Organization (Industry)
BIOMARIN PHARMACEUTICAL INC.
ClinicalTrials.gov lists BIOMARIN PHARMACEUTICAL INC. as lead sponsor of 112 trials, counting the companies grouped under it. BIOMARIN PHARMACEUTICAL INC. itself has 27 active development programs and 12 approved assets.
Search trials, drugs, companies and investigators, and follow each one to the others. Individual plan, $249/seat/mo. Card required.
Trials started per year with this organization as sponsor or collaborator, 1990 onward. Companies grouped under this one are not included. The series also counts trials where the organization is a collaborator; the lead-sponsor trial count does not.
2026 is partial: 3 registered with a 2026 start date. The hollow diamonds on the dashed line are a forecast of the full-year count, planned starts included: about 4 in 2026 and about 5 in 2027, the average of the 24 months through September, counted as registered and carried forward. The columns, the lighter one included, count trials registered so far with a start date in the year, planned starts included; the forecast looks ahead to the count for the whole year, so it is not the same measure. This method has not been backtested on organizations. In backtests on conditions with as many trial starts a year as this organization (5 to 20), forecasts made part-way through the year were typically off by about 28 percent and ran about 20 percent low against the count later registered; forecasts made before the year began were off by about 39 percent and ran about 37 percent low against the count later registered. These figures rest on a few years of history (target years from 2018 to 2025, varying by figure), place each condition as it is mapped today rather than as it was then, and score against a count for the newest year that was still being revised. Only condition series were scored.
Logo BioMarin Pharmaceutical, via Wikimedia Commons, Public domain. Shown to identify the organization; it may be a registered trademark.
This page covers BIOMARIN PHARMACEUTICAL INC. and 1 company grouped under it: 112 lead-sponsored trials (112 its own, 0 in the grouped companies) and 8 legal-entity registrations (7 its own, 1 in the grouped companies).
Companies grouped under this page (1)
| Company | Lead trials | Legal entities |
|---|---|---|
| BIOMARIN INTERNATIONAL LIMITED | 0 | 1 |
Disease areas (74)
Shown in this order: most trials first.
| Disease area | Trials |
|---|---|
| Phenylketonurias | 57 |
| Mucopolysaccharidosis IV | 21 |
| Dwarfism | 17 |
| Achondroplasia | 15 |
| Hemophilia A | 12 |
Showing 5 of 74 disease areas.
Trials by start year as sponsor or collaborator (26)
Shown in this order: most recent first. 2026 is partial: trials registered with a 2026 start date so far, not a full year.
Show the table of trials by start year as sponsor or collaborator
| Year | Trials started |
|---|---|
| 2026 (partial year) | 3 |
| 2025 | 5 |
| 2024 | 7 |
| 2023 | 9 |
| 2022 | 6 |
| 2021 | 2 |
| 2020 | 8 |
| 2019 | 7 |
| 2018 | 9 |
| 2017 | 5 |
Showing 10 of 26 years.
Prediction, not a registry value: median of 36 months from start to primary completion across 13 trials still running at the 2026-10-09 snapshot; registered median of 31 months across 76 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
Active development programs (27)
Shown in this order: max phase, then active trials.
| Drug | Disease area | Max phase | Trials |
|---|---|---|---|
| BMN 111 | Dwarfism | Phase 3 | 8 |
| BMN 111 | Achondroplasia | Phase 3 | 7 |
| Valoctocogene Roxaparvovec | Hemophilia A | Phase 3 | 7 |
| BMN 110 | Mucopolysaccharidosis IV | Phase 3 | 8 |
| BMN 333 | Dwarfism | Phase 2/3 | 1 |
Showing 5 of 27 active programs.
Approved assets (12)
Shown in this order: max phase first.
| Drug | Trials |
|---|---|
| Pegvaliase | 8 |
| KUVAN | 5 |
| N-acetylgalactosamine 4-sulfatase | 5 |
| RDD to Palynziq | 1 |
| METHOTREXATE | 1 |
Showing 5 of 12 approved assets.
Sponsor delivery record (1)
Enrollment hit target: the share of this sponsor's lead-sponsored trials whose actual enrollment reached the planned enrollment. On time: the share whose actual primary completion date was no later than the planned date. Each share counts only trials where we observed an estimated figure before the actual one, and n is that number of trials. Below 5 trials the count is shown as a fraction, not a percentage.
| Enrollment | Timeline |
|---|---|
| 35% hit target (n=31) | 53% on time (n=32) |
Journals published in (57)
Shown in this order: most published in first.
| Journal | Publications |
|---|---|
| Molecular genetics and metabolism | 20 |
| Orphanet journal of rare diseases | 9 |
| The New England journal of medicine | 7 |
| Haemophilia : the official journal of the World Federation of Hemophilia | 6 |
| Biochimica et biophysica acta | 4 |
Showing 5 of 57 journals.
