BIOMARIN PHARMACEUTICAL INC
BMRN evidence brief
What we can confirm
What it means
What to watch
Prepared research question
What matters most for BMRN—catalysts, financial risk and supporting evidence?
Traceable citations · Unknowns marked
BioSniper AI · BMRN
BioMarin Pharmaceutical is a global rare disease biotechnology company focused on developing and commercializing therapies for genetically defined conditions, with a portfolio of marketed products and a clinical pipeline.
Key risk: High debt levels
BioSniper Score
Based on 4 of 6 signals
Financial data as of Jun 30, 2026.
Tracking 30 clinical trials · 5 catalysts for BIOMARIN PHARMACEUTICAL INC.
BioMarin Pharmaceutical is a global rare disease biotechnology company focused on developing and commercializing therapies for genetically defined conditions, with a portfolio of marketed products and a clinical pipeline.
- BioMarin is a leading global rare disease biotechnology company founded in 1997, headquartered in San Rafael, California [sec_mda · as of 2026-05-05].
- As of 2026-06-30, the company reported total revenue of $989.7M, net income of $44.8M, cash & equivalents of $874.0M, shareholders' equity of $6,087.0M, and total debt of $4,844.3M [sec].
- The company has a portfolio of commercial therapies and a strong clinical and preclinical pipeline [sec_mda · as of 2026-05-05].
- In 2025, BioMarin achieved $3.2 billion in total revenues, including significant contribution from the expansion of VOXZOGO [sec_mda · as of 2026-02-26].
- The company faces potential liquidity needs, including repayment of convertible debt due in 2027, and may require additional financing [sec_mda · as of 2025-10-28].
What works
- BioMarin has a proven track record of innovation with a portfolio of commercial therapies and a strong clinical and preclinical pipeline [sec_mda · as of 2026-05-05].
- The company achieved $3.2 billion in total revenues in 2025, including significant contribution from the ongoing expansion of VOXZOGO [sec_mda · as of 2026-02-26].
- BioMarin reported net income of $44.8M for the six months ended June 30, 2026, indicating profitability in the period [sec_mda · as of 2026-08-06].
What to weigh
- BioMarin has substantial total debt of $4,844.3M as of 2026-06-30, which may impact financial flexibility [sec].
- The company may need to raise additional funds to repay convertible debt due in 2027 and for future operations, including commercialization and clinical trials [sec_mda · as of 2025-10-28].
- Macroeconomic conditions such as inflation, tariffs, trade tensions, and geopolitical instability could impact global revenue sources and business operations [sec_mda · as of 2026-08-06].
From the filings
Quoted directly from source documents.
BioMarin is a leading global rare disease biotechnology company.
“We are a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions.”
SEC MD&A · May 5, 2026
BioMarin achieved $3.2 billion in total revenues in 2025.
“In 2025, we achieved $3.2 billion in total revenues, including a significant contribution from our ongoing expansion of VOXZOGO”
SEC MD&A · February 26, 2026
BioMarin may need to raise additional funds for convertible debt repayment.
“We will need to raise additional funds by issuing equity, debt or convertible securities, taking loans or entering into collaborative or other agreements if we are unable to satisfy our liquidity requirements.”
SEC MD&A · October 28, 2025
Catalyst timeline
- 2027-10-01 · Phase3_Topline · phenylketonuria- — Phase 3 Topline: A Phase 3 Multi-Center Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegva — Topline data from a Phase 3 study of pegvaliase for phenylketonuria will be a key readout for the pipeline.
- 2029-03-01 · Phase3_Topline — Phase 3 Topline: GENEr8-JPN: A Phase 3 Open-Label, Single-Arm Study to Evaluate the Efficacy and Safety of BMN 270, a — Topline data from the Japan-specific Phase 3 study of valoctocogene roxaparvovec for hemophilia A will inform regulatory strategy.
- 2029-09-01 · Phase3_Topline — Phase 3 Topline: A Multicenter, Randomized, Operationally Seamless Phase 2/3 Study to Evaluate the Efficacy and Safet — Topline data from a seamless Phase 2/3 study will provide efficacy and safety results for an undisclosed candidate.
- 2031-06-01 · Phase3_Topline — Phase 3 Topline: A Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Ch — Long-term extension data for vosoritide in children with achondroplasia will assess durability of safety and efficacy.
