Active pipeline of 20 trials including Phase 3 programs in PKU (Pegvaliase, Sapropterin) and Hemophilia A (valoctocogene roxaparvovec) offers near-term clinical catalysts
Gene therapy and enzyme replacement therapy focus positions you at frontier of rare disease innovation with multiple expansion opportunities in nephrology and cardiovascular disorders

Watch Out For

Zero active job postings at time of analysis suggests limited hiring or possible recruitment freeze despite revenue growth
Two legacy products (ALDURAZYME, NAGLAZYME) approaching loss of exclusivity within 5-10 years creates patent cliff exposure that may trigger restructuring

Generated by Claude from BioMarin Pharmaceutical's SEC filings, pipeline programs, hiring velocity, FDA actions, and WARN data. Inference, not editorial — verify before quoting.

Automated analysis based on publicly available data (FDA, SEC, ClinicalTrials.gov). May be incomplete or delayed. This score does not reflect insider knowledge and should not be used as the sole basis for investment or employment decisions.

VIMIZIM

Mucopolysaccharidosis IV Type A (MPS IVA)

$17M Part D

Peak

Second-largest revenue contributor; enzyme replacement therapy for rare lysosomal storage disorder with stable reimbursement and patient base.

KUVAN

Achondroplasia, Hypochondroplasia

$7M Part D

Peak6.9yr

Cofactor therapy for PKU with extended exclusivity; Phase 3 studies ongoing for expanded indications in PKU population.

VOXZOGO

Peak16.1yr

First-in-class therapy for achondroplasia with longest patent tail (2042); represents expansion into growth disorder market with significant unmet need.

BRINEURA

Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)

Peak

Gene-adjacent CNS therapy for rare pediatric neurodegenerative disease; demonstrates company's expansion into CNS and ultra-rare indications.

ALDURAZYME

Mucopolysaccharidosis I

$7M Part D

LOE Approaching

Legacy enzyme replacement therapy approaching loss of exclusivity; revenue expected to erode as biosimilar competition intensifies.

NAGLAZYME

Mucopolysaccharidosis VI

$6M Part D

LOE Approaching

Aging MPS VI therapy approaching exclusivity loss; lower revenue base reflects mature lifecycle and emerging competitive therapies.

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

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

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