COMPANY

BioMarin Pharmaceutical

HQ: San Rafael, CA source · biomarin.com ↗ · West Coast

FDA-approved therapeutic company Clinical Phase 3

Location: San Rafael, CA · source · Oct 15, 2026

Profile checked Oct 10, 2026

Keywords: Phenylketonuria Achondroplasia Hypochondroplasia Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 Hemophilia A Idiopathic Short Stature Jansky-Bielschowsky Disease Batten Disease CLN2 Disease CLN2 Disorder PKU Noonan Syndrome Duchenne Muscular Dystrophy Hemophilia A With Inhibitor Hemophilia A With Anti Factor VIII Alpha 1-Antitrypsin Deficiency Maternal Phenylketonuria Gene Therapy Clotting Disorders Blood Disorder Severe Haemophilia A Hyperoxaluria Nonalcoholic Fatty Liver Disease Kidney Stone Hereditary Angioedema HAE Mucopolysaccharidosis IV Type A Morquio A Syndrome MPS IVA Late-onset Pompe Disease Hyperphenylalaninaemia Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency Vosoritide Pegvaliase Valoctocogene roxaparvovec BMN 111 BMN 351 RDD to Palynziq

112Trials (company is sponsor)1FDA records (2 yrs)4NIH grants

Timeline

May 29, 2026 Trial start Phase 4: Immune Modulation During Palynziq® Treatment in Adults (IMPALA) source ↗
Apr 20, 2026 Trial start Phase 2/Phase 3: Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia source ↗
Apr 20, 2026 Trial start Phase 2: An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy source ↗
Feb 27, 2026 FDA FDA New indication / efficacy supplement: PALYNZIQ (pegvaliase-pqpz) — BLA761079/31, Feb 27, 2026. Applicant at approval: BioMarin Pharmaceutical Inc. source ↗
Feb 12, 2026 Debt Debt: $850M (not counted as a raise), announced Feb 12, 2026. Source: SEC filing. source ↗
Jul 30, 2025 Trial start Phase 2: A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months source ↗
Jun 20, 2025 Trial start Phase 3: Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia source ↗
Feb 21, 2025 Trial start Phase 1: Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants source ↗
Feb 7, 2025 Trial start Phase 4: Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq source ↗
Nov 22, 2024 Trial start Phase 2: A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment source ↗
Oct 21, 2024 Trial start 2: A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature source ↗
Aug 8, 2024 Trial start —: A Study to Assess Growth in Children With Idiopathic Short Stature source ↗
Jun 17, 2024 Trial start Phase 3: Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia source ↗
Jan 20, 2024 Trial start —: A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade) source ↗
Jan 3, 2024 Trial start 1: A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy source ↗
Dec 25, 2023 Trial start 3: Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A source ↗
Nov 27, 2023 Trial start —: A Multicenter Multinational Observational Study of Children With Hypochondroplasia source ↗
Sep 26, 2023 Trial start Phase 1: A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And Hyperoxaluria source ↗
Mar 15, 2023 Trial start Phase 1: Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants source ↗
Mar 14, 2023 Trial start Not phased (device or behavioral study): A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV) source ↗
Feb 21, 2023 Trial start —: VIrtual STudy in Achondroplasia for the US (VISTA) source ↗
Jan 1, 2023 Trial start —: A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401) source ↗
Nov 22, 2022 Trial start —: A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding source ↗
Jun 17, 2022 Trial start Phase 3: Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria source ↗
Jun 6, 2022 Trial start —: A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace) source ↗

Showing 25 of 52 dated events.

Upcoming milestones

2027-03 (ESTIMATED) · Phase 2 · A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment · NCT06668805
Apr 14, 2027 (ESTIMATED) · 1 · A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy · NCT06280209
Sep 30, 2027 (ESTIMATED) · Phase 4 · Immune Modulation During Palynziq® Treatment in Adults (IMPALA) · NCT07477691
2027-12 (ESTIMATED) · Phase 2 · A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia · NCT04554940
2027-12 (ESTIMATED) · Phase 2 · A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH) · NCT02724228
2027-12 (ESTIMATED) · Phase 1/Phase 2 · AAV Gene Therapy Study for Subjects with PKU · NCT04480567
2028-02 (ESTIMATED) · · VIrtual STudy in Achondroplasia for the US (VISTA) · NCT06168201
Jun 30, 2028 (ESTIMATED) · Phase 2 · A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months · NCT07126262

Estimated primary completion per ClinicalTrials.gov. Dates move.

