Sponsor · clinical trials

BioMarin Pharmaceutical clinical trials

Source: ClinicalTrials.gov, U.S. National Library of Medicine and the EU Clinical Trials Information System (CTIS) — current trials in our USA, UK, Australia, Italy, Germany, Spain, Japan, France, Canada, Turkey, South Korea, Brazil, Netherlands, Belgium, Israel, Poland and EU (CTIS) registers with BioMarin Pharmaceutical as lead sponsor. Synced 12 Nov 2026

27Current trials

10Recruiting now

5Drugs in these trials

17Trial registers

Maintained by Sreepriya Prasannan

MSc Digital Transformation of Life Sciences (Innopharma Education / Griffith College); MSc & BSc Botany

Compiled from ClinicalTrials.gov and CTIS registry records · Synced 12 Nov 2026 · Editorial standards

At a glance

BioMarin Pharmaceutical is the lead sponsor of 27 current clinical trials in our USA, UK, Australia, Italy, Germany, Spain, Japan, France, Canada, Turkey, South Korea, Brazil, Netherlands, Belgium, Israel, Poland and EU (CTIS) registers. 10 are recruiting patients now. The most common phase is Observational. The drugs named most often in its trials are Vosoritide, Pegvaliase and BMN 351. The most-studied condition is Achondroplasia.

ClinicalTrials.gov trials by status

Recruiting1038%
Active, closed1350%
By invitation312%

Trials by phase

Observational831%
Phase 2727%
Phase 3519%
Phase 1/2312%
Phase 114%
Phase 2/314%
Phase 414%

Clinical trials sponsored by BioMarin Pharmaceutical

26 on ClinicalTrials.gov

Study	Phase	Status	Start	Registers
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months NCT07126262 · Hypochondroplasia Drug: Vosoritide	Phase 2	Recruiting	2025-07	UK, USA, Germany, France, Italy, Japan, Australia
A Multicenter Multinational Observational Study of Children With Hypochondroplasia NCT06212947 · Hypochondroplasia	Observational	Recruiting	2023-11	UK, USA, Germany, France, Spain, Italy, Canada, Brazil, Japan, Australia
A Study to Assess Growth in Children With Idiopathic Short Stature NCT06309979 · ClinicalTrials.gov ↗ · Idiopathic Short Stature	Observational	Recruiting	2024-08	USA
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia NCT07441876 · Achondroplasia Drug: Vosoritide	Phase 2/3	Recruiting	2026-04	UK, USA, Italy, Poland, Canada, Japan, South Korea, Australia
A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace) NCT05813678 · Phenylketonuria (PKU) Drug: Pegvaliase	Observational	Recruiting	2022-06	USA, Germany, Italy
A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature NCT06382155 · Idiopathic Short Stature Drugs: Human Growth Hormone, Vosoritide	Phase 2	Recruiting	2024-10	USA, Germany, France, Italy, South Korea, Australia
A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding NCT05579548 · Phenylketonuria, Maternal Drug: Pegvaliase	Observational	Recruiting	2022-11	USA, Germany, Italy, Canada
Immune Modulation During Palynziq® Treatment in Adults (IMPALA) NCT07477691 · Phenylketonuria Drugs: Methotrexate, Pegvaliase	Phase 4	Recruiting	2026-05	USA
VIrtual STudy in Achondroplasia for the US (VISTA) NCT06168201 · Achondroplasia	Observational	Recruiting	2023-02	USA
A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade) NCT06305234 · Phenylketonuria (PKU) Drug: Pegvaliase	Observational	Recruiting	2024-01	USA
An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy NCT07573631 · Duchenne Muscular Dystrophy (DMD) Drug: BMN 351	Phase 2	By invitation	2026-04	UK, Spain, Italy, Netherlands, Turkey
A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401) NCT05768386 · Hemophilia A	Observational	By invitation	2023-01	UK, USA, Germany, France, Spain, Italy, Belgium, Brazil, South Korea, Australia, Israel
Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia NCT07073014 · Hypochondroplasia Drug: Vosoritide	Phase 3	By invitation	2025-06	UK, USA, Germany, France, Spain, Italy, Canada, Japan, Australia
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy NCT06280209 · Duchenne Muscular Dystrophy Drug: BMN 351	Phase 1/2	Active, closed	2024-01	UK, Spain, Italy, Netherlands, Turkey
Cerliponase Alfa Observational Study in the US NCT04476862 · Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2	Observational	Active, closed	2020-08	USA
A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia NCT04554940 · Achondroplasia Drug: Vosoritide	Phase 2	Active, closed	2020-10	UK, Australia
Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A NCT06224907 · ClinicalTrials.gov ↗ · Hemophilia A	Phase 3	Active, closed	2023-12	Japan
An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia NCT03424018 · Achondroplasia Drug: Vosoritide	Phase 3	Active, closed	2017-12	UK, USA, Germany, Spain, Turkey, Japan, Australia
AAV Gene Therapy Study for Subjects with PKU NCT04480567 · Phenylketonuria (PKU)	Phase 1/2	Active, closed	2020-09	UK, USA
A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema NCT05121376 · Hereditary Angioedema, HAE	Phase 1/2	Active, closed	2022-02	USA, Spain, Australia
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia NCT06455059 · Hypochondroplasia Drug: Vosoritide	Phase 3	Active, closed	2024-06	UK, USA, Germany, France, Spain, Italy, Canada, Japan, Australia
Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants NCT06738017 · Alpha 1-Antitrypsin Deficiency	Phase 1	Active, closed	2025-02	UK, USA
A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH) NCT02724228 · Achondroplasia Drug: Vosoritide	Phase 2	Active, closed	2016-01	UK, USA, France, Australia
A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment NCT06668805 · Noonan Syndrome Drug: Vosoritide	Phase 2	Active, closed	2024-11	USA, Germany, France, Spain, Italy, Canada, Australia
An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia NCT03989947 · Achondroplasia	Phase 2	Active, closed	2019-06	UK, USA, Japan, Australia
Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria NCT05270837 · Phenylketonuria (PKU) Drug: Pegvaliase	Phase 3	Active, closed	2022-06	USA, Germany

Registers list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Titles and statuses are copied from the registry as published.

