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BIOMARIN PHARMACEUTICAL INC (BMRN)

BioMarin Pharmaceutical Inc. specializes in creating and bringing to market treatments for individuals suffering from severe and often fatal rare disorders and other critical medical conditions. The company currently markets several key pharmaceutical products. Among these are Vimizim, an enzyme replacement therapy targeting mucopolysaccharidosis (MPS) IV type A, a specific lysosomal storage disorder, and Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for individuals with MPS VI. Another notable offering is Kuvan, a unique synthetic oral version of 6R-BH4, prescribed for phenylketonuria (PKU), an inherited metabolic condition. Further expanding its product portfolio, BioMarin provides Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme administered via subcutaneous injection to lower blood Phe levels. Also available are Brineura, a recombinant human tripeptidyl peptidase 1 used in treating ceroid lipofuscinosis type 2, a variant of Batten disease; Voxzogo, a daily injectable c-type natriuretic peptide analog for achondroplasia; and Aldurazyme, a refined protein mirroring the naturally occurring human enzyme alpha-L-iduronidase. Beyond its established products, BioMarin has a robust development pipeline. This includes valoctocogene roxaparvovec, an adeno-associated virus vector undergoing Phase III clinical trials for severe hemophilia A. Additionally, BMN 307, an AAV5-mediated gene therapy, is in Phase 1/2 trials, aiming to normalize blood phenylalanine levels in PKU patients, while BMN 255 is also in Phase 1/2 trials for primary hyperoxaluria. Its clientele spans specialty pharmacies, hospitals, international government health bodies, and a network of distributors and pharmaceutical wholesalers. These services reach patients and healthcare providers across the United States, Europe, Latin America, and other global markets. BioMarin maintains strategic licensing and collaborative alliances with partners such as Sarepta Therapeutics, Ares Trading S.A., Catalyst Pharmaceutical Partners, Inc., and Asubio Pharma Co., Ltd. Established in 1996, the company's corporate headquarters are located in San Rafael, California.

Sector: Pharmaceutical Preparations

Industry: Biotechnology

BioMarin Pharmaceutical Inc. logo

BioMarin Pharmaceutical Inc.

NASDAQ:BMRNHealthcare
+1.77%$58.51
AH$58.56+0.08%vs close
Mkt Cap$11.33B
Float193.05M
O/S193.57M
Vol
$ Vol
RVOL
Inst Own99.8%
SI6.2%
Earnings
Dilution
BioMarin Pharmaceutical Inc. logoNASDAQ:BMRN$58.51+1.77%
Upcoming Catalysts
Phase Distribution
112 lead-sponsored trials·26 active·61 completed
Phase 17
Phase 1/214
Phase 229
Phase 2/31
Phase 326
Phase 44
Not applicable31
(59 more as collaborator)

Trials need funding. See this company's dilution risk and cash position.

Recent Deals
  • GC Biopharma Corp. and Hanmi Pharm Co., Ltd.LICENSE IN
    $70M upfront/ $405M totalOct 29, 2026
    HM15421/GC1134A
Clinical Trials
Sponsor role
Status
Phase
  • Phase 4
    Completed

    A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI

    Naglazyme

    Mucopolysaccharidosis VI, Maroteaux-Lamy Syndrome

    Enrollment
    4
    Start
    May 2006
    Primary Completion
    Apr 2009
    Completion
    Apr 2009
    NCT00299000
  • Phase 4
    Completed

    Kuvan®'s Effect on the Cognition of Children With Phenylketonuria

    Kuvan®

    Phenylketonuria

    Enrollment
    34
    Start
    Oct 2013
    Primary Completion
    Jan 2023
    Completion
    Jan 2023
    NCT01965912
  • Phase 4
    Completed

    Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq

    RDD to Palynziq

    Phenylketonuria, PKU

    Enrollment
    9
    Start
    Feb 2025
    Primary Completion
    Aug 2026
    Completion
    Aug 2026
    NCT06780332
  • Phase 4
    Recruiting

    Immune Modulation During Palynziq® Treatment in Adults (IMPALA)

    Pegvaliase, Methotrexate

    Phenylketonuria

    Enrollment
    12
    Start
    May 2026
    Primary Completion
    Sep 2027est.
    Completion
    Sep 2027est.
    NCT07477691
  • Phase 3
    Completed

    Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI

    Placebo, N-acetylgalactosamine 4-sulfatase

    Mucopolysaccharidosis VI

    Enrollment
    39
    Start
    Sep 2003
    Primary Completion
    -
    Completion
    Mar 2004
    NCT00067470
  • Phase 3
    Completed

    Study of rhASB in Patients With Mucopolysaccharidosis VI

    N-acetylgalactosamine 4-sulfatase, Placebo/rhASB

    Mucopolysaccharidosis VI

    Enrollment
    39
    Start
    Feb 2004
    Primary Completion
    Oct 2006
    Completion
    Oct 2006
    NCT00104234
  • Phase 3
    Completed

    Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels

    sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin

    Phenylketonurias

    Enrollment
    89
    Start
    Mar 2005
    Primary Completion
    -
    Completion
    Feb 2006
    NCT00104247
  • Phase 3
    Completed

    A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels

    sapropterin dihydrochloride

    Phenylketonurias

    Enrollment
    100
    Start
    Nov 2005
    Primary Completion
    -
    Completion
    Jun 2006
    NCT00225615
  • Phase 3
    Completed

    Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet

    Sapropterin Dihydrochloride, Placebo

    Phenylketonurias

    Enrollment
    45
    Start
    Feb 2006
    Primary Completion
    -
    Completion
    Nov 2006
    NCT00272792
  • Phase 3
    Completed

    Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006

    sapropterin dihydrochloride

    Phenylketonuria

    Enrollment
    111
    Start
    Jul 2006
    Primary Completion
    Aug 2009
    Completion
    Aug 2009
    NCT00332189
  • Phase 3
    Completed

    Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU

    sapropterin dihydrochloride

    Phenylketonuria

    Enrollment
    95
    Start
    Feb 2009
    Primary Completion
    Sep 2018
    Completion
    Sep 2018
    NCT00838435
  • Phase 3
    Completed

    Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients

    Sapropterin dihydrochloride, Placebo

    Phenylketonuria

    Enrollment
    206
    Start
    Aug 2010
    Primary Completion
    Mar 2013
    Completion
    Mar 2013
    NCT01114737
  • Phase 3
    Completed

    A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

    BMN 110 Weekly, Placebo

    MPS IV A

    Enrollment
    177
    Start
    Feb 2011
    Primary Completion
    Aug 2012
    Completion
    Aug 2012
    NCT01275066
  • Phase 3
    Completed

    Kuvan® in Phenylketonuria Patients Less Than 4 Years Old

    Kuvan®

    Phenylketonuria

    Enrollment
    56
    Start
    Jun 2011
    Primary Completion
    Jul 2014
    Completion
    Feb 2017
    NCT01376908
  • Phase 3
    Completed

    Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

    BMN 110 - Weekly, BMN 110 - Every Other Week

    Mucopolysaccharidosis IV A, Morquio A Syndrome +1

    Enrollment
    173
    Start
    Jul 2011
    Primary Completion
    Jun 2016
    Completion
    Jun 2016
    NCT01415427
  • Phase 3
    Terminated

    A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects

    Drisapersen, Drisapersen

    Muscular Dystrophies

    Enrollment
    53
    Start
    May 2013
    Primary Completion
    Jun 2016
    Completion
    Oct 2016
    NCT01803412
  • Phase 3
    Completed

    An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165

    BMN 165

    Phenylketonuria

    Enrollment
    261
    Start
    May 2013
    Primary Completion
    Nov 2015
    Completion
    Nov 2015
    NCT01819727
  • Phase 3
    Completed

    Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU

    BMN165 20mg/day, BMN165 40mg/day

    Phenylketonuria (PKU)

    Enrollment
    215
    Start
    Jul 2013
    Primary Completion
    Jan 2016
    Completion
    Feb 2019
    NCT01889862
  • Phase 3
    Terminated

    BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)

    BMN 701

    Late-onset Pompe Disease

    Enrollment
    24
    Start
    Apr 2014
    Primary Completion
    Sep 2016
    Completion
    Sep 2016
    NCT01924845
  • Phase 3
    Completed

    BMN 110 Phase 3B in Australian Patients

    BMN 110

    Mucopolysaccharidosis IVA (Morquio A Syndrome)

    Enrollment
    13
    Start
    Jul 2013
    Primary Completion
    Jul 2016
    Completion
    Dec 2016
    NCT01966029
  • Phase 3
    Completed

    A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

    BMN 111, Placebo

    Achondroplasia

    Enrollment
    121
    Start
    Dec 2016
    Primary Completion
    Oct 2019
    Completion
    Oct 2019
    NCT03197766
  • Phase 3
    Completed

    Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)

    valoctocogene roxaparvovec

    Hemophilia A

    Enrollment
    144
    Start
    Dec 2017
    Primary Completion
    Nov 2020
    Completion
    Nov 2024
    NCT03370913
  • Phase 3
    Completed

    Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg

    Valoctocogene Roxaparvovec

    Hemophilia A

    Enrollment
    1
    Start
    Mar 2018
    Primary Completion
    May 2019
    Completion
    Jun 2023
    NCT03392974
  • Phase 3
    Active, not recruiting

    An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

    BMN 111

    Achondroplasia

    Enrollment
    119
    Start
    Dec 2017
    Primary Completion
    Jun 2031est.
    Completion
    Jun 2031est.
    NCT03424018
  • Phase 3
    Completed

    Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU

    Pegvaliase

    Phenylketonuria (PKU)

    Enrollment
    37
    Start
    Sep 2018
    Primary Completion
    Jan 2021
    Completion
    Jan 2021
    NCT03694353

1-25 of 112

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