Funding

Debt: $850.0M. Last 2 years.

Feb 12, 2026 Debt: $850M (not counted as a raise), announced Feb 12, 2026. Source: SEC filing. source ↗

Clinical trials (lead sponsor) Showing 50 of 112

Record	Title	Phase	Status	Start	Primary completion
NCT06780332	Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq	Phase 4	Completed	Feb 7, 2025	Aug 17, 2026 (actual)
NCT07441876	Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia	Phase 2/Phase 3	Recruiting	Apr 20, 2026	2029-06 (estimated)
NCT06668805	A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment	Phase 2	Active, not recruiting	Nov 22, 2024	2027-03 (estimated)
NCT07073014	Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia	Phase 3	Enrolling by invitation	Jun 20, 2025	2044-10 (estimated)
NCT07573631	An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy	Phase 2	Enrolling by invitation	Apr 20, 2026	2031-12 (estimated)
NCT05270837	Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria	Phase 3	Active, not recruiting	Jun 17, 2022	Jan 14, 2025 (actual)
NCT04684940	Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors	Phase 1/Phase 2	Completed	Dec 10, 2020	Apr 20, 2026 (actual)
NCT06168201	VIrtual STudy in Achondroplasia for the US (VISTA)	Not listed	Recruiting	Feb 21, 2023	2028-02 (estimated)
NCT04476862	Cerliponase Alfa Observational Study in the US	Not listed	Active, not recruiting	Aug 19, 2020	Aug 31, 2030 (estimated)
NCT06280209	A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy	1	Active, not recruiting	Jan 3, 2024	Apr 14, 2027 (estimated)
NCT06309979	A Study to Assess Growth in Children With Idiopathic Short Stature	Not listed	Recruiting	Aug 8, 2024	Dec 31, 2040 (estimated)
NCT07477691	Immune Modulation During Palynziq® Treatment in Adults (IMPALA)	Phase 4	Recruiting	May 29, 2026	Sep 30, 2027 (estimated)
NCT06382155	A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature	2	Recruiting	Oct 21, 2024	2026-06 (estimated)
NCT06738017	Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants	Phase 1	Active, not recruiting	Feb 21, 2025	Aug 19, 2026 (estimated)
NCT07126262	A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months	Phase 2	Recruiting	Jul 30, 2025	Jun 30, 2028 (estimated)
NCT05813678	A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)	Not listed	Recruiting	Jun 6, 2022	Nov 1, 2033 (estimated)
NCT05579548	A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding	Not listed	Recruiting	Nov 22, 2022	Oct 4, 2032 (estimated)
NCT06305234	A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)	Not listed	Recruiting	Jan 20, 2024	Nov 18, 2033 (estimated)
NCT06224907	Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A	3	Active, not recruiting	Dec 25, 2023	Apr 16, 2025 (actual)
NCT04554940	A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia	Phase 2	Active, not recruiting	Oct 10, 2020	2027-12 (estimated)
NCT03989947	An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia	Phase 2	Active, not recruiting	Jun 12, 2019	2038-05 (estimated)
NCT03424018	An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia	Phase 3	Active, not recruiting	Dec 12, 2017	2031-06 (estimated)
NCT02724228	A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)	Phase 2	Active, not recruiting	Jan 26, 2016	2027-12 (estimated)
NCT06455059	Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia	Phase 3	Active, not recruiting	Jun 17, 2024	Aug 1, 2026 (estimated)
NCT05768386	A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)	Not listed	Enrolling by invitation	Jan 1, 2023	2040-01 (estimated)
NCT04323098	Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A	Phase 3	Completed	Dec 8, 2020	Jan 27, 2023 (actual)
NCT03520712	Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5	Phase 1/Phase 2	Terminated	Apr 24, 2018	Aug 7, 2024 (actual)
NCT02576795	Gene Therapy Study in Severe Haemophilia A Patients (270-201)	Phase 1/Phase 2	Completed	Sep 28, 2015	Feb 14, 2024 (actual)
NCT06212947	A Multicenter Multinational Observational Study of Children With Hypochondroplasia	Not listed	Recruiting	Nov 27, 2023	Dec 31, 2043 (estimated)
NCT03370913	Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)	Phase 3	Completed	Dec 19, 2017	Nov 16, 2020 (actual)
NCT02678689	A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease	Phase 2	Completed	Jan 22, 2016	Apr 20, 2022 (actual)
NCT04480567	AAV Gene Therapy Study for Subjects with PKU	Phase 1/Phase 2	Active, not recruiting	Sep 24, 2020	2027-12 (estimated)
NCT05580692	A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV)	Not phased (device or behavioral study)	Terminated	Mar 14, 2023	Jul 31, 2024 (actual)
NCT03583697	A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia	Phase 2	Completed	Jun 13, 2018	Jan 26, 2022 (actual)
NCT06138327	A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And Hyperoxaluria	Phase 1	Withdrawn	Sep 26, 2023	Mar 25, 2024 (actual)
NCT05121376	A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema	Phase 1/Phase 2	Active, not recruiting	Feb 15, 2022	2028-11 (estimated)
NCT04560933	A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A	Not listed	Completed	Aug 25, 2020	Sep 20, 2023 (actual)
NCT02294877	A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)	Not listed	Completed	2014-09	Feb 21, 2024 (actual)
NCT05813314	Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants	Phase 1	Terminated	Mar 15, 2023	Jun 14, 2023 (actual)
NCT03392974	Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg	Phase 3	Completed	Mar 14, 2018	May 22, 2019 (actual)
NCT01965912	Kuvan®'s Effect on the Cognition of Children With Phenylketonuria	Phase 4	Completed	2013-10	Jan 4, 2023 (actual)
NCT04452513	A Prospective Clinical Study of Phenylketonuria (PKU)	Not listed	Completed	Oct 11, 2019	Jun 2, 2022 (actual)
NCT02191917	A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)	Not listed	Terminated	Oct 21, 2014	Jun 22, 2016 (actual)
NCT00778206	PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry	Not listed	Completed	2008-09	Jan 29, 2021 (actual)
NCT02485899	An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease	Phase 1/Phase 2	Completed	2015-02	Dec 10, 2020 (actual)
NCT01016392	Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency	Not listed	Completed	2009-12	May 31, 2021 (actual)
NCT03197766	A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia	Phase 3	Completed	Dec 12, 2016	Oct 30, 2019 (actual)
NCT03694353	Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU	Phase 3	Completed	Sep 13, 2018	Jan 13, 2021 (actual)
NCT00924703	Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)	Phase 2	Completed	Jan 13, 2010	Jan 31, 2019 (actual)
NCT00789568	A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects	1	Completed	2008-10	2009-01 (actual)

