BioMarin Pharmaceutical

bmrnNASDAQModerate Risk

Enzyme replacement and gene therapy

Reviewed by Blane Jackson, DDS, MBA. Educational analysis only. Read the editorial policy and disclosures.

Rare Diseases Genetic Disorders

Market cap

Large cap

Cash position

$1.2B as of Q4 2025

Q4 2025 — 3 quarters ago

Revenue status

commercial stage

Pipeline assets

3 programs

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What does BioMarin Pharmaceutical do?

BioMarin is a well-established rare disease company that has successfully transitioned to commercial profitability. Their flagship product Voxzogo treats achondroplasia with strong uptake worldwide. Their gene therapy Roctavian for hemophilia A received FDA approval but adoption has been slower than expected. The company generates meaningful revenue and is cash flow positive. For investors BMRN represents a lower-risk biotech with real revenue growth though the stock price reflects much of the near-term opportunity. Key questions are whether Voxzogo reaches peak sales and whether Roctavian adoption accelerates.

What to watch

Quarterly Voxzogo revenue growth trajectory

Roctavian treatment center expansion and uptake

BMN 349 Phase 1 safety and PK data

Competitive readouts from Ascendis Pharma

Operating margin expansion as products mature

Pipeline

Drug	Indication	Phase	Expected data
Voxzogo	Achondroplasia	Approved	Not disclosed	▼
Roctavian	Hemophilia A	Approved	Not disclosed	▼
BMN 349	Complement-mediated diseases	Phase 1	Not disclosed	▼

Investment thesis

Bull case

BioMarin has transformed into a profitable rare disease leader with Voxzogo on a strong growth trajectory and peak sales estimates of $2-3B globally. Deep expertise in rare diseases with multiple approved products generating recurring revenue. Pipeline expanding beyond enzyme replacement into gene therapy and small molecules. Strong cash flow with no near-term dilution risk offers rare disease growth with commercial-stage stability.

Bear case

Roctavian uptake disappointing and may never reach peak sales estimates. Voxzogo faces potential competition from Ascendis Pharma TransCon CNP. Rare disease market inherently limited in size. Gene therapy manufacturing remains complex and expensive. Pipeline complement diversification is very early stage with high clinical risk.

Key upcoming catalysts

BMN 349 Phase 1 interim data

2026-09-01

Unverified — this date has not been checked against a primary source.

Data ReadoutModerate

Risk factors

⚠

Roctavian commercial adoption below expectations

⚠

Voxzogo competitive threats from next-gen therapies

⚠

Gene therapy manufacturing complexity

⚠

Rare disease market size limitations

⚠

Regulatory risk for gene therapy label expansions

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Financial snapshot

These figures are drafted by our AI pipeline, not read from SEC filings. Confirm anything you would act on against the company's latest 10-Q or 10-K.

Cash

$1.2B as of Q4 2025

Q4 2025 — 3 quarters ago

Quarterly burn

Cash flow positive

Cash runway

Not derivable

cash or burn not disclosed as a figure

Revenue

commercial stage

Institutional ownership

95%

Source: company profile text. These figures come from the company profile text, not from a filing we have parsed. Check them against the latest 10-Q before relying on them.

These figures are out of date

The cash position cited here is from Q4 2025, roughly 3 quarters ago. The company has almost certainly reported since. Check its latest filing before using any of this.

Sponsor's late-stage trial record

BioMarin Pharmaceutical has 70 Phase 2 or 3 studies on ClinicalTrials.gov: 41 completed, 13 stopped early, 16 ongoing.

Completed

Stopped early

Ongoing

Stopped early

GENEr8-AAV5+Phase 1/2 · started 2018 · terminated
Valoctocogene Roxaparvovec in hemophilia a · n=3
Reason given: Early rollover into long-term extension study
NCT02958202Phase 2 · started 2016 · terminated
BMN 044 IV 6 mg/kg in duchenne muscular dystrophy · n=7
Reason given: Regulatory approval was not obtained for drisapersen, hence BioMarin is stopping the development of all exon skipping oligonucleotides in DMD.
NCT02329769Phase 2 · started 2014 · terminated
PRO044 SC 6 mg/kg in duchenne muscular dystrophy · n=15
No reason recorded in the registry.
NCT01924845Phase 3 · started 2014 · terminated
BMN 701 in late-onset pompe disease · n=24
Reason given: Sponsor decision
SIGNALPhase 2 · started 2014 · terminated
Sapropterin in phenylketonuria · n=2
Reason given: Recruitment challenges
NCT01957059Phase 1/2 · started 2013 · terminated
Regimen Selection Phase Group 2 in duchenne muscular dystrophy · n=9
No reason recorded in the registry.
NCT01803412Phase 3 · started 2013 · terminated
Drisapersen in muscular dystrophies · n=53
No reason recorded in the registry.
NCT01826474Phase 1/2 · started 2013 · terminated
PRO045, 0.15 mg/kg/week in duchenne muscular dystrophy · n=15
No reason recorded in the registry.
NCT01697319Phase 2 · started 2012 · terminated
BMN 110 in mucopolysaccharidosis iva · n=16
No reason recorded in the registry.
NCT01609062Phase 2 · started 2012 · terminated
BMN 110 in mucopolysaccharidosis iva · n=25
No reason recorded in the registry.
NCT01435772Phase 2 · started 2011 · terminated
BMN 701 in pompe disease · n=21
Reason given: The study was terminated because BioMarin decided to end the overall development program based on competing corporate priorities.
NCT01242111Phase 1/2 · started 2010 · terminated
BMN 110 in mps iv a · n=20
No reason recorded in the registry.
NCT01910649Phase 2 · started 2008 · terminated
Drisapersen in muscular dystrophies · n=12
Reason given: Regulatory approval was not obtained for drisapersen, hence BioMarin is stopping the development of all exon skipping oligonucleotides in DMD.

Counts cover interventional Phase 2 and Phase 3 studies registered under this sponsor name, retrieved 2026-09-13 from ClinicalTrials.gov API v2. Trials stop for many reasons — futility, safety, enrollment, funding, or a decision to back a different programme — and the registry often does not say which. This is the sponsor's record, not a prediction about any specific readout.