BIOMARIN PHARMACEUTICAL INC
BMRN evidence brief
What we can confirm
- BioSniper Score:
- 6.5 / 10
- Market Cap:
- $12.75B
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 Inc. (BMRN) is a leading global rare disease biotechnology company with nine commercial therapies, a strong pipeline, and recent acquisitions expanding its portfolio, though it faces development setbacks and macroeconomic uncertainties.
Key risk: Pipeline setback
BioSniper Score
Based on 2 of 6 signals
Limited data coverage
Market Cap
$12.75B
Cash Runway
—
Lead Asset
—
Next Catalyst
—
Financial data as of Jun 30, 2026.
Tracking 30 clinical trials for BIOMARIN PHARMACEUTICAL INC.
BioMarin Pharmaceutical Inc. (BMRN) is a leading global rare disease biotechnology company with nine commercial therapies, a strong pipeline, and recent acquisitions expanding its portfolio, though it faces development setbacks and macroeconomic uncertainties.
- BioMarin is a leading, global rare disease biotechnology company with nine commercial therapies and a strong clinical and preclinical pipeline [sec_mda · as of 2026-08-06].
- As of 2026-06-30, the company reported cash & equivalents of $874.0M, total revenue of $989.7M, net income of $44.8M, shareholders' equity of $6,321.7M, and total debt of $4,250.0M [sec].
- In April 2026, BioMarin acquired Amicus Therapeutics, adding GALAFOLD and POMBILITI + OPFOLDA to its commercial portfolio [sec_mda · as of 2026-08-06].
- In August 2026, BioMarin discontinued development of BMN 401 (for ENPP1 deficiency) after the pivotal ENERGY 3 trial data readout [sec_mda · as of 2026-08-06].
- In August 2026, BioMarin acquired Alesta Therapeutics for $275 million upfront, gaining access to ALE1, an oral treatment for hypophosphatasia (HPP) in early-stage trials [news · published 2026-08-19].
What works
- BioMarin has a proven track record of innovation with nine commercial therapies and a strong pipeline, demonstrating commercial presence in rare disease [sec_mda · as of 2026-08-06].
- The company delivered revenue growth of 18% in 2024 and 13% in 2025, with improved profitability and cash flow generation [sec · filed 2026-04-21].
- Recent acquisitions (Amicus, Alesta) expand the commercial portfolio and add pipeline assets, leveraging BioMarin's rare disease capabilities [sec_mda · as of 2026-08-06] [news · published 2026-08-19].
What to weigh
- BioMarin discontinued development of BMN 401 (ENPP1 deficiency) after the pivotal ENERGY 3 trial failed to meet endpoints, indicating pipeline risk [sec_mda · as of 2026-08-06].
- The company carries substantial total debt of $4,250.0M as of 2026-06-30, which may constrain financial flexibility [sec].
- Macroeconomic uncertainties such as inflation, tariffs, and geopolitical instability could impact global revenue sources and operations [sec_mda · as of 2026-08-06].
From the filings
Quoted directly from source documents.
BioMarin is a leading rare disease company with nine commercial therapies
“We are a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, our San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline.”
SEC MD&A · August 6, 2026
BioMarin discontinued BMN 401 after ENERGY 3 trial
“Following the pivotal ENERGY 3 trial data readout, in August 2026, we made the decision to discontinue development of BMN 401 across all indications.”
SEC MD&A · August 6, 2026
BioMarin acquired Alesta Therapeutics for $275 million upfront
“BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is expanding its rare-disease business with the acquisition of Alesta Therapeutics for $275 million upfront.”
News · August 19, 2026
BioMarin delivered 13% revenue growth in 2025
“Across BioMarin's 80-country footprint, the company delivered revenue growth of 18% in 2024 and 13% in 2025.”
SEC filings · April 21, 2026
Risks & what to watch (3)›
Key risks
- Pipeline setback — Discontinued BMN 401 after ENERGY 3 trial failure [sec_mda · as of 2026-08-06]
- High debt load — Total debt of $4,250.0M as of 2026-06-30 [sec]
- Macroeconomic uncertainty — Inflation, tariffs, geopolitical instability could impact revenue [sec_mda · as of 2026-08-06]
What to watch
- Progress of ALE1 (HPP) from Alesta acquisition, still in early-stage trials [news · published 2026-08-19]
- Integration and revenue contribution from Amicus acquisition (GALAFOLD, POMBILITI + OPFOLDA) [sec_mda · as of 2026-08-06]
- Next-generation long-acting CNP therapy for achondroplasia and Duchenne muscular dystrophy program [sec · filed 2026-04-21]
- Impact of macroeconomic factors on global operations [sec_mda · as of 2026-08-06]
Sources: Catalysts · News · SEC filings · SEC MD&A · Generated October 7, 2026 · How we verify sources →
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Evidence & data
Score breakdownBased on 2 of 6 signals · derived from public data
Pipeline inputs are unavailable for this score. It uses only the available signals shown below.
Cash runway data unavailable
D/E 0.85 · 1% dilution
No tracked pipeline phase
No tracked pipeline candidates
No upcoming catalysts tracked
0 buys · 1 sell (180d)
Regulatory
Financials
Institutional Holdings
Source: SEC Form 13F · as of Mar 2026Institutional holders
550
Total value held
$10.12B
| Holder | Shares | Value |
|---|---|---|
| blackrock, inc. | 19,818,301 | $1.12B |
| dodge & cox | 14,561,759 | $822.6M |
| primecap management co/ca/ | 14,194,005 | $801.8M |
| vanguard capital management llc | 8,643,164 | $488.3M |
| 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 | 4,512,634 | $254.9M |
| geode capital management, llc | 3,819,441 | $215.8M |
Showing top 10 of 550 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 Inc. (BMRN) is a leading global rare disease biotechnology company with nine commercial therapies, a strong pipeline, and recent acquisitions expanding its portfolio, though it faces development setbacks and macroeconomic uncertainties.
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.