BMRN Stock — BioMarin Pharmaceutical Inc.: Cash, EV, Pipeline

What they do

Our San Rafael, California-based company, founded in 1997, has a proven track record of innovation, with eight commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, we seek to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients.

Source: 10-K FY2025 via SEC EDGAR

Share price

$56.03

Market cap

$10,852.7M

Cash

$736.0M

Enterprise value

$14,290.4M

Balance sheet

Cash	$736.0M
Debt	$4,173.7M
Net cash	-$3,437.7M
Enterprise value	$14,290.4M
Basic shares	193.7M shares
Fully diluted shares	211.2M shares
Fully diluted market cap	$11,835.7M
Cash per share	$3.80
Quarterly burn	+$168.1M generated

Cash figures as of 2026-09-30. Database snapshot: 2026-11-07.

Share structure

Common

193.5M

Pre-funded warrants

0.0M

Options

5.6M

RSUs

5.7M

Warrants

0.0M

Converts

4.4M

Conv preferred

0.0M

ESPP reserve

2.0M

FDSO

211.2M

Cap table as of 2026-06-30.

Price history

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Next catalyst

BMN 349 · Now - Nov 11, 2026 Now · Phase 1 · Alpha 1-Antitrypsin Deficiency

Recently reported

BMN 351 Phase 1/2 Negative · 2026-10-02

Source release →

Upcoming catalysts

Expected	Drug	Phase	Indication	Mgmt guide
Now - Nov 11, 2026 Now	BMN 349	Phase 1	Alpha 1-Antitrypsin Deficiency	—
Nov 25, 2026 - Jan 6, 2027 in 18d	INZ-701	Phase 3	Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency; Autosomal Recessive Hypophosphatemic Rickets; Generalized Arterial Calcification of Infancy 1	—
Nov 25, 2026 - Jan 6, 2027 in 18d	INZ-701	Phase 1	Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency; Autosomal Recessive Hypophosphatemic Rickets; Generalized Arterial Calcification of Infancy	—
GUIDEDFeb 28, 2027 in 16 wks	vosoritide (VOXZOGO) sNDA PDUFA for full approval in children with achondroplasia	Not disclosed	full approval	February 28, 2027
Apr 12 - May 24 in 5 mo	Vosoritide Injection	Phase 2	Noonan Syndrome	—
May 26 - Jul 7 in 7 mo	BMN 351	Phase 2	Duchenne Muscular Dystrophy	—
GUIDEDEst. Jul 2, 2027 in 8 wks guidance midpoint	BMN 333 registration-enabling Phase 2/3 study in achondroplasia, data update from dose-finding segment	Phase 2/3	achondroplasia	2027
Jan 12 - Feb 23 in 14 mo	BMN 111	Phase 2	Achondroplasia	—
Jan 12 - Feb 23 in 14 mo	BMN 307	Phase 2	Phenylketonuria (PKU)	—
Jan 12 - Feb 23 in 14 mo	vosoritide	Phase 2	Achondroplasia	—
Aug 11 - Sep 22 in 21 mo	Vosoritide	Phase 2	Hypochondroplasia	—
Jul 13 - Aug 24 in 33 mo	BMN 333	Phase 3	Achondroplasia	—
Jul 13 - Aug 24 in 57 mo	BMN 111	Phase 3	Achondroplasia	—
Jan 12 - Feb 23 in 63 mo	BMN 351	Phase 2	Duchenne Muscular Dystrophy (DMD)	—
Nov 12 - Dec 24 in 219 mo	Vosoritide	Phase 3	Hypochondroplasia	—

Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.

Full readout calendar →

Pipeline assets

VOXZOGO

Approved · Active

Aliases vosoritide

Target FGFR3 pathway

Indications

Achondroplasia (Approved)
Hypochondroplasia (Phase 3)
Idiopathic short stature (Stage not disclosed)
Noonan syndrome (Stage not disclosed)

Catalysts

— February 28, 2027

Status note Phase 3 CANOPY-HCH-3 study in hypochondroplasia met its primary endpoint (May 2026); sNDA submitted July 2026.

PALYNZIQ

Approved · Active

Aliases pegvaliase-pqpz

Target phenylalanine

Indications

Phenylketonuria (PKU) (Approved)

Status note European Commission approved for adolescents 12+ (Jun 2026); FDA approved for adolescents 12+ (Feb 2026).

BRINEURA

Approved · Active

Aliases cerliponase alfa

Target TPP1

Indications

CLN2 disease (Batten disease) (Approved)

Status note Approved commercial product.

NAGLAZYME

Approved · Active

Aliases galsulfase

Target glycosaminoglycans (GAGs)

Indications

Mucopolysaccharidosis VI (MPS VI) (Approved)

Status note Approved commercial product.

KUVAN

Approved · Active

Aliases sapropterin dihydrochloride

Target PAH

Indications

Phenylketonuria (PKU) (Approved)

Status note Generic versions available in the U.S. and EU, adversely affecting revenues.

ALDURAZYME

Approved · Active

Aliases laronidase

Target glycosaminoglycans (GAGs)

Partnerships Marketed worldwide by Sanofi

Indications

Mucopolysaccharidosis I (MPS I) (Approved)

Status note Marketed worldwide by Sanofi.

GALAFOLD

Approved · Active

Aliases migalastat

Indications

Fabry disease (Approved)

Status note First oral treatment for Fabry disease; added via Amicus acquisition (closed April 27, 2026).

