BioMarin Pharmaceutical

BioMarin Pharmaceutical(BMRN)

NOVATO, CA
Pharmaceutical1 H-1B visas (FY2023)
WebsiteLinkedInCareers Page

Focus: Biologics

BioMarin Pharmaceutical is a life sciences company focused on Biologics.

Rare DiseasesCardiovascularOncologyNephrology
Funding Stage
PUBLIC
Open Jobs
0

Products & Portfolio (8)

ALDURAZYME
laronidase
LOE Approaching
INTRAVENOUS · VIAL
(GAG). Mucopolysaccharidosis I (MPS I) is characterized by the deficiency of α-L-iduronidase, a lysosomal hydrolase which catalyzes the hydrolysis of terminal α-L-iduronic acid residues of dermatan sulfate and heparan sulfate. Reduced or absent α-L-iduronidase activity results in the accumulation of the GAG substrates, dermatan sulfate and heparan sulfate, throughout the body and leads to widespread cellular, tissue, and organ dysfunction. The rationale of ALDURAZYME therapy in MPS I is to provide exogenous enzyme for uptake into lysosomes and increase the catabolism of GAG. ALDURAZYME uptake by cells into lysosomes is most likely mediated by the mannose-6-phosphate-terminated oligosaccharide chains of laronidase binding to specific mannose-6-phosphate receptors. Because many proteins in the blood are restricted from entry into the central nervous system (CNS) by the blood brain barrier, effects of intravenously administered ALDURAZYME on cells within the CNS cannot be inferred from activity in sites outside the CNS. The ability of ALDURAZYME to cross the blood brain barrier has not been evaluated in animal models or in clinical studies.
Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I)for the treatment of patients with the Scheie form of MPS I
2003
30
BRINEURA
cerliponase alfa
Peak
INJECTION · INJECTABLE
CLN2 disease is a neurodegenerative disease caused by deficiency of the lysosomal enzyme tripeptidyl peptidase-1 (TPP1), which catabolizes polypeptides in the CNS. TPP1 has no known substrate specificity. Deficiency in TPP1 activity results in the accumulation of lysosomal storage materials normally metabolized by this enzyme in the central nervous system (CNS), leading to progressive decline in motor function. Cerliponase alfa (rhTTP1), a proenzyme, is taken up by target cells in the CNS and is translocated to the lysosomes through the Cation Independent Mannose-6-Phosphate Receptor (CI-MPR, also known as M6P/IGF2 receptor). Cerliponase alfa is activated in the lysosome and the activated proteolytic form of rhTPP1 cleaves tripeptides from the N-terminus of proteins.
2017
30
KUVAN
sapropterin dihydrochloride
LOE Approaching
ORAL · TABLET
BH4, the cofactor for the enzyme phenylalanine hydroxylase (PAH). PAH hydroxylates Phe through an oxidative reaction to form tyrosine. In patients with PKU, PAH activity is absent or deficient. Treatment with BH4 can activate residual PAH enzyme activity, improve the normal oxidative metabolism of Phe, and decrease Phe levels in some patients.
blood phenylalanine (Phe) levels in adultolder with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin‑ (BH4‑) responsive Phenylketonuria (PKU)
2007
25
KUVAN
sapropterin dihydrochloride
Peak
ORAL · POWDER
BH4, the cofactor for the enzyme phenylalanine hydroxylase (PAH). PAH hydroxylates Phe through an oxidative reaction to form tyrosine. In patients with PKU, PAH activity is absent or deficient. Treatment with BH4 can activate residual PAH enzyme activity, improve the normal oxidative metabolism of Phe, and decrease Phe levels in some patients.
blood phenylalanine (Phe) levels in adultolder with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin‑ (BH4‑) responsive Phenylketonuria (PKU)
2013
0
NAGLAZYME
galsulfase
LOE Approaching
INJECTION · INJECTABLE
GAG. MPS VI is characterized by the absence or marked reduction in N-acetylgalactosamine 4-sulfatase. The sulfatase activity deficiency results in the accumulation of the GAG substrate, dermatan sulfate, throughout the body. This accumulation leads to widespread cellular, tissue, and organ dysfunction. NAGLAZYME is intended to provide an exogenous enzyme that will be taken up into lysosomes and increase the catabolism of GAG. Galsulfase uptake by cells into lysosomes is most likely mediated by the binding of mannose-6-phosphate-terminated oligosaccharide chains of galsulfase to specific mannose-6-phosphate receptors.
2005
30
PALYNZIQ
pegvaliase-pqpz
Peak
INJECTION · INJECTABLE
Pegvaliase-pqpz is a PEGylated phenylalanine ammonia lyase (PAL) enzyme that converts phenylalanine to ammonia and trans -cinnamic acid. It substitutes for the deficient phenylalanine hydroxylase (PAH) enzyme activity in patients with PKU and reduces blood phenylalanine concentrations.
blood phenylalanine concentrations in adultolder with phenylketonuria (PKU)
2018
30
VIMIZIM
elosulfase alfa
Peak
INJECTION · INJECTABLE
2014
30
VOXZOGO
vosoritide
Peak
PeptideSUBCUTANEOUS · POWDER
2021
0