- 2040-12-01 · Phase3_Topline — Phase 3 Topline: A Phase 3, Open-Label, Long-Term Extension Study to Evaluate the Safety and Efficacy of Vosoritide i — Very long-term extension data for vosoritide will provide extended safety and efficacy information.
Risks & what to watch (3)›
Key risks
- High debt levels — Total debt of $4,844.3M as of 2026-06-30 may constrain financial flexibility [sec].
- Liquidity and financing needs — May require additional financing for convertible debt due in 2027 and future operations [sec_mda · as of 2025-10-28].
- Macroeconomic uncertainty — Inflation, tariffs, trade tensions, and geopolitical instability could impact revenue and operations [sec_mda · as of 2026-08-06].
What to watch
- Phase 3 topline data for phenylketonuria candidate (pegvaliase) expected around 2027-10-01 [catalyst].
- Phase 3 topline data for BMN 270 (valoctocogene roxaparvovec) in Japan expected around 2029-03-01 [catalyst].
- Phase 2/3 topline data for an undisclosed candidate expected around 2029-09-01 [catalyst].
- Phase 3 topline data for BMN 111 (vosoritide) long-term extension expected around 2031-06-01 [catalyst].
Sources: SEC filings · SEC MD&A · Generated August 29, 2026 · How we verify sources →
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Evidence & data
Score breakdownBased on 4 of 6 signals · derived from public data
No clinical pipeline tracked — this score reflects financial & catalyst signals only.
Profitable (positive operating cash flow)
D/E 0.85 · 1% dilution
No tracked pipeline phase
No tracked pipeline candidates
5 upcoming, next in ~397d
0 buys · 2 sells (180d)
Catalysts
Regulatory
Financials
Institutional Holdings
Source: SEC Form 13F · as of Mar 2026Institutional holders
553
Total value held
$10.83B
| Holder | Shares | Value |
|---|---|---|
| blackrock, inc. | 19,818,301 | $1.12B |
| vanguard capital management llc | 17,286,328 | $976.5M |
| dodge & cox | 14,561,759 | $822.6M |
| primecap management co/ca/ | 14,194,005 | $801.8M |
| vanguard portfolio management llc | 8,298,475 | $468.8M |
| aqr capital management llc | 7,676,541 | $429.3M |
| state street corp | 7,295,079 | $412.1M |
| viking global investors lp | 7,062,077 | $398.9M |
| citadel advisors llc | 4,697,834 | $265.4M |
| geode capital management, llc | 3,819,441 | $215.8M |
Showing top 10 of 553 institutional holders of BIOMARIN PHARMACEUTICAL INC. Open the full 13F history after sign-in.
Short Interest
as of May 20, 2026- % of float
- 5.6%
- Days to cover
- 6.9
- Shares short
- 10,792,859
- Daily short volume
- -
Short percent of float and days-to-cover reflect reported short positioning. Daily short volume is the exchange-reported short share of a single day's trading volume and is noisier. See the most-shorted biotech ranking →
BMRN short interestGovernance
Frequently asked questions
What is the AI investment thesis for BIOMARIN PHARMACEUTICAL INC?
BioMarin Pharmaceutical is a global rare disease biotechnology company focused on developing and commercializing therapies for genetically defined conditions, with a portfolio of marketed products and a clinical pipeline.
What is BIOMARIN PHARMACEUTICAL INC's next catalyst?
BIOMARIN PHARMACEUTICAL INC's next tracked catalyst is a Phase3_Topline event — "Phase 3 Topline: A Phase 3 Multi-Center Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegva" — expected October 1, 2027.
Who is the CEO of BIOMARIN PHARMACEUTICAL INC?
Mr. Alexander Hardy is the chief executive officer of BIOMARIN PHARMACEUTICAL INC (BMRN).
Where is BIOMARIN PHARMACEUTICAL INC headquartered?
BIOMARIN PHARMACEUTICAL INC (BMRN) is headquartered in San Rafael, CA, United States.
What therapeutic areas does BIOMARIN PHARMACEUTICAL INC focus on?
BIOMARIN PHARMACEUTICAL INC develops therapies across Oncology, Rare Disease, Gene Therapy, Immunology, Dermatology, Hematology, Endocrinology.
The AI overview and BioSniper Score are neutral, data-derived signals built from public SEC filings, ClinicalTrials.gov and FDA records — not investment advice.