US granted patents (1)
Patents are not classified by disease area in the source, so this is the organization's overall portfolio, not a split by disease.
| US patents granted | Granted in the last 5 years | Most recent grant |
|---|---|---|
| 135 | 22 | 2025-03-04 |
SEC-reported annual financials (11)
Shown in this order: newest fiscal year first; SEC EDGAR XBRL companyfacts, USD as reported.
| Fiscal year | Revenue (USD) | Net income (USD) | R&D (USD) |
|---|---|---|---|
| 2025 | 3,221,253,000 | 348,901,000 | 921,930,000 |
| 2024 | 2,853,915,000 | 426,859,000 | 747,184,000 |
| 2023 | 2,419,226,000 | 167,645,000 | 746,773,000 |
| 2022 | 2,096,039,000 | 141,561,000 | 649,606,000 |
| 2021 | 1,846,275,000 | -64,080,000 | 628,793,000 |
| 2020 | 1,860,455,000 | 854,029,000 | 628,116,000 |
| 2019 | 1,704,048,000 | -23,848,000 | 715,007,000 |
| 2018 | 1,491,212,000 | -77,211,000 | 696,328,000 |
| 2017 | 1,313,646,000 | -117,042,000 | 610,753,000 |
| 2016 | 1,116,854,000 | -630,210,000 | 661,905,000 |
Showing 10 of 11 fiscal years.
Financing and grant events (23)
Shown in this order: most recent first.
| Date | Round type | Amount (USD) | Source |
|---|---|---|---|
| 2026-06-02 | Other financing | not disclosed | Sec extracted |
| 2026-04-27 | Debt financing | 2,800 | Sec extracted |
| 2026-04-27 | Debt financing | not disclosed | Sec extracted |
| 2026-02-12 | Debt financing | 850,000,000 | Sec extracted |
| 2026-01-29 | Debt financing | not disclosed | Sec extracted |
Showing 5 of 23 events.
FDA inspection record (1)
| Latest classification | Inspections |
|---|---|
| NAI | 15 |
FDA import refusals (1)
| Refused import lines | Latest refusal |
|---|---|
| 1 | 2017-05-12 |
Manufacturing and distribution sites (1)
| Facility | Location | Regulators | Member organization |
|---|---|---|---|
| BioMarin Pharmaceutical Inc. | Novato, US | EMA, FDA, Health Canada |
Climate targets, as filed (1)
| Reporting year | Net-zero target |
|---|---|
| 2025 | not disclosed |
Workforce and board diversity, as filed (1)
| Reporting year | Workforce % women | Board % women |
|---|---|---|
| 2025 | not disclosed | not disclosed |
Organization relationships (75)
Shown in this order: most evidence first.
| Related organization | Relationship | Evidence |
|---|---|---|
| Government of the United States of America | Co sponsors trial with | 5 |
| University of Missouri System | Co sponsors trial with | 4 |
| Emory University | Co sponsors trial with | 4 |
| Washington University in St. Louis | Co sponsors trial with | 3 |
| The University of Texas System | Co sponsors trial with | 3 |
Showing 5 of 75 relationships.
Latest dated events (345)
Shown in this order: newest first.
| Date | Event | Source |
|---|---|---|
| 2026-06-02 | Financing | Funding rounds and grants (NIH RePORTER + SEC EDGAR) |
| 2026-05-29 | Trial started | Clinical trials (ClinicalTrials.gov) (ClinicalTrials.gov, U.S. National Library of Medicine; AACT database, Clinical Trials Transformation Initiative (CTTI)) |
| 2026-04-27 | Debt facility | Funding rounds and grants (NIH RePORTER + SEC EDGAR) |
| 2026-04-27 | Debt facility | Funding rounds and grants (NIH RePORTER + SEC EDGAR) |
| 2026-04-20 | Trial started | Clinical trials (ClinicalTrials.gov) (ClinicalTrials.gov, U.S. National Library of Medicine; AACT database, Clinical Trials Transformation Initiative (CTTI)) |
Showing 5 of 345 events.
CLIA clinical laboratory certificates (1)
| Lab | Certificate | Status |
|---|---|---|
| BIOMARIN PHARMACEUTICAL INC | Certificate of compliance | Terminated |
Biomarker capability evidence (332)
Shown in this order: tier desc, then gene symbol.
| Gene | Role | Evidence |
|---|---|---|
| ABCC6 | Trial collaborator | Named in trial inclusion criteria |
| ABR | Trial sponsor | Primary endpoint in a Phase 3 trial |
| ACAN | Trial sponsor | Named in trial inclusion criteria |
| ACD | Trial sponsor | Named in trial inclusion criteria |
| ACD | Trial collaborator | Named in trial inclusion criteria |
Showing 5 of 332 evidence rows.
Not on this page
Checked, with nothing recorded for this organization: related companies not grouped here, fda warning letters, fda untitled letters, fda recalls, and biomarkers this organization publishes on.