Pipeline & Clinical Trials

N/A
Clinical Trials (1)
NCT04404530Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
N/A
N/A
Clinical Trials (1)
NCT02636686Extension Study of Drisapersen in DMD Subjects
N/A
N/A
Clinical Trials (1)
NCT03820804Nutritional Status in Phenylketonuria
N/A
The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With
Classical Phenylketonuria(PKU)
N/A
Clinical Trials (1)
NCT01650909The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With Classical PKU
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
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
Questionnaire and interview
Hemophilia
N/A
Clinical Trials (1)
NCT05314751Learning to Live With Non-severe Haemophilia
N/A
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
N/A
N/A
Clinical Trials (1)
NCT01707433Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease
N/A
Thoracic MRI
Pompe Disease
N/A
Clinical Trials (1)
NCT02354664Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
N/A
A Prospective Clinical Study of Phenylketonuria (PKU)
Phenylketonurias
N/A
Clinical Trials (1)
NCT04452513A Prospective Clinical Study of Phenylketonuria (PKU)
N/A
N/A
Sapropterin
Phenylketonuria
N/A
Clinical Trials (1)
NCT00730080Sapropterin in Individuals With Phenylketonuria
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
A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease
Late-onset Pompe Patients Untreated or Treated With rhGAA
N/A
Clinical Trials (1)
NCT02221362A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease
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
Sapropterin
Phenylketonuria
N/A
Clinical Trials (1)
NCT00964236The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)
N/A
A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
Achondroplasia
N/A
Clinical Trials (1)
NCT01603095A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
N/A
Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis
Mucopolysaccharidosis Type IVA
N/A
Clinical Trials (1)
NCT01733615Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA
N/A
VIrtual STudy in Achondroplasia for the US (VISTA)
Achondroplasia
N/A
Clinical Trials (1)
NCT06168201VIrtual STudy in Achondroplasia for the US (VISTA)
N/A
Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninem
Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency
N/A
Clinical Trials (1)
NCT01016392Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency
N/A
A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults
Phenylketonuria (PKU)
N/A
Clinical Trials (1)
NCT03505125A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults
N/A
N/A
Clinical Trials (1)
NCT01541397Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy
N/A
N/A
Clinical Trials (1)
NCT02294877A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
N/A
Study to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU) With Maladaptive Be
Phenylketonuria
N/A
Clinical Trials (1)
NCT00728676Study to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU) With Maladaptive Behaviors
N/A
Administration of CANTAB and Subject Global Assessment
Phenylketonuria
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
N/A
Clinical Trials (1)
NCT02156674Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome
N/A
Pegvaliase-Pqpz
Phenylketonurias
N/A
Clinical Trials (1)
NCT05356377Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU
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
Achondroplasia Natural History Multicenter Clinical Study
Achondroplasia
N/A
Clinical Trials (1)
NCT02597881Achondroplasia Natural History Multicenter Clinical Study
N/A
N/A
Clinical Trials (1)
NCT00778206PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
N/A
KuvanTM Therapy
Phenylketonuria
N/A
Clinical Trials (1)
NCT00688844Nutritional and Neurotransmitter Changes in PKU Subjects on BH4
N/A
CLN2 Treatment
Batten Disease
N/A
Clinical Trials (1)
NCT03862274Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease
N/A
Lifetime Impact of Achondroplasia Study in Europe-LIAISE
Achondroplasia
N/A
Clinical Trials (1)
NCT03449368Lifetime Impact of Achondroplasia Study in Europe-LIAISE
N/A
N/A
Clinical Trials (1)
NCT04368624PKU Skin Stripping
N/A
Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase
Phenylketonurias
N/A
Clinical Trials (1)
NCT03856203Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase
N/A
N/A
Clinical Trials (1)
NCT01806051A Pilot Study on Diurnal Variation
N/A
A Study to Assess Growth in Children With Idiopathic Short Stature
Idiopathic Short Stature
N/A
Clinical Trials (1)
NCT06309979A Study to Assess Growth in Children With Idiopathic Short Stature
N/A
Gait Analysis in MPS IVA
MPS IVA
N/A
Clinical Trials (1)
NCT01920828Gait Analysis in MPS IVA
N/A
Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia
Phenylketonuria
N/A
Clinical Trials (1)
NCT01869972Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia
N/A
ATHN 11: Liver Transplantation Outcomes Study
Hemophilia A and B
N/A
Clinical Trials (1)
NCT04055051ATHN 11: Liver Transplantation Outcomes Study
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
N/A
Clinical Trials (1)
NCT05813678A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
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
N/A
Clinical Trials (1)
NCT03792451Nutrition Status of Adults Treated With 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
N/A
Clinical Trials (1)
NCT06305234A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
N/A
Lifetime Impact Study for Achondroplasia
Achondroplasia
N/A
Clinical Trials (1)
NCT03872531Lifetime Impact Study for Achondroplasia
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

Open Jobs (0)

No open positions listed yet. Check their careers page directly.

Interview Prep Quick Facts
Founded: 1997
Portfolio: 8 approved products, 148 clinical trials
Top TAs: Rare Diseases, Cardiovascular, Nephrology
H-1B (2023): 1 approval
SEC Filings: 2 available
Portfolio Health
Peak5 (63%)
LOE Approaching3 (38%)
8 total products
Therapeutic Area Focus
Rare Diseases
6 marketed79 pipeline
Cardiovascular
6 pipeline
Nephrology
2 pipeline
Oncology
2 pipeline
Hematology
1 pipeline
Marketed
Pipeline

Financials (FY2025)

Revenue
$2.4B15%
R&D Spend
$747M(31%)15%
Net Income
$168M
Cash
$943M
Source: SEC 10-K Filing

Visa Sponsorship

Sponsors Work Visas
H-1B Petitions (FY2023)
1
Approved
0
Denied
100%
Rate

Source: USCIS H-1B Employer Data Hub