POMBILITI + OPFOLDA

Approved · Active

Aliases cipaglucosidase alfa-atga, miglustat

Indications

Pompe disease (Approved)

Status note Two-component therapy added via Amicus acquisition (closed April 27, 2026).

BMN 820

Phase 3 · Active

Aliases DMX-200

Target CCR2

Partnerships Dimerix: U.S. commercialization rights licensed from Dimerix; up to $510M milestones plus tiered royalties low-teens to low-twenties

Indications

Focal segmental glomerulosclerosis (FSGS) (Phase 3)

Status note Potential first-in-class oral CCR2 inhibitor for a rare fatal kidney disease; U.S. rights acquired via Amicus acquisition.

BMN 333

Phase 2/3 · Active Modality: long-acting C-type natriuretic peptide (CNP)

Target CNP

Indications

Achondroplasia (Phase 2/3)

Status note Registration-enabling Phase 2/3 study; first patient enrolled April 2026.

ROCTAVIAN

Approved · Terminated

Target F8 (factor VIII)

Indications

Severe hemophilia A (Approved)

Status note In 2026 the company announced it will no longer market ROCTAVIAN.

BMN 401

Phase 3 · Terminated Modality: enzyme replacement therapy

Aliases INZ-701

Target ENPP1

Indications

ENPP1 deficiency (Phase 3)

Status note Did not meet one of two co-primary endpoints in the pivotal ENERGY 3 trial; development discontinued across all indications August 2026. Acquired via Inozyme Pharma (Jul 2025).

Per-asset detail extracted from the 10-Q filed 2026-08-06. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.

Pipeline

Lead drug	Pegvaliase
Lead indication	Phenylketonuria
Lead phase	Phase 4
Active trials	27
Collaborator trials	7

Active clinical trials

Trial	Phase	Status	Role	Sponsor	BP	Condition(s)	Mgmt guide
NCT07441876	Phase 3	Recruiting	Sponsor	BMRN	—	Achondroplasia	2027 Est. Jul 2, 2027 · guidance midpoint
NCT03424018	Phase 3	Active, not recruiting	Sponsor	BMRN	—	Achondroplasia	—
NCT07073014	Phase 3	Enrolling by invitation	Sponsor	BMRN	—	Hypochondroplasia	—
NCT05270837	Phase 3	Active, not recruiting	Sponsor	BMRN	—	Phenylketonuria (PKU)	—
NCT06224907	Phase 3	Active, not recruiting	Sponsor	BMRN	—	Hemophilia A	—
NCT06455059	Phase 3	Active, not recruiting	Sponsor	BMRN	—	Hypochondroplasia	—
NCT06668805	Phase 2	Recruiting	Sponsor	BMRN	—	Noonan Syndrome	—
NCT06280209	Phase 2	Active, not recruiting	Sponsor	BMRN	—	Duchenne Muscular Dystrophy	—
NCT02724228	Phase 2	Active, not recruiting	Sponsor	BMRN	—	Achondroplasia	—
NCT04480567	Phase 2	Active, not recruiting	Sponsor	BMRN	—	Phenylketonuria (PKU)	—
NCT04554940	Phase 2	Active, not recruiting	Sponsor	BMRN	—	Achondroplasia	—
NCT07126262	Phase 2	Recruiting	Sponsor	BMRN	—	Hypochondroplasia	—

Sponsor-matched active trials from ClinicalTrials.gov (27 as sponsor, 7 as collaborator); "Sponsor" = company is the lead sponsor, "Collaborator" = company is a collaborator. The BP column flags big-pharma involvement as lead sponsor or collaborator (excluding the company itself). The Mgmt guide column shows what management guided for readout timing when a guided program matches the trial (hover for the quote, click for the source). Trial listings are not a company pipeline slide.

View all 34 active trials for BioMarin Pharmaceutical on ClinicalTrials.gov

Specialist fund holders

Tracked specialist funds: ADAR1 Capital Management, Aisling Capital, Ally Bridge Group, Avoro Capital Advisors, BVF Partners, Baker Bros. Advisors, Boxer Capital, Braidwell LP, Casdin Capital, Cormorant Asset Management, DAFNA Capital, Deerfield Management, EcoR1 Capital, Frazier Life Sciences, Great Point Partners, Ikarian Capital, Logos Global Management, OrbiMed Advisors, Perceptive Advisors, RA Capital Management, RTW Investments, Sphera Biotech, Stonepine Capital, Tang Capital Partners, Vivo Capital.

Fund	Shares	Q/Q change	% of portfolio	Filing
Deerfield Management	1,332,836	+481,280	0.80%	2026-08-14
Sphera Biotech	328,550	+188,650	2.69%	2026-08-14
ADAR1 Capital Management	49,000	+30,800	0.13%	2026-08-14
Braidwell LP	0	-497,020	0.00%	2026-08-14

Specialist-fund positions from 13F filings. Q/Q change is vs the prior quarter's filing; % of portfolio is the fund's own reported portfolio weight.

Latest news

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BMRN — BioMarin Pharmaceutical Inc. ☆

What they do

Balance sheet

Share structure

Price history

Next catalyst

Recently reported

Upcoming catalysts

Pipeline assets

Pipeline

Active clinical trials

Specialist fund holders

Latest news

BMRN — BioMarin Pharmaceutical Inc.