BioMarin Pharmaceutical in the EU trials register (CTIS)

1 trial

Authorised or ongoing trials in the EU Clinical Trials Information System with BioMarin Pharmaceutical as sponsor. Some are the same study as a ClinicalTrials.gov record above; the two registries share no common ID. Trials with a site in Ireland are listed first.

Study	Phase	Status	Countries
A Multicenter, Randomized, Operationally Seamless Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 versus Vosoritide in Children with Achondroplasia 2025-523811-12-00 · CTIS ↗ · Congenital, Hereditary, and Neonatal Diseases and Abnormalities Drug: Vosoritide	Phase 3	Ongoing	Poland, Italy, Romania + 4 more

Drugs in BioMarin Pharmaceutical trials

5 compounds

Drugs and biologicals named as interventions in these trials, with the number of this sponsor's trials that name each one. Placebos and generic comparators are excluded.

Vosoritide10 Pegvaliase5 BMN 3512 Human Growth Hormone1 Methotrexate1

Conditions in BioMarin Pharmaceutical trials

14 conditions

Achondroplasia6Hypochondroplasia4Phenylketonuria (PKU)4Hemophilia A2Idiopathic Short Stature2Alpha 1-Antitrypsin Deficiency1Duchenne Muscular Dystrophy (DMD)1Duchenne Muscular Dystrophy1Late-Infantile Neuronal Ceroid Lipofuscinosis Type 21Phenylketonuria1Phenylketonuria, Maternal1Noonan Syndrome1Hereditary Angioedema1HAE1

By trial register

Register	Trials	Recruiting
Clinical trials in USA	22	10
Clinical trials in UK	14	3
Clinical trials in Australia	13	4
Clinical trials in Italy	12	6
Clinical trials in Germany	11	5
Clinical trials in Spain	9	1
Clinical trials in Japan	8	3
Clinical trials in France	8	3
Clinical trials in Canada	6	3
Clinical trials in Turkey	3	0
Clinical trials in South Korea	3	2
Clinical trials in Brazil	2	1
Clinical trials in Netherlands	2	0
Clinical trials in Belgium	1	0
Clinical trials in Israel	1	0
Clinical trials in Poland	1	1
EU register (CTIS)	1	—

A trial with sites in more than one country appears in each of those registers.

Other sponsors in Achondroplasia

Sponsor	Trials
Ascendis Pharma	2
QED Therapeutics, a BridgeBio company	2
Tyra Biosciences	1
SYSNAV	1

Sponsors with the most current trials registered for this condition.

Questions about BioMarin Pharmaceutical trials

How many clinical trials does BioMarin Pharmaceutical sponsor?

BioMarin Pharmaceutical is the lead sponsor of 26 current trials on ClinicalTrials.gov in our USA, UK, Australia, Italy, Germany, Spain, Japan, France, Canada, Turkey, South Korea, Brazil, Netherlands, Belgium, Israel and Poland registers, and 1 authorised or ongoing trial in the EU register (CTIS), as of the registry sync on 12 November 2026. Completed and withdrawn studies are not counted.

Are BioMarin Pharmaceutical trials recruiting?

Yes. 10 of its 26 ClinicalTrials.gov trials are recruiting. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.

Which drugs is BioMarin Pharmaceutical testing?

Its current trials name 5 drugs and biologicals as interventions. The most frequent are Vosoritide (10), Pegvaliase (5), BMN 351 (2), Human Growth Hormone (1) and Methotrexate (1). The number in brackets is how many of its trials name each drug.

Where does this information come from?

Every trial, status, phase and condition on this page is copied from public ClinicalTrials.gov and EU CTIS records by a daily sync, grouped by the sponsor name each registry records. It says nothing about the organisation beyond its registered trials, and it is not medical advice.

How this page is built

Everything on this page is copied from public registry records — it is not written from memory, and nothing here describes the organisation beyond its registered trials. Each trial links to its own record so you can check it yourself.

ClinicalTrials.gov (U.S. National Library of Medicine) ↗ Trial IDs, titles, lead sponsor, phase, overall status, start date, conditions and intervention names
EU Clinical Trials Information System (CTIS) ↗ EU trial numbers, titles, sponsor, phase, status, therapeutic area and member-state countries

This page is built automatically: a daily sync job reads the registry records behind our trial registers and groups every current trial by its lead sponsor name, ignoring legal-form suffixes such as Inc. or Ltd. A small, hand-checked list joins the same organisation's different registry names (for example its EU legal entity). There is no AI drafting and no per-sponsor editorial pass. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The the registries data on this page was last retrieved on 12 Nov 2026. How every register is built: methodology · fixes we have made: corrections log.

Please verify before you rely on this. This page is general information for life-science and pharmaceutical professionals. It is not medical advice, and it has not been reviewed by a clinician — our editorial team holds life-science qualifications, not clinical ones. It is not exhaustive and may not reflect the most recent label change. Always check the official prescribing information (US Prescribing Information or EU SmPC) and speak to your doctor or pharmacist before acting on anything here. Drugs in the same class are not automatically interchangeable, and approvals, brand names and indications differ between the US, the EU/Ireland (EMA/HPRA) and other regions. Spotted an error? Tell us — we correct promptly and log it.