FDA records (last 2 years)

Feb 27, 2026 FDA New indication / efficacy supplement: PALYNZIQ (pegvaliase-pqpz) — BLA761079/31, Feb 27, 2026. Applicant at approval: BioMarin Pharmaceutical Inc. source ↗

NIH awards 4

Annual award amounts. Total $1.8M.

Aug 16, 2002 NIH award 5R44DK059205-03 FY2002: $790,623 awarded for that fiscal year; activity R44 (SBIR); PI KAKKIS, EMIL DENNIS; recipient as written: BIOMARIN PHARMACEUTICAL, INC., NOVATO, CA. PRODUCTION OF BETA-GLUCURONIDASE source ↗
Sep 10, 2001 NIH award 4R44DK059205-02 FY2001: $756,088 awarded for that fiscal year; activity R44 (SBIR); PI KAKKIS, EMIL DENNIS; recipient as written: BIOMARIN PHARMACEUTICAL, INC., NOVATO, CA. PRODUCTION OF BETA-GLUCURONIDASE source ↗
Mar 15, 2001 NIH award 1R44DK059205-01 FY2001: $186,641 awarded for that fiscal year; activity R44 (SBIR); PI KAKKIS, EMIL DENNIS; recipient as written: BIOMARIN PHARMACEUTICAL, INC., NOVATO, CA. PRODUCTION OF BETA-GLUCURONIDASE source ↗
Sep 30, 1998 NIH award 1R43HL062924-01 FY1998: $100,000 awarded for that fiscal year; activity R43 (SBIR); PI CONARY, JON T; recipient as written: BIOMARIN PHARMACEUTICAL, INC., NOVATO, CA. OPTICALLY ACTIVATED GENETIC THERAPIES source ↗

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